This section highlights FDA-related milestones and regulatory updates for drugs developed by Regeneron Pharmaceuticals (REGN).
Over the past two years, Regeneron Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Fianlimab, Otarmeni, EYLEA, Dupixent, olatorepatide, garetosmab, and Aflibercept. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Fianlimab FDA Regulatory Events
Fianlimab is a drug developed by Regeneron Pharmaceuticals for the following indication: Targeting the immune checkpoint receptor LAG-3 on T cells.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Fianlimab
- Announced Date:
- May 15, 2026
- Indication:
- Targeting the immune checkpoint receptor LAG-3 on T cells
Announcement
Regeneron Pharmaceuticals, Inc today reviewed results from the Phase 3 trial evaluating two dose levels of fianlimab (LAG-3 inhibitor) in combination with cemiplimab (PD-1 inhibitor) as a first-line treatment for patients with unresectable locally advanced or metastatic melanoma.
AI Summary
Regeneron Pharmaceuticals said results from a Phase 3 trial of fianlimab, a LAG-3 inhibitor, plus cemiplimab, a PD-1 inhibitor, in first-line treatment for unresectable locally advanced or metastatic melanoma did not meet the study’s main goal. The trial did not show a statistically significant improvement in progression-free survival compared with pembrolizumab alone.
Even so, the high-dose fianlimab combination showed a median PFS improvement of 5.1 months. Regeneron said a separate Phase 3 head-to-head study testing the high-dose combination against Opdualag is still underway. The company also said full results will be shared at an upcoming medical meeting.
The treatment is still investigational, and its safety and effectiveness have not been reviewed by any regulatory authority.
Read Announcement
Otarmeni FDA Regulatory Events
Otarmeni is a drug developed by Regeneron Pharmaceuticals for the following indication: First and Only Gene Therapy for Genetic Hearing Loss.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Otarmeni
- Announced Date:
- April 23, 2026
- Indication:
- First and Only Gene Therapy for Genetic Hearing Loss
Announcement
Regeneron Pharmaceuticals, Inc announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni™ (lunsotogene parvec-cwha), the first gene therapy and second new molecular entity approved under the FDA Commissioner's National Priority Voucher program.
AI Summary
The U.S. Food and Drug Administration granted accelerated approval to Otarmeni™ (lunsotogene parvec-cwha), a gene therapy to treat an ultra‑rare form of genetic hearing loss. The decision allows Regeneron to make the therapy available sooner for patients with this serious condition while further confirmatory studies continue.
Otarmeni is notable as the first gene therapy and only the second new molecular entity approved under the FDA Commissioner’s National Priority Voucher program, a policy designed to speed review of medicines that address urgent or unmet medical needs. That designation highlights the therapy’s public‑health importance and the agency’s focus on incentivizing treatments for rare disorders.
Regeneron said it will provide Otarmeni for free in the United States, a step aimed at widening access for eligible patients and supporting those affected by this rare genetic hearing loss.
Read Announcement
EYLEA (aflibercept) FDA Regulatory Timeline and Events
EYLEA (aflibercept) is a drug developed by Regeneron Pharmaceuticals for the following indication: Wet age-related macular degeneration (wet AMD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- EYLEA (aflibercept)
- Announced Date:
- April 2, 2026
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved the extension of dosing intervals for EYLEA HD® (aflibercept) up to every 20 weeks for patients with wet age-related macular degeneration (wAMD) and diabetic macular edema (DME) following one year of successful response based on visual and anatomic outcomes.
AI Summary
Regeneron said the U.S. Food and Drug Administration has approved extending dosing intervals for EYLEA HD® (aflibercept) up to every 20 weeks for patients with wet age-related macular degeneration (wAMD) and diabetic macular edema (DME). The longer interval applies after one year of a successful treatment response, judged by both visual outcomes and retinal anatomic measures. In practice, that means clinicians may space injections farther apart for patients who show stable vision and retinal health after the first 12 months of therapy.
This approval could reduce the number of injections and clinic visits for eligible patients, lowering treatment burden while maintaining close monitoring. Individual treatment plans should still be guided by a doctor’s assessment of visual and anatomic results, since not all patients will qualify for extended intervals and adjustments must reflect each patient’s clinical needs.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- August 21, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that the European Commission has approved Mynzepli® as a biosimilar to Eylea® (aflibercept), in a pre-filled syringe and vial.
AI Summary
Alvotech and Advanz Pharma announced that the European Commission has approved Mynzepli® (aflibercept) as a biosimilar to Eylea® for eye disease treatment. The product will come in a pre-filled syringe and a vial.
The approval covers all European Economic Area member states, including the EU’s 27 countries, Norway, Iceland, and Liechtenstein. Mynzepli® is approved for adults with neovascular (wet) age-related macular degeneration (AMD), macular edema from retinal vein occlusion (branch or central RVO), diabetic macular edema (DME), and myopic choroidal neovascularization (myopic CNV).
This decision was based on extensive analytical, non-clinical, and clinical data showing strong similarity to Eylea®, including a confirmatory efficacy study in patients with wet AMD.
Joseph McClellan, Chief Scientific and Technical Officer of Alvotech, said the approval will improve access to vision-preserving care across Europe. Nick Warwick, Chief Medical Officer of Advanz Pharma, called the milestone key to expanding affordable, high-quality treatment options.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- June 23, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human use (CHMP) adopted a positive opinion recommending approval for AVT06, Alvotech's proposed biosimilar to Eylea® (aflibercept 2 mg).
AI Summary
Alvotech announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion recommending approval for AVT06, its proposed biosimilar to Eylea® (aflibercept 2 mg). This important step means that AVT06 could soon receive marketing authorization by the European Commission for use in the European Economic Area, which includes the 27 European Union member states along with Norway, Iceland, and Liechtenstein.
The CHMP’s recommendation is a significant milestone for Alvotech as it moves closer to providing a high-quality, cost-effective treatment option for adults suffering from various eye disorders such as wet age-related macular degeneration and diabetic macular oedema. This positive progress is expected to increase access to vital biologic treatments for patients and their caregivers throughout Europe.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- February 18, 2025
- Target Action Date:
- Q4 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that The process to obtain regulatory approval is anticipated to be completed in the fourth quarter of 2025.
AI Summary
Alvotech announced that the U.S. Food and Drug Administration (FDA) is reviewing its Biologics License Application (BLA) for AVT06, a biosimilar candidate to Eylea®. The company expects that the process to obtain regulatory approval will be completed in the fourth quarter of 2025. This milestone is a key step in making AVT06 available as a treatment option for patients with eye disorders that can lead to vision loss or blindness. Alvotech believes that gaining approval for AVT06 will improve patient access by providing a cost-saving alternative. The company’s efforts highlight its strong focus on biosimilar development, supported by in-house research and manufacturing capabilities. This regulatory review is an important part of Alvotech’s strategy to expand its reach and offer high-quality, affordable treatments to patients worldwide.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- February 18, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that the U.S. Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for AVT06, Alvotech's proposed biosimilar to Eylea® (aflibercept), a biologic used to treat eye disorders, including diseases which can lead to vision loss or blindness.
AI Summary
Alvotech announced that the FDA has accepted for review a Biologics License Application (BLA) for AVT06, the company’s proposed biosimilar to Eylea® (aflibercept). Eylea® is a biologic medicine used to treat eye disorders that can lead to vision loss or blindness. With this acceptance, the FDA will review AVT06, marking an important step toward making an affordable treatment option available for patients with retinal diseases.
The approval process is expected to be completed by the fourth quarter of 2025. This milestone shows Alvotech’s strong capabilities in research, development, and manufacturing. By advancing AVT06 through the regulatory review, the company aims to offer improved access and cost-saving alternatives for patients and caregivers managing serious eye conditions.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- January 13, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Teva Pharmaceuticals announced that it has entered into a strategic collaboration with Klinge Biopharma GmbH (Klinge) and Formycon AG (FSE: FYB) for the semi-exclusive commercialization of FYB203, Formycon's biosimilar candidate to Eylea® (aflibercept) in Europe, excluding Italy, and in Israel.
AI Summary
Teva Pharmaceuticals International GmbH announced a strategic collaboration with Klinge Biopharma GmbH and Formycon AG for the semi-exclusive commercialization of FYB203, Formycon’s biosimilar candidate to Eylea® (aflibercept), in Europe (excluding Italy) and Israel. The partnership leverages Teva’s extensive marketing experience in biosimilars and its robust distribution network to help bring this promising treatment to patients suffering from severe retinal diseases. Under the agreement, FYB203 will be marketed under the brand name AHZANTIVE® once regulatory approval is secured. Klinge, which holds the exclusive global commercialization rights to FYB203 from Formycon, will benefit from milestone payments and revenue sharing. This move expands Teva’s biosimilars portfolio and builds on previous successful collaborations, reinforcing its commitment to improving patient access to high-quality and innovative treatments.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- September 11, 2024
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Regeneron Pharmaceuticals, Inc announced new analyses of EYLEA HD® (aflibercept) Injection 8 mg and EYLEA® (aflibercept) Injection 2 mg will be presented at the Annual Meeting of the European Society of Retina Specialists (EURETINA) in Barcelona from September 19 to 22, 2024.
AI Summary
Regeneron Pharmaceuticals announced that new analyses of its treatments, EYLEA HD® (aflibercept) Injection 8 mg and EYLEA® (aflibercept) Injection 2 mg, will be showcased at the Annual Meeting of the European Society of Retina Specialists (EURETINA) in Barcelona from September 19 to 22, 2024. These studies include several post-hoc analyses focusing on rapid and sustained control of retinal fluid, as well as consistent safety outcomes over two years in patients with wet age-related macular degeneration. One analysis applies disease activity criteria used in faricimab trials to explore extended dosing intervals, while others evaluate improvements in central retinal thickness and visual acuity. The findings aim to support EYLEA HD as a potential new standard of care by offering a treatment regimen that could extend the time between injections, benefiting both patients and retinal specialists.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- August 15, 2024
- Target Action Date:
- Q3 2025
- Estimated Target Date Range:
- July 1, 2025 - September 30, 2025
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that The process to obtain marketing authorization could be completed in the third quarter of 2025.
AI Summary
Alvotech has announced that the process to obtain marketing authorization for its biosimilar candidate AVT06 could be completed in the third quarter of 2025. The European Medicines Agency (EMA) has accepted the Marketing Authorization Application, which is a significant step forward for the company. AVT06 is designed as a biosimilar version of Eylea®, a well-known treatment for serious eye disorders such as wet Age-related Macular Degeneration. This milestone is expected to bring the biosimilar closer to being available in Europe, offering patients and caregivers more treatment options.
The targeted timeline of a Q3 2025 approval highlights Alvotech's commitment to advancing affordable biologic medicines through their end-to-end biosimilars platform. The progression of this application reflects ongoing efforts to make high-quality treatments more accessible to those in need.
Read Announcement- Drug:
- EYLEA (aflibercept)
- Announced Date:
- August 15, 2024
- Indication:
- Wet age-related macular degeneration (wet AMD)
Announcement
Alvotech announced that the European Medicines Agency (EMA) has accepted a Marketing Authorization Application for AVT06, Alvotech's proposed biosimilar to Eylea® (aflibercept).
AI Summary
Alvotech announced that the European Medicines Agency (EMA) has accepted its Marketing Authorization Application for AVT06, a proposed biosimilar to Eylea® (aflibercept). The acceptance marks an important milestone for the company and brings AVT06 one step closer to being available in Europe. Joseph McClellan, Chief Scientific Officer at Alvotech, highlighted that this development supports broader access to affordable biologic medicines for patients and caregivers.
The review process is expected to be completed in the third quarter of 2025. In addition, Advanz Pharma holds the responsibility for the registration and commercialization of AVT06 in most European countries, aiming to offer more treatment options for patients. This move reflects Alvotech’s commitment to leveraging its comprehensive biosimilars platform to increase market access to essential therapies.
Read Announcement
Dupixent (dupilumab) FDA Regulatory Timeline and Events
Dupixent (dupilumab) is a drug developed by Regeneron Pharmaceuticals for the following indication: Moderate-to-severe asthma.
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Dupixent (dupilumab)
- Announced Date:
- March 24, 2026
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has approved Dupixent® (dupilumab) for the treatment of adults with moderate-to-severe bullous pemphigoid (BP).
AI Summary
Regeneron and Sanofi announced that Japan’s Ministry of Health, Labour and Welfare has approved Dupixent® (dupilumab) for adults with moderate-to-severe bullous pemphigoid (BP). The approval was based on pivotal trial results showing more than four times as many Dupixent-treated patients achieved sustained disease remission through Week 36 compared with placebo. This is the seventh approved indication for Dupixent in Japan.
Bullous pemphigoid is a chronic, relapsing skin disease driven by type 2 inflammation, marked by intense itch and painful blisters or other lesions. In the trial, treatment-related adverse events occurred in 26% of Dupixent patients versus 15% with placebo; the most common treatment-related event with Dupixent was conjunctivitis (4%).
Dupixent is now available in Japan as a 300 mg pre-filled syringe or pen for adults. It is given by subcutaneous injection every two weeks after an initial loading dose and can be administered in clinic or at home after proper training.Read Announcement
- Drug:
- Dupixent (dupilumab)
- Announced Date:
- February 27, 2026
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Dupixent® (dupilumab) in the European Union (EU) for the treatment of chronic spontaneous urticaria (CSU).
AI Summary
Regeneron announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Dupixent (dupilumab) in the European Union for children aged 2 to 11 with chronic spontaneous urticaria (CSU) whose symptoms persist despite standard antihistamine treatment. If the European Commission approves the recommendation, Dupixent would become the first targeted therapy in the EU indicated for this young age group with CSU.
CSU is a chronic skin condition driven by type 2 inflammation that can cause long-lasting hives and intense itching. Dupixent is being developed jointly by Sanofi and Regeneron and is already approved for CSU in certain adults and adolescents in multiple countries; over 1.4 million patients use Dupixent globally across various indications. Regulators note the safety and efficacy of Dupixent specifically for children aged 2 to 11 with CSU have not yet been fully evaluated.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- February 24, 2026
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of adult and pediatric patients aged 6 years and older with allergic fungal rhinosinusitis (AFRS) who have a history of sino-nasal surgery.
AI Summary
Regeneron announced that the U.S. Food and Drug Administration approved Dupixent (dupilumab) for adults and children aged 6 and older with allergic fungal rhinosinusitis (AFRS) who have a history of sino-nasal surgery. Approval was supported by the Phase 3 LIBERTY-AFRS-AIMS trial, which showed Dupixent significantly reduced nasal signs and symptoms and lowered the need for systemic corticosteroids or additional surgery compared with placebo.
AFRS is a chronic type 2 inflammatory disease of the sinuses caused by an allergic reaction to fungi. It often requires surgery and has high rates of recurrence after operation. The trial’s primary endpoint measured change in sinus opacification by CT (Lund-Mackay score) at Week 52; key secondary measures included nasal congestion, nasal polyp score, and loss of smell, plus rates of surgery or systemic steroid use.
The safety findings aligned with prior studies in CRSwNP; common adverse reactions included injection site reactions, conjunctivitis, arthralgia, gastritis, insomnia, eosinophilia, and toothache. Dupilumab is being co-developed with Sanofi, and additional regulatory submissions are planned globally.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- February 10, 2026
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced 36 abstracts across its immunology and inflammation portfolio and pipeline will be presented at the 2026 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting being held February 27 to March 2 in Philadelphia, Pennsylvania.
AI Summary
Regeneron said 36 abstracts from its immunology and inflammation portfolio and pipeline will be presented at the 2026 AAAAI meeting, Feb. 27–Mar. 2 in Philadelphia. The program includes first‑time Phase 3 presentations for two investigational allergen‑blocking antibodies to treat cat and birch allergies. Cat and birch programs are part of a broader pipeline aiming to eliminate IgE‑mediated allergies; their safety and efficacy have not been evaluated by any regulatory authority.
New Dupixent (dupilumab) data will show real‑world impact across dermatology, respiratory and gastrointestinal diseases, including analyses of food‑allergy sensitization in children with atopic dermatitis. Two real‑world asthma analyses in adults and adolescents will compare adding Dupixent versus escalating inhaled corticosteroids or adding other biologics, measuring reductions in exacerbations and systemic corticosteroid use. Additional registration‑enabling trials for both allergy programs are initiating this year.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- December 23, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted marketing and manufacturing authorization for Dupixent® (dupilumab) for the treatment of bronchial asthma in children aged 6 to 11 years with severe or refractory disease whose symptoms are inadequately controlled with existing therapy.
AI Summary
Regeneron and Sanofi said Japan’s Ministry of Health, Labour and Welfare has approved Dupixent (dupilumab) for bronchial asthma in children 6–11 years with severe or refractory disease not controlled by current therapy. The decision was based on VOYAGE, a global Phase 3 trial, and supportive data from EXCURSION. In VOYAGE, adding Dupixent to standard care cut severe exacerbations by 54–65% and improved lung function by 4.68–5.32% versus placebo. Dupixent is the first and only biologic to show improved lung function in this young age group in a randomized Phase 3 trial; a Japanese pediatric sub-study showed lung function gains at 12 weeks and low exacerbation rates over one year.
Dupixent is a fully human antibody that blocks interleukin‑4 and interleukin‑13, two key drivers of type 2 inflammation in asthma. Common treatment-related side effects included injection-site reactions, fever, oral herpes and eosinophilia. In Japan it is available as 200 mg and 300 mg pre-filled syringes or pens for subcutaneous injection every two or four weeks based on weight; caregivers should administer doses for children under 12 after training.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- November 25, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that the European Commission (EC) has approved Dupixent® (dupilumab) for the treatment of moderate-to-severe chronic spontaneous urticaria (CSU) in adult and adolescent patients 12 years and above with inadequate response to histamine-1 antihistamines (H1AH) and who are naïve to anti-immunoglobulin E (IgE) therapy for CSU.
AI Summary
Regeneron and Sanofi announced the European Commission has approved Dupixent (dupilumab) for moderate-to-severe chronic spontaneous urticaria (CSU) in adults and adolescents 12 years and older who remain symptomatic despite H1 antihistamines and who are naïve to anti-IgE therapy. Eligible patients can use Dupixent as a first-line targeted treatment option.
The approval was based on the LIBERTY-CUPID Phase 3 program. Studies A and C (284 patients) evaluated Dupixent added to antihistamines versus antihistamines alone and showed significant reductions in itch and hives and higher rates of well-controlled disease or complete response at 24 weeks; Study B (108 patients) provided additional safety data for those intolerant or inadequate responders to anti-IgE therapy. Dupixent inhibits IL-4 and IL-13, central drivers of type 2 inflammation. In the EU, about 270,000 people aged 12+ have CSU not controlled by antihistamines. Common side effects included injection site reactions, eye inflammation, joint pain, oral herpes and eosinophilia.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- November 7, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced the presentation of positive results from the pivotal LIBERTY-AFRS-AIMS Phase 3 trial evaluating the investigational use of Dupixent® (dupilumab) in adults and children aged 6 years and older with allergic fungal rhinosinusitis (AFRS).
AI Summary
Regeneron and Sanofi reported positive Phase 3 results from the LIBERTY-AFRS-AIMS trial of Dupixent® (dupilumab) in adults and children aged 6 years and older with allergic fungal rhinosinusitis (AFRS). Compared to placebo, Dupixent significantly reduced sinus opacification on CT scans, eased nasal congestion, and shrank nasal polyps. These improvements were seen at both 24 and 52 weeks, marking the first-ever positive Phase 3 findings in AFRS.
In the 52-week, randomized trial of 62 patients, Dupixent cut the need for systemic corticosteroids or surgery by 92%. The safety profile matched expectations for approved respiratory uses, with similar overall adverse event rates between treatment and placebo groups.
The U.S. Food and Drug Administration has accepted the supplemental Biologics License Application (sBLA) for Dupixent in AFRS and granted Priority Review, targeting an action date of February 28, 2026. If approved, Dupixent would become the first and only therapy specifically indicated for AFRS, its ninth FDA-approved use.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- June 20, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc and Sanofi today announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of adult patients with bullous pemphigoid (BP).
AI Summary
Regeneron Pharmaceuticals and Sanofi announced that the FDA has approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid (BP). BP is a rare, chronic skin disease that mainly affects older adults, causing painful blisters, intense itch, and skin lesions. In clinical trials, Dupixent showed significant improvements by helping patients achieve sustained disease remission, reducing itch, and minimizing the need for oral corticosteroids compared to a placebo.
This approval marks a major milestone as Dupixent becomes the first targeted therapy for BP. By addressing the underlying type 2 inflammation common in BP, Dupixent provides a new treatment option that may offer better disease management and quality of life for those affected. This breakthrough is important for a condition that has limited treatment choices and predominantly impacts an elderly patient population.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- May 1, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. announced 24 abstracts on Dupixent® (dupilumab) clinical data and real-world analyses in respiratory diseases will be presented at the American Thoracic Society (ATS) International Conference 2025 being held from May 18 to 21 in San Francisco, California.
AI Summary
Regeneron Pharmaceuticals, Inc. announced that 24 abstracts featuring new clinical and real-world analyses of Dupixent® (dupilumab) in respiratory diseases will be presented at the American Thoracic Society International Conference 2025 in San Francisco from May 18 to 21. Among these presentations are one oral presentation and four late-breaking poster sessions that focus on Dupixent’s benefits in patients with chronic obstructive pulmonary disease (COPD) and asthma. The data include findings from landmark Phase 3 trials, highlighting improvements in lung function, reductions in exacerbations, and enhanced health-related quality of life. The abstracts underscore Dupixent’s impact on targeting type 2 inflammation in a diverse patient population, including those with or without emphysema. This initiative, in collaboration with Sanofi, reflects Regeneron’s commitment to advancing the scientific understanding of respiratory diseases and improving patient outcomes.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- April 18, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. and Sanofi announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of adults and adolescents aged 12 years and older with chronic spontaneous urticaria (CSU) who remain symptomatic despite histamine-1 (H1) antihistamine treatment.
AI Summary
Regeneron Pharmaceuticals, Inc. and Sanofi announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for treating chronic spontaneous urticaria (CSU) in adults and adolescents aged 12 and older who remain symptomatic despite treatment with histamine-1 antihistamines. This approval marks Dupixent as the first new targeted therapy for CSU in over a decade and the seventh indication for patients with conditions tied to type 2 inflammation. Based on Phase 3 trial data, Dupixent has been shown to significantly reduce intense itch and hives compared to placebo, offering a new therapeutic option for over 300,000 U.S. patients who previously had limited choices. Administered as a subcutaneous injection, Dupixent provides support for better disease management and improved quality of life for individuals suffering from CSU.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- March 28, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted marketing and manufacturing authorization for Dupixent® (dupilumab) for the treatment of chronic obstructive pulmonary disease (COPD) in adults whose disease is not adequately controlled with existing therapy.
AI Summary
Regeneron Pharmaceuticals, Inc. announced that Japan’s Ministry of Health, Labour and Welfare has granted marketing and manufacturing authorization for Dupixent® (dupilumab) to treat adults with chronic obstructive pulmonary disease (COPD) whose condition is not well controlled with existing therapies. This approval followed a successful Phase 3 BOREAS trial that showed Dupixent significantly reduced flare-ups and improved lung function when added to standard inhaled therapy. The trial focused on patients with elevated blood eosinophils, a key marker linked to COPD exacerbations.
This milestone marks the first new treatment approach for COPD in Japan in more than a decade and extends Dupixent’s use beyond its originally approved indications. The drug is administered via subcutaneous injection every other week and maintains a safety profile consistent with its other approved uses, further solidifying its role in managing conditions driven by type 2 inflammation.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- March 8, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc today presented positive results from the pivotal ADEPT Phase 2/3 trial evaluating the investigational use of Dupixent® (dupilumab) in adults with moderate-to-severe bullous pemphigoid (BP).
AI Summary
Regeneron Pharmaceuticals, Inc. presented promising results from the ADEPT Phase 2/3 trial evaluating Dupixent in adults with moderate-to-severe bullous pemphigoid, a chronic skin disease marked by intense itch and painful blisters. The trial showed that patients treated with Dupixent achieved sustained disease remission at 36 weeks at a rate five times higher than those on placebo. Additionally, significant improvements were noted with 40% of Dupixent-treated patients experiencing a near-complete reduction in disease severity and a meaningful reduction in itch. Dupixent also helped to significantly reduce the use of oral corticosteroids and rescue medications. These positive outcomes highlight the potential of Dupixent as a targeted treatment addressing the underlying type 2 inflammation that drives bullous pemphigoid, offering hope for transforming the management of this challenging condition.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- February 18, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat adults with bullous pemphigoid (BP).
AI Summary
The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat adults with bullous pemphigoid (BP), a chronic, relapsing skin disease. If approved, Dupixent would become the first and only targeted treatment for BP in the United States, with the FDA decision expected by June 20, 2025.
The Priority Review was granted based on positive results from a pivotal study where patients treated with Dupixent achieved sustained disease remission at a rate five times higher than those on placebo. The study also showed significant improvements in reducing disease severity, itch, and reliance on oral corticosteroids. Dupixent’s potential approval would mark a major advance for approximately 27,000 U.S. adults suffering from BP uncontrolled by current treatments.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- November 15, 2024
- Target Action Date:
- April 18, 2025
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that The target action date for the FDA decision is April 18, 2025.
AI Summary
Regeneron Pharmaceuticals announced a key milestone for Dupixent (dupilumab) as the FDA has set the target action date for its decision on the resubmitted supplemental biologics license application at April 18, 2025. This application focuses on the treatment of chronic spontaneous urticaria (CSU) in patients who do not achieve adequate control with H1 antihistamines. New pivotal data from the Phase 3 LIBERTY-CUPID clinical program showed that Dupixent significantly reduced both itch and hives in patients suffering from this condition. With over 300,000 Americans affected by CSU, the potential approval of Dupixent could offer a much-needed targeted therapy option. Regeneron’s positive developments reflect ongoing efforts to advance targeted therapies and improve patient care for those who have limited alternatives for managing CSU.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- November 15, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. and Sanofi announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat adults and pediatric patients aged 12 years and older with chronic spontaneous urticaria (CSU) whose disease is not adequately controlled with H1 antihistamine treatment.
AI Summary
Regeneron Pharmaceuticals and Sanofi announced that the U.S. FDA has accepted for review the resubmitted Supplemental Biologics License Application (sBLA) for Dupixent (dupilumab). This application targets the treatment of chronic spontaneous urticaria (CSU) in adults and pediatric patients aged 12 years and older whose symptoms remain uncontrolled despite H1 antihistamine treatment.
The resubmission, backed by new pivotal data from the LIBERTY-CUPID Phase 3 clinical program, shows that Dupixent significantly reduces both itch and hive activity. This is a crucial development for the more than 300,000 CSU patients in the US who have limited treatment options. The FDA is expected to make a decision by April 18, 2025, and, if approved, Dupixent would be the first targeted therapy for CSU in a decade.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- September 27, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc and Sanofi SA’s Dupixent (dupilumab) as an add-on maintenance treatment of inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype, generally known as “smoker’s lung.” Dupixent is the first biologic medicine approved in the U.S. to treat these patients.
AI Summary
Regeneron Pharmaceuticals and Sanofi have received FDA approval for Dupixent (dupilumab) as an add-on maintenance treatment for adults with inadequately controlled chronic obstructive pulmonary disease (COPD) who have an eosinophilic phenotype, commonly referred to as “smoker’s lung.” This marks Dupixent as the first biologic medicine approved in the U.S. for this specific COPD patient group.
The approval is based on data from two landmark Phase 3 trials. Patients treated with Dupixent experienced notable reductions in moderate to severe COPD exacerbations along with improvements in lung function and overall quality of life compared to those receiving a placebo. Building on its success in treating conditions like asthma and atopic dermatitis, Dupixent now offers new hope for approximately 300,000 COPD patients in the U.S. who have struggled with daily symptoms and unpredictable flare-ups.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- September 11, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc. announced that a Dupixent® (dupilumab) confirmatory Phase 3 trial (LIBERTY-CUPID Study C) met the primary and key secondary endpoints for the investigational treatment of patients with uncontrolled, biologic-naïve chronic spontaneous urticaria (CSU) receiving background therapy with antihistamines.
AI Summary
Regeneron announced that its confirmatory Phase 3 trial, LIBERTY-CUPID Study C, met both the primary and key secondary endpoints for Dupixent in treating uncontrolled chronic spontaneous urticaria (CSU). The study involved 151 biologic-naïve patients, all of whom were still experiencing symptoms despite using background H1 antihistamine therapy. Results showed nearly a 50% reduction in both itch and overall urticaria activity scores compared to placebo. These findings indicate that Dupixent may significantly relieve the debilitating symptoms of CSU, which affects a large number of people who do not respond well to standard treatments. Regeneron plans to share this positive data with the FDA, with the goal of supporting a supplemental biologics application, potentially offering a new therapeutic option for CSU patients.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- August 26, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced 20 abstracts across Dupixent® (dupilumab) and investigational therapy itepekimab will be presented at the European Respiratory Society (ERS) Congress 2024 being held from September 7 to 11 in Vienna, Austria.
AI Summary
Regeneron Pharmaceuticals, Inc. announced that it will present 20 abstracts at the upcoming European Respiratory Society (ERS) Congress 2024 in Vienna, Austria, from September 7 to 11. These abstracts, which include four oral presentations, will showcase new research on Dupixent® (dupilumab) and the investigational therapy itepekimab. The studies focus on treating respiratory conditions such as chronic obstructive pulmonary disease (COPD), asthma, and chronic rhinosinusitis with nasal polyps (CRSwNP), and they highlight advances in reducing exacerbations, improving lung function, and enhancing overall patient quality of life.
In collaboration with Sanofi, the presented research emphasizes the potential of targeting key drivers of type 2 inflammation and other related pathways. With detailed clinical and real-world data, the presentations aim to provide fresh insights into managing difficult-to-treat respiratory diseases and demonstrate Regeneron’s commitment to improving patient outcomes worldwide.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- July 3, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced that the European Commission (EC) has approved Dupixent® (dupilumab) as an add-on maintenance treatment for adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils.
AI Summary
Regeneron Pharmaceuticals, Inc. announced that the European Commission has approved Dupixent® (dupilumab) as an add-on maintenance treatment for adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils. This approval targets patients already taking standard-of-care inhaled therapies, including combinations of an inhaled corticosteroid (ICS), a long-acting beta2-agonist (LABA) and a long-acting muscarinic antagonist (LAMA), or a LABA and LAMA when ICS is not appropriate.
The decision is a groundbreaking step in COPD treatment, offering a new option for about 220,000 EU patients. Data from two Phase 3 trials showed that Dupixent significantly reduces exacerbations, improves lung function, and enhances health-related quality of life. Regeneron’s achievement marks the first global approval of a biologic for COPD driven by type 2 inflammation, setting the stage for further reviews in other regions worldwide.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- May 31, 2024
- Target Action Date:
- September 27, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
Regeneron Pharmaceuticals, Inc announced the U.S. Food and Drug Administration (FDA) has extended by three months the target action date of its priority review of the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD). The revised target action date is September 27, 2024.
AI Summary
Regeneron Pharmaceuticals has announced that the U.S. FDA has extended the target action date for its priority review of the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab). The review now has a revised target action date of September 27, 2024. Dupixent is being examined as an add-on maintenance treatment for adult patients with uncontrolled chronic obstructive pulmonary disease (COPD) who show signs of type 2 inflammation.
This extension comes after the agency received additional efficacy analyses from pivotal clinical trials, which the FDA determined to be a major amendment to the sBLA. Regeneron and its partner Sanofi remain confident that this extra data strongly supports Dupixent’s potential benefits for patients with COPD, reinforcing their commitment to advancing new treatment options for this challenging condition.
Read Announcement- Drug:
- Dupixent (dupilumab)
- Announced Date:
- May 31, 2024
- Indication:
- Moderate-to-severe asthma
Announcement
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Dupixent (dupilumab) in the European Union (EU) as an add-on maintenance treatment in adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils.
AI Summary
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending Dupixent (dupilumab) for approval as an add-on maintenance treatment in the European Union. This treatment is specifically aimed at adults with uncontrolled chronic obstructive pulmonary disease (COPD) who have raised blood eosinophils. The recommendation comes after two Phase 3 trials, which showed that Dupixent significantly reduced moderate to severe COPD exacerbations and improved lung function across a 52-week period.
If approved by the European Commission in the coming months, Dupixent will become the first-ever targeted therapy for COPD in the EU and the first new treatment approach for this disease in more than a decade. This development could offer a valuable option for patients struggling with a challenging respiratory condition that impacts daily life and overall health.
Read Announcement
Olatorepatide FDA Regulatory Events
Olatorepatide is a drug developed by Regeneron Pharmaceuticals for the following indication: treatment of adults with obesity or who are overweight.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- olatorepatide
- Announced Date:
- March 9, 2026
- Indication:
- treatment of adults with obesity or who are overweight
Announcement
Regeneron Pharmaceuticals, Inc announced Hansoh Pharmaceutical Group Company Limited has shared positive topline data from its Phase 3 trial in Chinese patients evaluating olatorepatide for the treatment of adults with obesity or who are overweight.
AI Summary
Regeneron Pharmaceuticals said Hansoh Pharmaceutical reported positive topline results from a Phase 3 trial in Chinese adults with obesity or who are overweight testing olatorepatide. Patients lost up to 19% body weight by week 48. Regeneron plans to begin a global Phase 3 registrational program later this year.
Olatorepatide is a novel dual GLP‑1/GIP receptor agonist that affects appetite and metabolism. The trial showed favorable gastrointestinal tolerability, with lower rates of GI side effects and fewer treatment discontinuations than other published Phase 3 dual‑incretin trials. Average nausea was below 10% and vomiting below 5%. Detailed data will be presented at a medical meeting; safety and efficacy have not yet been reviewed by any regulatory authority.
Under the companies' agreement, Hansoh retains development and commercial rights in Greater China, while Regeneron holds rights outside the Chinese Mainland, Hong Kong and Macau.
Read Announcement
Garetosmab FDA Regulatory Events
Garetosmab is a drug developed by Regeneron Pharmaceuticals for the following indication: Treatment of Fibrodysplasia Ossificans Progressiva (FOP).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- garetosmab
- Announced Date:
- February 19, 2026
- Indication:
- Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for garetosmab for the treatment of adults with fibrodysplasia ossificans progressiva (FOP).
AI Summary
Regeneron Pharmaceuticals announced the U.S. Food and Drug Administration has accepted for Priority Review its Biologics License Application for garetosmab to treat adults with fibrodysplasia ossificans progressiva (FOP). FOP is an ultra‑rare genetic disorder that causes abnormal bone to form in muscles, tendons, ligaments and other connective tissues, leading to severe disability. If approved, garetosmab would be the first and only treatment shown to reduce the number and volume of new heterotopic bone lesions in adults with FOP.
OPTIMA, a Phase 3 multinational trial, assessed garetosmab’s effect on new bone formation and monitored safety. At 56 weeks among 63 adults, serious treatment‑emergent adverse events occurred in 1 patient on 3 mg/kg garetosmab, 2 patients on 10 mg/kg, and 2 on placebo. The most common adverse reactions (≥30%) were nosebleeds, increased hair growth, abscess and acne. Safety and efficacy remain investigational and have not been fully evaluated or approved.
OPTIMA evaluated whole‑body CT scans for heterotopic ossification, flare‑ups, CAJIS joint function scores, and changes in disease severity; safety checks included adverse events, vitals, exams and coagulation tests.
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Aflibercept FDA Regulatory Timeline and Events
Aflibercept is a drug developed by Regeneron Pharmaceuticals for the following indication: For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Aflibercept
- Announced Date:
- February 2, 2026
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc announced upcoming presentations from its ophthalmology portfolio and pipeline at the virtual Angiogenesis (Angiogenesis, Exudation, and Degeneration) annual meeting on February 7, 2026.
AI Summary
Regeneron Pharmaceuticals said it will present data from its ophthalmology portfolio and development pipeline at the virtual Angiogenesis (Angiogenesis, Exudation, and Degeneration) annual meeting on February 7, 2026. The company expects to share clinical and pipeline updates, including results from trials run with Bayer. Regeneron indicated attendees can expect information on efficacy, safety, dosing strategies aimed at reducing injection frequency, and additional clinical findings; a full list of abstracts and presentation times will be provided ahead of the meeting.
Regeneron highlighted EYLEA HD (aflibercept 8 mg), developed to match EYLEA’s efficacy and safety while requiring fewer injections. EYLEA HD is approved in the U.S. for wet AMD, diabetic macular edema (DME), diabetic retinopathy (DR), and macular edema following retinal vein occlusion (RVO). Common adverse reactions (≥3%) include cataract, conjunctival hemorrhage, corneal epithelium defect, increased intraocular pressure, eye pain or irritation, retinal hemorrhage, blurred vision, vitreous detachment, and floaters. EYLEA 2 mg is approved for similar retinal diseases and ROP (0.4 mg). Regeneron co-develops these medicines with Bayer; Regeneron holds U.S. rights while Bayer markets outside the U.S., sharing profits equally.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- November 19, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved EYLEA HD® (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO) with up to every 8-week dosing after an initial monthly dosing period.
AI Summary
Regeneron announced that the U.S. Food and Drug Administration has approved EYLEA HD (aflibercept) Injection 8 mg to treat macular edema following retinal vein occlusion (RVO). The approval allows EYLEA HD to be given every 8 weeks after an initial monthly dosing period (3–5 monthly doses), potentially cutting the number of injections patients receive. The decision was supported by the Phase 3 QUASAR trial, in which patients receiving EYLEA HD every 8 weeks achieved non‑inferior visual acuity gains at 36 weeks compared with patients getting EYLEA 2 mg every 4 weeks. Results were consistent across branch, central, and hemiretinal vein occlusions.
Regeneron also notes a monthly (every 4‑week) dosing option for some patients across approved indications. In RVO trials, common adverse reactions (≥3%) included increased intraocular pressure, blurred vision, cataract, conjunctival hemorrhage, eye discomfort or pain, and vitreous detachment.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- April 28, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced the upcoming presentation of 27 abstracts, including eight oral presentations on EYLEA HD® (aflibercept) Injection 8 mg in wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR).
AI Summary
Regeneron Pharmaceuticals, Inc. announced that they will present 27 abstracts at an upcoming conference, featuring eight oral presentations. These presentations will focus on EYLEA HD® (aflibercept) Injection 8 mg, highlighting new data for its use in treating serious eye conditions. In particular, the studies will examine its efficacy and safety in patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy (DR). The higher dose of EYLEA HD® is being evaluated to determine how it may improve treatment outcomes and provide enhanced benefits for patients suffering from these conditions. By sharing these findings, Regeneron aims to support ongoing research and assist healthcare professionals in making informed decisions when considering treatment options for individuals with these vision-threatening disorders.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- April 18, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the supplemental Biologics License Application (sBLA) for the addition of extended dosing intervals (up to every 24 weeks) for EYLEA HD® (aflibercept) Injection 8 mg across all approved indications.
AI Summary
Regeneron Pharmaceuticals announced on April 18, 2025, that the US FDA issued a complete response letter (CRL) for its supplemental Biologics License Application (sBLA) to extend the dosing interval for EYLEA HD® (aflibercept) Injection 8 mg. The company proposed dosing intervals as long as every 24 weeks across all approved indications, which could reduce the treatment burden for patients. Although the FDA confirmed that there are no concerns with the safety or efficacy of EYLEA HD in its current use, it did not agree to change the label beyond the current maximum of every 16 weeks.
Regeneron is now reviewing the FDA’s feedback to determine its next steps. The CRL underlines the FDA’s strict adherence to established dosing guidelines while the company explores ways to enhance treatment convenience for patients.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- April 17, 2025
- Target Action Date:
- August 19, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced that The FDA target action date is August 19, 2025, following the use of a Priority Review voucher.
AI Summary
Regeneron Pharmaceuticals announced that the FDA has accepted the supplemental Biologics License Application for EYLEA HD under Priority Review. The application seeks approval for the treatment of macular edema following retinal vein occlusion (RVO) and to broaden the dosing schedule, including a monthly option for all approved indications. Importantly, the FDA has set a target action date of August 19, 2025, after utilizing a Priority Review voucher. This review is based on data from the EYLEA HD clinical program, which includes positive results from the Phase 3 QUASAR trial. The trial showed that patients who received the treatment every 8 weeks after an initial monthly dosing achieved similar visual improvements compared to those treated monthly with the existing therapy. This potential new dosing schedule could reduce the treatment burden for patients and provide more flexibility for doctors.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- April 17, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for EYLEA HD® (aflibercept) Injection 8 mg.
AI Summary
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its supplemental Biologics License Application (sBLA) for EYLEA HD® (aflibercept) Injection 8 mg. The application aims to expand treatment options by seeking approval for EYLEA HD to treat macular edema following retinal vein occlusion (RVO) and to broaden its dosing schedule. If approved, patients with RVO could receive injections every eight weeks after an initial period of monthly dosing instead of the standard monthly injections used with other anti-VEGF therapies. This approach could reduce the overall number of injections needed, easing the treatment burden on patients. The submission is supported by data from the Phase 3 QUASAR trial, which demonstrated that the vision improvements with EYLEA HD are comparable to those achieved with standard dosing of EYLEA.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- February 8, 2025
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals, Inc. announced the first presentation of positive results from the Phase 3 QUASAR trial investigating EYLEA HD® (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO), including those with central, branch and hemiretinal vein occlusions.
AI Summary
Regeneron Pharmaceuticals, Inc. announced positive results from its Phase 3 QUASAR trial evaluating EYLEA HD® (aflibercept) Injection 8 mg for macular edema following retinal vein occlusion (RVO). This trial included patients with central, branch, and hemiretinal RVO. The study met its primary endpoint at 36 weeks, demonstrating that patients treated with EYLEA HD—administered every 8 weeks following initial monthly doses—achieved visual acuity gains that were non-inferior to those receiving the standard EYLEA® 2 mg every 4 weeks. The results suggest that the higher dose regimen may offer the potential to reduce the number of required injections, which could improve treatment adherence and patient outcomes. Based on these promising findings, Regeneron plans to support a supplemental Biologics License Application submission to the U.S. FDA in the first quarter of 2025.
Read Announcement- Drug:
- Aflibercept
- Announced Date:
- December 17, 2024
- Indication:
- For the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR),
Announcement
Regeneron Pharmaceuticals announced the primary endpoint was met in the Phase 3 QUASAR trial investigating EYLEA HD® (aflibercept) Injection 8 mg for the treatment of patients with macular edema following retinal vein occlusion (RVO), including those with central, branch and hemiretinal vein occlusions.
AI Summary
Regeneron Pharmaceuticals announced that the primary endpoint was met in the Phase 3 QUASAR trial evaluating EYLEA HD® (aflibercept) Injection 8 mg for macular edema following retinal vein occlusion (RVO), including central, branch, and hemiretinal types. The study demonstrated that, after an initial series of monthly doses, patients on an 8‑week dosing schedule achieved non-inferior visual improvements compared to those receiving the approved 2 mg dose every 4 weeks.
This finding suggests that EYLEA HD could offer comparable vision gains with fewer injections, potentially reducing the treatment burden for RVO patients. The safety profile was consistent with known data for both EYLEA HD and EYLEA. Regeneron plans to present these results at an upcoming medical meeting and to submit a supplemental biologics license application to the FDA in the first quarter of 2025.
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Linvoseltamab FDA Regulatory Events
Linvoseltamab is a drug developed by Regeneron Pharmaceuticals for the following indication: multiple myeloma (NDMM).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- linvoseltamab
- Announced Date:
- December 7, 2025
- Indication:
- multiple myeloma (NDMM)
Announcement
Regeneron Pharmaceuticals, Inc announced encouraging data from the Phase 1/2 LINKER-MM4 trial evaluating Lynozyfic™ (linvoseltamab) in adults with newly diagnosed multiple myeloma (NDMM) who were transplant eligible or ineligible were shared in an oral presentation at the American Society of Hematology (ASH) Annual Meeting.
AI Summary
Regeneron reported encouraging Phase 1/2 LINKER-MM4 results for Lynozyfic (linvoseltamab) in adults with newly diagnosed multiple myeloma (transplant eligible or ineligible) in an ASH oral presentation. LINKER-MM4 is the first trial to test a BCMA×CD3 bispecific antibody as monotherapy in this frontline setting and is part of a broader development program. In 45 treated patients (28 transplant eligible, 17 ineligible) given step-up dosing then 50, 100 or 200 mg, median time to response was 1.2 months. All three dose groups showed VGPR or better (VGPR+) rates ≥70% with limited follow-up, and responses are expected to deepen. Among VGPR+ patients evaluable for minimal residual disease (MRD), 95% (19 of 20) were MRD-negative at 10‑5 sensitivity.
Safety findings showed cytokine release syndrome (all Grade 1) in 44%, neutropenia in 38% (Grade 3/4 in 33%), and infections in 84% (mostly within the first three months). There were no ≥Grade 4 infections, no Grade 5 treatment-emergent adverse events, and no dose-limiting toxicities. Ten patients proceeded to autologous stem cell transplant with acceptable CD34+ yields. A Phase 2 expansion at 200 mg is ongoing.
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TSRA-196 FDA Regulatory Events
TSRA-196 is a drug developed by Regeneron Pharmaceuticals for the following indication: for Alpha-1 Antitrypsin Deficiency (AATD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TSRA-196
- Announced Date:
- December 1, 2025
- Indication:
- for Alpha-1 Antitrypsin Deficiency (AATD)
Announcement
Regeneron Pharmaceuticals, Inc and Tessera Therapeutics, Inc.,announced a global collaboration to develop and commercialize TSRA-196, Tessera's lead investigational in vivo Gene Writing program for the treatment of alpha-1 antitrypsin deficiency (AATD), an inherited monogenic disease that can affect the lungs, liver, or both organs, and currently impacts approximately 200,000 people in the U.S. and Europe.
AI Summary
Regeneron and Tessera announced a global collaboration to develop and commercialize TSRA-196, Tessera’s lead in vivo Gene Writing program for alpha-1 antitrypsin deficiency (AATD), which affects about 200,000 people in the U.S. and Europe. Tessera will receive $150 million from Regeneron (cash plus equity) and may earn up to $125 million in near- and mid-term milestones. The companies will share worldwide development costs and future profits 50:50. Tessera will lead the initial first-in-human trial and Regeneron will lead subsequent global development and commercialization. An Investigational New Drug filing is expected by the end of the year.
TSRA-196 is designed as a one-time, intravenous treatment to precisely correct the SERPINA1 mutation that causes AATD and restore functional AAT protein. Preclinical studies in mice and nonhuman primates showed durable, high-fidelity liver editing, high specificity, no germline or off-target editing, and favorable safety using Tessera’s non-viral lipid nanoparticle delivery. The deal pairs Regeneron’s genetics and clinical development experience with Tessera’s Gene Writing and delivery platforms to accelerate clinical development.
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Libtayo (cemiplimab) FDA Regulatory Timeline and Events
Libtayo (cemiplimab) is a drug developed by Regeneron Pharmaceuticals for the following indication: Advanced Cervical Cancer.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- November 19, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc announced that the European Commission (EC) has approved the PD-1 inhibitor Libtayo® (cemiplimab) as an adjuvant treatment for adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation.
AI Summary
Regeneron announced that the European Commission has approved Libtayo (cemiplimab), a PD‑1 inhibitor, as an adjuvant treatment for adults with cutaneous squamous cell carcinoma (CSCC) who are at high risk of recurrence after surgery and radiation. This approval expands Libtayo’s use in the EU to include patients earlier in their treatment course and could change the care pathway for those with high‑risk disease.
The decision was based on the global Phase 3 C‑POST trial, which showed Libtayo was the first immunotherapy to significantly improve disease‑free survival in the adjuvant setting. Libtayo reduced the risk of recurrence or death by 68% (hazard ratio 0.32; 95% CI 0.20–0.51; p<0.0001) versus placebo in 415 randomized patients. Detailed results were published in the New England Journal of Medicine.
Safety in the trial matched the known profile of Libtayo: grade ≥3 adverse events occurred in 24% of treated patients versus 14% with placebo. Common side effects included fatigue, itching, rash, diarrhea, joint pain and hypothyroidism. Treatment was permanently stopped due to adverse events in 10% of Libtayo patients versus 2% with placebo.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- October 17, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc. announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Libtayo® (cemiplimab) as an adjuvant treatment for adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation.
AI Summary
Regeneron Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion on using Libtayo® (cemiplimab) as an adjuvant treatment for adults with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation. The European Commission is expected to decide on the application in the coming months.
The recommendation is based on results from the global Phase 3 C-POST trial, which compared adjuvant Libtayo to placebo in high-risk CSCC patients. Libtayo reduced the risk of disease recurrence or death by 68% versus placebo (hazard ratio 0.32; 95% CI: 0.20–0.51; p<0.0001). Locoregional recurrence occurred in 4% of Libtayo-treated patients versus 17% with placebo, and distant recurrence was 5% versus 13%, respectively.
Safety findings were consistent with known profiles for Libtayo monotherapy. Adverse events occurred in 91% of patients on Libtayo and 89% on placebo. The most common side effects (≥10%) included fatigue, rash, itching, diarrhea, joint pain and hypothyroidism. Hypertension was the only grade 3-4 event above 2%. Overall, these data support Libtayo’s benefit-risk for preventing CSCC recurrence.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- July 30, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Sensei Biotherapeutics, Inc that clinical data from the dose expansion cohort of the Phase 1/2 trial of solnerstotug alone and in combination with Libtayo® (cemiplimab), Regeneron's PD-1 inhibitor, will be presented in a mini oral session at the European Society for Medical Oncology (ESMO) Congress 2025, being held October 17-21, 2025 in Berlin, Germany..
AI Summary
Sensei Biotherapeutics announced that clinical data from the dose expansion cohort of the Phase 1/2 trial testing solnerstotug alone and in combination with Libtayo® (cemiplimab), Regeneron’s PD-1 inhibitor, will be presented in a mini oral session at the European Society for Medical Oncology (ESMO) Congress 2025 in Berlin. This study focuses on patients with advanced solid tumors resistant to prior PD-(L)1 therapy.
The mini oral session, titled “Results from a Phase 1 expansion cohort of solnerstotug combined with cemiplimab,” is scheduled for Friday, October 17, from 2:00 to 3:30 CEST. Dr. Kyriakos Papadopoulos of START in San Antonio will present abstract 3933. Solnerstotug is designed to block VISTA in the tumor microenvironment, aiming to unleash T cells against tumors. The presentation will highlight safety and preliminary efficacy in this hard-to-treat group.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- May 31, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc. announced detailed analyses from the Phase 3 C-POST trial, which evaluated PD-1 inhibitor Libtayo® (cemiplimab) in patients with high-risk cutaneous squamous cell carcinoma (CSCC) after surgery.
AI Summary
Regeneron Pharmaceuticals announced new detailed analyses from the Phase 3 C-POST clinical trial, which evaluated the PD-1 inhibitor Libtayo (cemiplimab) in patients with high-risk cutaneous squamous cell carcinoma (CSCC) after surgery. The trial showed that Libtayo reduced the risk of disease recurrence or death by 68% compared to placebo, marking a significant improvement in disease-free survival. Additionally, Libtayo reduced the risk of locoregional recurrence by 80% and distant recurrence by 65% when compared with placebo.
These promising results were presented at the 2025 American Society of Clinical Oncology Annual Meeting and published in the New England Journal of Medicine. The findings suggest that cemiplimab could become an important systemic therapy to prevent relapse in high-risk CSCC patients following surgery. Regulatory applications for this new potential indication have been submitted in both the United States and the European Union.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- May 1, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc. announced new and updated data from its oncology and hematology portfolio will be shared at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place from May 30 to June 3 in Chicago, IL.
AI Summary
Regeneron Pharmaceuticals announced that it will share new and updated data from its oncology and hematology portfolio at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, which will be held from May 30 to June 3 in Chicago, IL. The company plans to showcase 18 presentations covering findings from both approved and investigational treatment regimens.
Key highlights include updates from trials involving the PD-1 inhibitor Libtayo and early data from studies on the investigational BCMAxCD3 bispecific antibody linvoseltamab. These studies focus on difficult-to-treat cancers such as various skin cancers, lung cancer, lymphoma, and multiple myeloma, aiming to improve treatment outcomes. Regeneron’s research efforts promise to offer fresh insights and potential new therapeutic options for patients battling these challenging diseases.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- January 13, 2025
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc announced positive results from the Phase 3 C-POST trial, which demonstrated that adjuvant treatment with PD-1 inhibitor Libtayo® (cemiplimab) led to a statistically significant and clinically meaningful improvement in the primary endpoint of disease-free survival (DFS) in patients with high-risk cutaneous squamous cell carcinoma (CSCC) after surgery.
AI Summary
Regeneron Pharmaceuticals announced positive Phase 3 C-POST trial results for Libtayo® (cemiplimab) used as an adjuvant treatment in patients with high-risk cutaneous squamous cell carcinoma (CSCC) after surgery. The study’s first prespecified interim analysis, with a median follow-up of 24 months, showed that Libtayo reduced the risk of cancer recurrence or death by 68% compared to placebo, marking a statistically significant and clinically meaningful improvement in disease-free survival (DFS).
This achievement positions Libtayo as the first immunotherapy to show such benefits in the adjuvant setting for high-risk CSCC, highlighting its potential role in delaying cancer recurrence in patients who have limited treatment options. The trial enrolled 415 patients, and further follow-up is planned, including an analysis of overall survival, to solidify these promising results.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- September 9, 2024
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals, Inc announced five-year results from the final pre-specified overall survival (OS) analysis of the Phase 3 EMPOWER-Lung 1 trial, which evaluated Libtayo® (cemiplimab) monotherapy versus chemotherapy as a first-line treatment for adults with advanced non-small cell lung cancer (NSCLC) with ≥50% PD-L1 expression and no EGFR, ALK or ROS1 aberrations.
AI Summary
Regeneron Pharmaceuticals recently announced five-year results from the final overall survival (OS) analysis of the Phase 3 EMPOWER-Lung 1 trial. The study compared Libtayo® (cemiplimab) as a monotherapy with chemotherapy for adults with advanced non-small cell lung cancer (NSCLC) that has high PD-L1 levels (≥50%) and without EGFR, ALK, or ROS1 mutations. Libtayo nearly doubled the median OS compared to chemotherapy and reduced the risks of death and disease progression by 41% and 50%, respectively.
The results underscore Libtayo’s durable survival benefit and its effectiveness as a first-line treatment in patients with high PD-L1 expression. Moreover, the trial provided data on patients who added chemotherapy after their disease progressed on Libtayo, showing further improvements in survival and response rates. These findings offer important insights for doctors in developing treatment strategies for advanced NSCLC patients.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- June 3, 2024
- Indication:
- Advanced Cervical Cancer
Announcement
FibroGen, Inc announced a clinical trial supply agreement with Regeneron Pharmaceuticals to evaluate FibroGen's immuno-oncology assets, FG-3165 and FG-3175, in combination with Regeneron's anti-PD-1 therapy, LIBTAYO® (cemiplimab), in patients with solid tumors.
AI Summary
FibroGen, Inc. recently announced a clinical trial supply agreement with Regeneron Pharmaceuticals. The deal will evaluate FibroGen’s immuno-oncology assets, FG-3165 and FG-3175, when combined with Regeneron’s anti-PD-1 therapy, LIBTAYO® (cemiplimab), in patients with solid tumors. In preclinical studies, both FG-3165, an antibody targeting galectin-9, and FG-3175, an antibody targeting CCR8, showed complementary effects when paired with PD-1 inhibitors. The collaboration aims to explore whether combining these drugs might enhance the overall treatment benefits. Under the agreement, Regeneron will supply the drugs while FibroGen will sponsor the Phase 1 clinical trials testing these combination therapies. Both companies maintain full rights to their individual compounds. This partnership highlights a promising new approach in cancer treatment by potentially improving outcomes for patients with select solid tumors.
Read Announcement- Drug:
- Libtayo (cemiplimab)
- Announced Date:
- May 23, 2024
- Indication:
- Advanced Cervical Cancer
Announcement
Regeneron Pharmaceuticals announced positive new results from an ongoing Phase 1/2 trial evaluating its first-in-class costimulatory bispecific antibody, REGN7075 (EGFRxCD28), in combination with Libtayo® (cemiplimab) in patients with advanced solid tumors.
AI Summary
Regeneron Pharmaceuticals recently announced promising early results from its ongoing Phase 1/2 trial evaluating REGN7075 (EGFRxCD28), a first-in-class costimulatory bispecific antibody, in combination with Libtayo® (cemiplimab) for patients with advanced solid tumors. In the trial’s dose-escalation phase, the combination showed anti-tumor activity in patients with microsatellite stable colorectal cancer (MSS CRC), a type that typically does not respond to immunotherapy. Among the MSS CRC patients treated, some achieved a complete or partial response, while others experienced stable disease. The safety profile was acceptable with manageable infusion-related reactions and no dose-limiting toxicities observed. These early findings are significant as they represent one of the first indications of potential immunotherapy activity in MSS CRC, supporting further evaluation of this novel treatment strategy across additional tumor types.
Read Announcement
Libtayo (Cemiplimab-Rwlc) FDA Regulatory Events
Libtayo (Cemiplimab-Rwlc) is a drug developed by Regeneron Pharmaceuticals for the following indication: Advanced Non-Small Cell Lung Cancer (NSCLC) with PD-L1.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Libtayo (Cemiplimab-Rwlc)
- Announced Date:
- October 8, 2025
- Indication:
- Advanced Non-Small Cell Lung Cancer (NSCLC) with PD-L1
Announcement
Regeneron Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has approved the PD-1 inhibitor Libtayo® (cemiplimab-rwlc) as an adjuvant treatment for adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation.
AI Summary
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration has approved Libtayo® (cemiplimab-rwlc) as an adjuvant treatment for adult patients with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation. Libtayo, already the standard of care in advanced CSCC, is now the first immunotherapy approved to help prevent disease return in this earlier setting. The FDA granted Priority Review, underscoring its potential to improve outcomes for patients facing a serious risk of relapse.
The approval was based on data from the Phase 3 C-POST trial. In that study, adjuvant Libtayo cut the risk of disease recurrence or death by 68% compared with placebo (hazard ratio 0.32; 95% confidence interval 0.20–0.51; p<0.0001). These results demonstrate a significant gain in disease-free survival and suggest Libtayo could change the treatment paradigm for high-risk CSCC.
The safety profile in the adjuvant setting was consistent with known effects in advanced cancers. Common reactions included rash, itching, and hypothyroidism, while serious adverse events occurred in 18% of patients. This approval offers a much-needed, practice-changing option for adults at high risk of CSCC recurrence.
Read Announcement
Evkeeza FDA Regulatory Events
Evkeeza is a drug developed by Regeneron Pharmaceuticals for the following indication: treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Evkeeza
- Announced Date:
- September 26, 2025
- Indication:
- treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH).
Announcement
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Evkeeza® (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH).
AI Summary
Regeneron announced that the U.S. Food and Drug Administration has approved Evkeeza® (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies to treat children aged 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH).
HoFH is a rare inherited condition in which two faulty genes cause extremely high levels of “bad” LDL cholesterol—often above 400 mg/dL—putting patients at risk for early artery disease and heart attacks. Evkeeza works by blocking the ANGPTL3 protein, helping to lower LDL cholesterol by about 50% when added to standard treatments.
This extension was supported by clinical data from six very young children treated under expanded access or compassionate use programs, with no new safety concerns identified. The most common side effects (≥ 5%) include nasopharyngitis, flu-like illness, dizziness, runny nose, nausea and fatigue.
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Garetosma FDA Regulatory Events
Garetosma is a drug developed by Regeneron Pharmaceuticals for the following indication: Fibrodysplasia Ossificans Progressiva (FOP).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- garetosma
- Announced Date:
- September 17, 2025
- Indication:
- Fibrodysplasia Ossificans Progressiva (FOP)
Announcement
Regeneron Pharmaceuticals, Inc announced the primary endpoint was met in the Phase 3 OPTIMA trial investigating garetosmab in adults with fibrodysplasia ossificans progressiva (FOP).
AI Summary
Fibrodysplasia ossificans progressiva (FOP) is a rare disease where muscles, ligaments, and tendons turn into bone, causing severe loss of movement. Garetosmab is the first treatment to show a strong drop in both the number and size of new bone lesions in adults with FOP.
Regeneron announced that in its Phase 3 OPTIMA trial, garetosmab met its main goal at 56 weeks. Patients who received 3 mg/kg saw a 94% cut in new lesions, and those on 10 mg/kg saw a 90% cut, compared to placebo.
Post-hoc analysis also showed a more than 99% drop in the total volume of new bone lesions. Garetosmab targets the Activin A protein to stop abnormal bone growth.
Based on these results and safety data, an independent committee advised switching all placebo patients to garetosmab. Regeneron plans to submit for U.S. approval by year-end 2025.
Read Announcement
Semaglutide (GLP-1 receptor agonist) FDA Regulatory Timeline and Events
Semaglutide (GLP-1 receptor agonist) is a drug developed by Regeneron Pharmaceuticals for the following indication: induced Weight Loss by Preserving Lean Mass.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Semaglutide (GLP-1 receptor agonist)
- Announced Date:
- September 17, 2025
- Indication:
- induced Weight Loss by Preserving Lean Mass
Announcement
Regeneron Pharmaceuticals, Inc announced updated analyses from the ongoing Phase 2 COURAGE trial investigating novel combinations of semaglutide (GLP-1 receptor agonist) and trevogrumab (anti-GDF8/anti-myostatin) with or without garetosmab (anti-activin A) for the treatment of obesity.
AI Summary
Regeneron Pharmaceuticals announced updated results from its Phase 2 COURAGE trial testing novel combinations of semaglutide, a GLP-1 agonist, with trevogrumab (anti-GDF8/myostatin) and, in some groups, garetosmab (anti-activin A) in adults with obesity. At 26 weeks, adding trevogrumab with or without garetosmab significantly reduced the loss of lean muscle mass normally seen with semaglutide alone.
Analysis showed semaglutide by itself led to a 6.5% drop in lean mass (about 33% of total weight lost). All combination groups cut that loss by about half, while also boosting fat loss and overall weight reduction, with the highest-dose triplet achieving a 13.4% body weight decrease.
Across all groups, patients had numeric improvements in waist circumference, blood pressure, cholesterol, triglycerides and A1C. The combinations were generally well tolerated, supporting further study of trevogrumab’s role in preserving muscle during weight loss.
Read Announcement- Drug:
- Semaglutide (GLP-1 receptor agonist)
- Announced Date:
- July 28, 2025
- Indication:
- induced Weight Loss by Preserving Lean Mass
Announcement
Lexaria Bioscience Corp. provides the following partial 8-week positive interim results update on the phase 1b, 12-week chronic study GLP-1-H24-4 (the "Study" or the "Lexaria Study"), currently underway in Australia, focusing on the DehydraTECH ("DHT") glucagon-like peptide-1 ("GLP-1") study arms 2 and 5 relative to the Rybelsus® control study arm 4.
AI Summary
Lexaria Bioscience reported positive interim results after 8 weeks in its phase 1b, 12-week GLP-1-H24-4 study in Australia. Focusing on DehydraTECH (DHT) study arms 2 (DHT-semaglutide; n=24) and 5 (DHT-tirzepatide; n=25) versus the Rybelsus® control arm 4 (n=25), arm 2 saw a 20.8% reduction in participants experiencing any adverse event (79.2% vs 100%) and a 36.5% decrease in total adverse events. Gastrointestinal side effects fell by 43.5% with DHT-semaglutide. Arm 5 reported 72% of participants with adverse events and total gastrointestinal events at 43.5% of the control.
These interim data align with the study’s primary safety and tolerability endpoint, demonstrating DehydraTECH’s potential to reduce unwanted side effects compared to the only approved oral GLP-1 therapy. The trial remains on schedule, with full 12-week efficacy and lab results expected by the end of 2025.
Read Announcement- Drug:
- Semaglutide (GLP-1 receptor agonist)
- Announced Date:
- June 2, 2025
- Indication:
- induced Weight Loss by Preserving Lean Mass
Announcement
Regeneron Pharmaceuticals, Inc. announced interim results from the ongoing Phase 2 COURAGE trial investigating novel combinations of semaglutide (GLP-1 receptor agonist) and trevogrumab (anti-GDF8/anti-myostatin) with or without garetosmab (anti-activin A) for the treatment of obesity.
AI Summary
Regeneron Pharmaceuticals recently announced interim results from its ongoing Phase 2 COURAGE trial focused on treating obesity. The study is testing innovative combinations of semaglutide—a GLP-1 receptor agonist—with trevogrumab, an antibody that blocks GDF8/anti-myostatin, with or without garetosmab, an anti-activin A agent. The trial found that about 35% of the weight loss from semaglutide alone is due to loss of lean mass. However, when semaglutide was combined with trevogrumab (with or without garetosmab), patients preserved much of their lean muscle while achieving greater fat loss.
These results indicate that combining semaglutide with muscle-preserving antibodies can improve the quality of weight loss by sparing crucial muscle mass and increasing fat loss. The full data set is expected later this year to help optimize dosing regimens in future trials.
Read Announcement
REGN1908 FDA Regulatory Events
REGN1908 is a drug developed by Regeneron Pharmaceuticals for the following indication: Antibody-Blockers of Cat and Birch Allergies.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- REGN1908
- Announced Date:
- September 8, 2025
- Indication:
- Antibody-Blockers of Cat and Birch Allergies
Announcement
Regeneron Pharmaceuticals, Inc announced results evaluating its first-in-class investigational allergen-blocking antibodies in allergen-challenge Phase 3 trials in adults with moderate-to-severe cat or birch allergies. Both trials met their respective primary and key secondary endpoints.
AI Summary
Regeneron reported positive results from two Phase 3 trials of new allergen-blocking antibodies in adults with moderate-to-severe cat or birch allergies. A single dose was followed by an allergen challenge. Both trials met their primary goal of reducing eye itch, redness and skin test reactivity.
In the cat trial, the antibodies targeting the Fel d 1 protein cut itch by 52%, redness by 39% and skin reactivity by 44%, all with high statistical significance. The treatment was well tolerated with no serious related adverse events. A confirmatory trial is planned for early 2026.
In the birch trial, antibodies against the Bet v 1 protein reduced itch by 51%, redness by 46% and skin test reactivity by 44% with no serious side effects. These results will be presented at an upcoming medical conference and will guide further trials starting by year-end.
Read Announcement
Cemdisiran FDA Regulatory Events
Cemdisiran is a drug developed by Regeneron Pharmaceuticals for the following indication: Immunoglobulin A Nephropathy (IgAN).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Cemdisiran
- Announced Date:
- August 26, 2025
- Indication:
- Immunoglobulin A Nephropathy (IgAN)
Announcement
Regeneron Pharmaceuticals, announced that the primary and key secondary endpoints were met in the Phase 3 NIMBLE trial assessing investigational cemdisiran monotherapy in adults with generalized myasthenia gravis (gMG).
AI Summary
Regeneron announced that cemdisiran monotherapy achieved the Phase 3 NIMBLE trial’s primary endpoint in adults with generalized myasthenia gravis (gMG). Patients given 600 mg subcutaneously every 12 weeks saw a 2.3-point placebo-adjusted improvement on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score at Week 24. The key secondary endpoint, change in Quantitative Myasthenia Gravis (QMG) score, also showed significant benefit. Cemdisiran, an siRNA targeting complement factor 5 (C5), achieved 74% average complement inhibition.
These data show that partial C5 blockade can deliver strong symptom relief in gMG. The treatment was generally well tolerated, with no discontinuations due to adverse events. Regeneron plans a U.S. regulatory submission in the first quarter of 2026, pending FDA discussions. These results highlight cemdisiran’s potential as a convenient quarterly treatment for gMG.
Read Announcement
Linvoseltamab-gcpt FDA Regulatory Events
Linvoseltamab-gcpt is a drug developed by Regeneron Pharmaceuticals for the following indication: For Treatment of Relapsed or Refractory Multiple Myeloma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- linvoseltamab-gcpt
- Announced Date:
- July 2, 2025
- Indication:
- For Treatment of Relapsed or Refractory Multiple Myeloma
Announcement
Regeneron Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Lynozyfic™ (linvoseltamab-gcpt) to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti‑CD38 monoclonal antibody.
AI Summary
The U.S. FDA has granted accelerated approval to Lynozyfic™ (linvoseltamab‐gcpt) for treating adult patients with relapsed or refractory multiple myeloma. This approval is for patients who have already received at least four prior treatments, including a proteasome inhibitor, an immunomodulatory agent, and an anti‑CD38 monoclonal antibody. The decision was based on promising response rates and the lasting effects seen in the LINKER-MM1 trial.
Lynozyfic is notable for being the first FDA-approved BCMAxCD3 bispecific antibody that can transition to a dosing schedule of every two weeks at week 14, and potentially every four weeks if a very good partial response is maintained after 24 weeks. Patients start on a step-up dosing regimen while under hospital supervision to ensure their safety. Continued approval will depend on further clinical evidence confirming its benefits.
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Itepekimab FDA Regulatory Events
Itepekimab is a drug developed by Regeneron Pharmaceuticals for the following indication: For Chronic Obstructive Pulmonary Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- itepekimab
- Announced Date:
- May 30, 2025
- Indication:
- For Chronic Obstructive Pulmonary Disease
Announcement
Regeneron Pharmaceuticals, Inc announced that a Phase 3 trial, AERIFY-1, evaluating the investigational use of itepekimab in adults who were former smokers with inadequately controlled chronic obstructive pulmonary disease (COPD) met the primary endpoint of significantly reducing moderate or severe acute exacerbations by 27% compared to placebo at week 52, a clinically meaningful benefit.
AI Summary
Regeneron Pharmaceuticals announced that its Phase 3 AERIFY-1 trial met the primary endpoint for itepekimab in adults who were former smokers with inadequately controlled COPD. The trial demonstrated a significant 27% reduction in moderate or severe acute exacerbations compared to placebo at 52 weeks, offering a clinically meaningful benefit for patients regardless of their eosinophilic status. This positive outcome highlights the potential of itepekimab as a new treatment option for individuals with COPD who continue to experience frequent exacerbations despite maximal therapy.
Although a second Phase 3 trial, AERIFY-2, did not meet its primary endpoint, the safety profile of itepekimab remained consistent and well tolerated across both studies. Regeneron and its partner Sanofi are currently reviewing the data and are expected to discuss next steps with regulatory authorities.
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Linvoseltamab (BCMAxCD3) FDA Regulatory Timeline and Events
Linvoseltamab (BCMAxCD3) is a drug developed by Regeneron Pharmaceuticals for the following indication: Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- May 22, 2025
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals, Inc. announced initial results from two cohorts of the Phase 1b LINKER-MM2 trial evaluating linvoseltamab in combination with two different proteasome inhibitors (PI) – carfilzomib or bortezomib – in patients with relapsed/refractory (R/R) multiple myeloma (MM).
AI Summary
Regeneron Pharmaceuticals announced early results from two cohorts of the Phase 1b LINKER‑MM2 trial in patients with relapsed or refractory multiple myeloma. The study evaluated the bispecific antibody linvoseltamab in combination with two proteasome inhibitors, carfilzomib and bortezomib. Patients enrolled had progressed after at least two lines of therapy and many had been previously treated with proteasome inhibitors. In the carfilzomib cohort, linvoseltamab demonstrated compelling clinical activity with a 90% overall response rate, including high complete response rates, even in high‑risk patients. The second cohort combining linvoseltamab with bortezomib also showed promising efficacy with an 85% overall response rate. These results suggest that pairing linvoseltamab with either PI could be an effective strategy for difficult‑to‑treat multiple myeloma cases. Further clinical data will be presented at the upcoming ASCO Annual Meeting.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- February 28, 2025
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals, Inc announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization of linvoseltamab to treat adults with relapsed and refractory (R/R) multiple myeloma (MM).
AI Summary
Regeneron Pharmaceuticals has announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for the conditional marketing authorization of linvoseltamab. This new bispecific antibody is aimed at treating adults with relapsed and refractory multiple myeloma who have already received at least three prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti‐CD38 monoclonal antibody. Patients must have experienced disease progression on the last therapy to be eligible. The CHMP’s decision is based on promising results from the pivotal LINKER-MM1 trial, which assessed both the safety and anti-tumor activity of the treatment. The European Commission is expected to make a final decision in the coming months, potentially offering a new treatment option for patients who have exhausted conventional therapies.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- February 11, 2025
- Target Action Date:
- July 10, 2025
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals announced that FDA decision expected by July 10, 2025
AI Summary
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for review its resubmitted Biologics License Application for linvoseltamab. This investigational treatment, intended for adult patients with relapsed or refractory multiple myeloma who have undergone several prior therapies, is now progressing through the FDA’s review process. The company expects a decision from the FDA by July 10, 2025. Regeneron is moving forward with this submission based on strong clinical trial data, which supports the potential benefits of linvoseltamab for patients in need of new treatment options. This important milestone brings hope for patients facing limited alternatives and marks a significant step in the development of a therapy that could improve outcomes for those with advanced multiple myeloma.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- February 11, 2025
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for linvoseltamab for the treatment of adult patients with relapsed/refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy or those who received three prior lines of therapy and are refractory to the last line of therapy.
AI Summary
Regeneron Pharmaceuticals announced that the FDA has accepted for review the resubmission of its Biologics License Application (BLA) for linvoseltamab. The application targets the treatment of adults with relapsed or refractory multiple myeloma who have already undergone at least four lines of therapy, or three lines where the last treatment was ineffective. The resubmission comes after resolving third-party fill/finish manufacturing challenges, which had previously been the only issue affecting approvability. The FDA’s review is based on data from the pivotal LINKER-MM1 trial and carries a target decision date of July 10, 2025. Linvoseltamab is an investigational bispecific antibody designed to bridge B-cell maturation antigen on myeloma cells with CD3 on T cells, aiming to activate the immune system to attack cancer cells in this hard-to-treat patient population.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- August 20, 2024
- Target Action Date:
- Q2 2024
- Estimated Target Date Range:
- April 1, 2024 - June 30, 2024
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for linvoseltamab in relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies. This anticipated outcome was previously disclosed during Regeneron's second quarter 2024 earnings call.
AI Summary
Regeneron Pharmaceuticals announced that the FDA has issued a Complete Response Letter (CRL) for its Biologics License Application for linvoseltamab. The drug is being developed to treat relapsed or refractory multiple myeloma in patients who have already received at least three prior therapies. The CRL arose due to concerns found during a pre-approval inspection at a third-party fill/finish facility, which was involved with another company’s product candidate. However, the manufacturer has reported that the issues have been resolved and that a reinspection by the FDA is expected in the coming months. Regeneron had previously indicated during its second quarter 2024 earnings call that this outcome was anticipated. The company is working closely with the manufacturer and the FDA to address the matter and hopes to bring linvoseltamab to patients as soon as possible.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- August 20, 2024
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for linvoseltamab in relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies.
AI Summary
Regeneron Pharmaceuticals announced that the U.S. FDA has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for linvoseltamab. The medication is being developed for relapsed/refractory multiple myeloma that has worsened after a patient has had at least three treatments. The FDA’s letter is based on issues found during a pre-approval inspection at a third-party fill/finish manufacturer involved with another company’s product. This is the only approvability concern mentioned by the agency.
The third-party manufacturer has informed Regeneron that it believes the issues are resolved and that the facility will soon undergo a reinspection by the FDA. Regeneron said it will continue to work closely with both the manufacturer and the FDA to help ensure that linvoseltamab reaches patients in need as quickly as possible.
Read Announcement- Drug:
- Linvoseltamab (BCMAxCD3)
- Announced Date:
- June 16, 2024
- Indication:
- Designed to bridge B-cell maturation antigen (BCMA) on multiple myeloma cells with CD3-expressing T cells to facilitate T-cell activation and cancer-cell killing.
Announcement
Regeneron Pharmaceuticals, Inc. announced that 14-month median follow-up data from the pivotal Phase 1/2 LINKER-MM1 trial of linvoseltamab in patients with relapsed/refractory (R/R) multiple myeloma (MM) were shared during an oral presentation at the European Hematology Association (EHA) Congress 2024 and published in the Journal of Clinical Oncology.
AI Summary
Regeneron Pharmaceuticals announced key results from the Phase 1/2 LINKER-MM1 trial of linvoseltamab in patients with relapsed or refractory multiple myeloma. With a 14-month median follow-up, the data—presented at the European Hematology Association Congress 2024 and published in the Journal of Clinical Oncology—show that 50% of patients achieved a complete response or better, while 71% had an overall response. These longer-term results suggest that the treatment continues to produce deeper responses over time. Linvoseltamab is a bispecific antibody that connects cancer cells with T cells, triggering the immune system to fight the cancer. The encouraging efficacy data support the potential of linvoseltamab as a promising future option for patients whose cancer has not responded to standard therapies, fueling further research and regulatory review in this challenging area of medicine.
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NTLA-2001 FDA Regulatory Timeline and Events
NTLA-2001 is a drug developed by Regeneron Pharmaceuticals for the following indication: Transthyretin (ATTR) Amyloidosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- NTLA-2001
- Announced Date:
- March 26, 2025
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
AI Summary
Intellia Therapeutics announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its CRISPR-based treatment, nexiguran ziclumeran (nex-z, also known as NTLA-2001). This important designation is for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a serious condition that affects heart function. The RMAT status is part of an effort under the 21st Century Cures Act to speed up the development and review of promising therapies aimed at life-threatening diseases. It offers benefits such as early interactions with the FDA, discussions on surrogate endpoints, and potentially faster review times. Intellia’s CEO, John Leonard, M.D., said this designation represents a significant step toward accelerating the treatment’s development so that patients might receive this transformative therapy sooner.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- November 25, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN).
AI Summary
Intellia Therapeutics, Inc. announced that the U.S. FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational treatment nexiguran ziclumeran (nex-z, also known as NTLA-2001) for hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is a one-time, in vivo CRISPR-based therapy designed to inactivate the TTR gene, which stops the body from producing the TTR protein that forms harmful amyloid deposits. Early interim Phase 1 data showed that nex-z achieved rapid, deep, and lasting TTR reduction, suggesting the treatment could halt and possibly reverse disease progression. With RMAT designation, Intellia will benefit from early FDA interactions that may help speed up development and review. This status is expected to promote a faster path toward potential regulatory approval, providing hope for patients with this serious and life-threatening condition.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- November 16, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for ATTR amyloidosis. .
AI Summary
Intellia Therapeutics announced positive Phase 1 clinical data for nexiguran ziclumeran (nex-z, also known as NTLA-2001), an investigational in vivo CRISPR-based gene editing therapy. Nex-z is being developed as a one-time treatment for patients with transthyretin (ATTR) amyloidosis. The clinical trial data show that a single infusion of nex-z can produce rapid, deep, and durable reduction in serum TTR levels. This significant TTR reduction is linked with signs of disease stabilization and improvement, particularly in patients with ATTR cardiomyopathy and hereditary ATTR amyloidosis with polyneuropathy. The observed efficacy suggests that greater TTR reduction may lead to improved clinical benefit by potentially slowing disease progression. Moreover, the data support the potential of nex-z as a breakthrough approach in treating ATTR amyloidosis with a favorable safety and tolerability profile.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- October 1, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc announced that interim data from the ongoing Phase 1 study of nexiguran ziclumeran (nex-z, also known as NTLA-2001) will be presented in a late-breaking oral presentation at the 2024 American Heart Association (AHA) Scientific Sessions, taking place November 16 – 18 in Chicago, Illinois.
AI Summary
Intellia Therapeutics, Inc. announced that interim data from its ongoing Phase 1 study of nexiguran ziclumeran (nex-z or NTLA-2001) will be shared during a late-breaking oral presentation at the 2024 American Heart Association Scientific Sessions in Chicago, Illinois from November 16 to 18. The study focuses on using this investigational in vivo CRISPR gene editing therapy as a potential one-time treatment for transthyretin (ATTR) amyloidosis, a disease that can cause heart complications. During the session led by Dr. Marianna Fontana, the presentation will reveal important information on safety, reductions in serum TTR levels, and biomarkers of disease progression. This data may provide key insights into how nex-z affects functional capacity in patients with ATTR amyloidosis with cardiomyopathy. The upcoming presentation is part of Intellia’s continued efforts to develop innovative therapies using CRISPR technology for life-changing treatments.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- June 25, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, presented new data demonstrating for the first time the potential for redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy.
AI Summary
Intellia Therapeutics has revealed promising new data showing for the first time that redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy is possible. In their ongoing Phase 1 study of NTLA-2001, an experimental treatment for transthyretin (ATTR) amyloidosis, patients who initially received a lower dose were later given a follow-on 55 mg dose. This second dose resulted in a 90% median reduction in the serum TTR protein by day 28, and a cumulative 95% reduction from baseline, demonstrating an additive effect when the redose was administered. The 55 mg dose was generally well tolerated, with only one patient experiencing a mild infusion-related reaction. Although redosing is not planned for the current ATTR amyloidosis program, these findings highlight the potential of Intellia’s non-viral, lipid nanoparticle (LNP)-based delivery platform for future therapies where multiple doses could be beneficial.
Read Announcement- Drug:
- NTLA-2001
- Announced Date:
- June 17, 2024
- Indication:
- Transthyretin (ATTR) Amyloidosis
Announcement
Intellia Therapeutics, Inc. announced the acceptance of an abstract featuring redosing data from the Phase 1 study of NTLA-2001 has been selected for an oral presentation at the Peripheral Nerve Society Annual Meeting, taking place June 22 – 25 in Montreal, Canada. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis.
AI Summary
Intellia Therapeutics, Inc. announced that an abstract featuring redosing data from its Phase 1 study of NTLA-2001 has been accepted for oral presentation at the Peripheral Nerve Society Annual Meeting in Montreal, Canada, from June 22–25, 2024. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed as a one-time treatment for transthyretin (ATTR) amyloidosis. In the Phase 1 study, the first three patients were given a low initial dose of 0.1 mg/kg, followed by a higher follow-on dose of 55 mg. These findings provide the first clinical data on repeat dosing with an in vivo CRISPR candidate, shedding light on its safety profile and pharmacodynamic effects. While NTLA-2001 is intended as a single-dose treatment, the option to redose could be an important benefit for future therapies using Intellia’s non-viral lipid nanoparticle delivery platform.
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Odronextamab FDA Regulatory Timeline and Events
Odronextamab is a drug developed by Regeneron Pharmaceuticals for the following indication: To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Odronextamab
- Announced Date:
- February 26, 2025
- Target Action Date:
- July 30, 2025
- Indication:
- To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
Announcement
Regeneron Pharmaceuticals, Inc. announced that The target action date for the FDA decision is July 30, 2025.
AI Summary
Regeneron Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has accepted the resubmission of its Biologics License Application for odronextamab, a treatment aimed at patients with relapsed/refractory follicular lymphoma. Along with supporting Phase 1 and Phase 2 trial data, the company met an enrollment target for its Phase 3 confirmatory trial, OLYMPIA-1. This submission marks the current step in seeking approval to offer odronextamab as a new treatment option.
The FDA plans to reach its decision on the application by July 30, 2025. This target action date is a critical milestone in Regeneron’s development of odronextamab, as it could provide an important new therapy for patients who have exhausted other treatment options in their fight against advanced follicular lymphoma.
Read Announcement- Drug:
- Odronextamab
- Announced Date:
- February 26, 2025
- Indication:
- To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
Announcement
Regeneron Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for odronextamab in relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. .
AI Summary
Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for review its resubmitted Biologics License Application (BLA) for odronextamab. The application is aimed at treating patients with relapsed/refractory follicular lymphoma who have undergone two or more lines of systemic therapy. The FDA’s decision process is set to target a conclusion by July 30, 2025.
This resubmission comes after Regeneron successfully met a key enrollment target in the Phase 3 confirmatory trial for odronextamab. The application is supported by data from earlier Phase 1 and Phase 2 clinical trials, where an overall response rate of 80% and a complete response in 74% of patients were reported. The review indicates potential advancement in treatment options for patients facing relapsed or refractory follicular lymphoma.
Read Announcement- Drug:
- Odronextamab
- Announced Date:
- December 9, 2024
- Indication:
- To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
Announcement
Regeneron Presents New And Updated Data For Odronextamab At ASH Annual Meeting
AI Summary
At the 66th American Society of Hematology Annual Meeting in San Diego, Regeneron presented new and updated data for its bispecific antibody odronextamab. The data included compelling findings from the Phase 3 OLYMPIA-1 trial’s safety lead-in, where odronextamab monotherapy produced complete responses in all evaluable patients with previously untreated follicular lymphoma by week 12. Regeneron also highlighted results from additional trials, including the ELM-1 study that showed durable responses in diffuse large B-cell lymphoma patients who had progressed after CAR-T therapy and the ELM-2 trial in marginal zone lymphoma, which reported a high complete response rate in heavily pretreated patients. These presentations underscore odronextamab’s potential as a promising, chemotherapy-free treatment option across various subtypes of B-cell non-Hodgkin lymphoma.
Read Announcement- Drug:
- Odronextamab
- Announced Date:
- August 26, 2024
- Indication:
- To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
Announcement
Regeneron Pharmaceuticals, Inc announced that the European Commission (EC) has approved Ordspono™ (odronextamab) to treat adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy.
AI Summary
Regeneron Pharmaceuticals announced that the European Commission has approved Ordspono™ (odronextamab) for adult patients with relapsed or refractory follicular lymphoma (FL) or diffuse large B-cell lymphoma (DLBCL) after at least two lines of systemic therapy. This approval marks the first regulatory nod for Ordspono and highlights its role as Regeneron’s first bispecific antibody. Ordspono works by linking lymphoma cells to killer T cells, which helps activate a targeted immune response against the cancer. Clinical trials have shown robust and durable responses in patients, including those who have previously received CAR-T therapy. The treatment offers a promising new option for both indolent and aggressive forms of lymphoma and can be administered in an outpatient setting, providing an off-the-shelf alternative with the potential for complete remission.
Read Announcement- Drug:
- Odronextamab
- Announced Date:
- June 28, 2024
- Indication:
- To treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies.
Announcement
Regeneron Pharmaceuticals, announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization of odronextamab to treat adults with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), after two or more lines of systemic therapy.
AI Summary
Regeneron Pharmaceuticals announced that the European Medicines Agency’s CHMP has given a positive opinion for the conditional marketing authorization of odronextamab. This new treatment is targeted at adults with relapsed/refractory follicular lymphoma (FL) or diffuse large B-cell lymphoma (DLBCL) who have received at least two lines of systemic therapy. FL is often a slow-growing but incurable disease, while DLBCL is an aggressive lymphoma with a risk of early relapse, making treatment options limited for these patients.
The positive opinion is based on promising results from the Phase 1 ELM-1 and Phase 2 ELM-2 trials, which showed robust and durable responses along with an acceptable safety profile. The final decision by the European Commission is expected in the coming months, which could offer a new therapeutic option for patients with challenging lymphoma subtypes.
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Pozelimab FDA Regulatory Events
Pozelimab is a drug developed by Regeneron Pharmaceuticals for the following indication: It is an IgG4 antibody that binds with high affinity to wild-type and variant human C5.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Pozelimab
- Announced Date:
- December 7, 2024
- Indication:
- It is an IgG4 antibody that binds with high affinity to wild-type and variant human C5.
Announcement
Regeneron Pharmaceuticals, Inc announced positive updated Phase 3 data of an exploratory cohort from the ACCESS-1 trial investigating its first-in-class pozelimab and cemdisiran (poze-cemdi) combination treatment against ravulizumab, a standard-of-care complement factor 5 (C5) inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
AI Summary
Regeneron Pharmaceuticals announced positive updated Phase 3 data from an exploratory cohort in the ACCESS-1 trial for its first-in-class combination treatment, pozelimab and cemdisiran (poze-cemdi). This study compared poze-cemdi with the standard treatment ravulizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The results showed that 96% of patients receiving poze-cemdi achieved adequate control of lactate dehydrogenase (LDH) levels, an important marker of hemolysis, compared to 80% in the ravulizumab group. Additionally, most patients maintained target LDH levels over the 26-week period, and those who switched from ravulizumab to poze-cemdi experienced remarkable improvement. These findings suggest that the complementary action of an antibody and an siRNA in poze-cemdi offers rapid and durable C5 inhibition, supporting further clinical development for PNH treatment.
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Sarilumab FDA Regulatory Events
Sarilumab is a drug developed by Regeneron Pharmaceuticals for the following indication: for the Treatment of Active Polyarticular Juvenile Idiopathic Arthritis (pJIA).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- sarilumab
- Announced Date:
- June 11, 2024
- Indication:
- for the Treatment of Active Polyarticular Juvenile Idiopathic Arthritis (pJIA)
Announcement
Regeneron Pharmaceuticals, Inc and Sanofi announced that the U.S. Food and Drug Administration has approved Kevzara® (sarilumab) for the treatment of patients weighing 63 kg or greater with active polyarticular juvenile idiopathic arthritis (pJIA), a form of arthritis that impacts multiple joints at a time.
AI Summary
Regeneron Pharmaceuticals and Sanofi announced that the U.S. Food and Drug Administration has approved Kevzara® (sarilumab) for treating patients weighing 63 kg or greater who suffer from active polyarticular juvenile idiopathic arthritis (pJIA). pJIA is a chronic condition where multiple joints become inflamed, often causing pain, stiffness, and swelling, which can seriously affect daily life and potentially lead to long-term joint damage. This approval is supported by robust studies, including evidence gathered from adult patients with rheumatoid arthritis and dedicated pediatric research. The new indication offers a much-needed treatment option for vulnerable pediatric patients and their families, aiming to improve quality of life by reducing the debilitating effects of the disease. This milestone reinforces Kevzara’s established efficacy and safety profile in managing inflammatory conditions.
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