This section highlights FDA-related milestones and regulatory updates for drugs developed by Biogen (BIIB).
Over the past two years, Biogen has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BIIB080/IONIS-MAPT, dapirolizumab, Felzartamab, Lecanemab, LEQEMBI®, nusinersen, and salanersen. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BIIB080/IONIS-MAPT - FDA Regulatory Timeline and Events
BIIB080/IONIS-MAPT is a drug developed by Biogen for the following indication: Mild Alzheimer's 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:
- BIIB080/IONIS-MAPT
- Announced Date:
- April 2, 2025
- Indication:
- Mild Alzheimer's disease
Announcement
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer's disease.
AI Summary
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its investigational therapy BIIB080. This novel antisense oligonucleotide (ASO) is designed to target tau protein, which plays a key role in Alzheimer’s disease. Fast Track designation is granted to therapies that address serious conditions and unmet medical needs, allowing for a faster review process. Dr. Priya Singhal, Head of Development at Biogen, stated that the FDA’s decision underlines the urgent need for new treatments targeting tau pathology. BIIB080 is the first tau-targeting ASO to move into clinical development for Alzheimer’s disease and is currently being evaluated in a global Phase 2 study, offering hope for innovative treatment options to patients and their families.
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dapirolizumab pegol - FDA Regulatory Timeline and Events
dapirolizumab pegol is a drug developed by Biogen for the following indication: In Systemic Lupus Erythematosus.
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:
- dapirolizumab pegol
- Announced Date:
- June 12, 2025
- Indication:
- In Systemic Lupus Erythematosus
Announcement
UCB and Biogen Inc today presented additional detailed results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol (DZP), a novel Fc-free anti-CD40L drug candidate.
AI Summary
UCB and Biogen Inc. presented new detailed results from the Phase 3 PHOENYCS GO study, which explored the effects of dapirolizumab pegol (DZP), a novel Fc‐free anti-CD40L drug candidate. The study focused on patients with moderate-to-severe systemic lupus erythematosus (SLE) and showed that DZP helped reduce fatigue and improve disease activity. At Week 48, a higher number of patients treated with DZP experienced low or no disease activity than those receiving the standard of care, with improvements seen as early as Week 12. Additionally, key patient-reported outcomes using scales like FACIT-Fatigue indicated a greater benefit for the DZP group. These promising results, presented at the European Alliance of Associations for Rheumatology (EULAR) 2025 meeting in Barcelona, suggest DZP could offer a new option to better manage SLE symptoms and improve patients’ quality of life.
Read Announcement- Drug:
- dapirolizumab pegol
- Announced Date:
- November 19, 2024
- Indication:
- In Systemic Lupus Erythematosus
Announcement
UCB and Biogen Inc. today presented detailed results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol (DZP), a novel Fc-free anti-CD40L drug candidate, demonstrating significant clinical improvement in disease activity in people living with moderate-to-severe systemic lupus erythematosus (SLE).
AI Summary
UCB and Biogen Inc. presented detailed results from the Phase 3 PHOENYCS GO study, which evaluated dapirolizumab pegol (DZP), a new anti-CD40L drug candidate, in people with moderate-to-severe systemic lupus erythematosus (SLE). The study met its primary endpoint by showing statistically and clinically significant improvement across all organ systems as measured by the BICLA, a tool that assesses disease activity. Notably, participants receiving DZP along with standard care experienced a 14.6% higher response rate, 50% fewer severe disease flares, and better outcomes on several clinical measures including reduced steroid use. These promising results suggest that DZP, by targeting the CD40L pathway, has the potential to offer a meaningful treatment option for those suffering from this chronic autoimmune disease.
Read Announcement- Drug:
- dapirolizumab pegol
- Announced Date:
- September 24, 2024
- Indication:
- In Systemic Lupus Erythematosus
Announcement
UCB and Biogen Inc. announced positive topline results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol, a novel Fc-free anti-CD40L drug candidate, in people living with moderate-to-severe systemic lupus erythematosus (SLE).
AI Summary
UCB and Biogen Inc. announced positive topline results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol, a novel Fc-free anti-CD40L drug candidate for people with moderate-to-severe systemic lupus erythematosus (SLE). In the study, dapirolizumab pegol, taken alongside standard-of-care treatment, met its primary endpoint by showing significant improvement in disease activity after 48 weeks, as measured by the British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA). The trial also showed improvements in key secondary endpoints that tracked disease activity and the frequency of flares.
With these encouraging results, UCB and Biogen are moving forward with further clinical trials, hoping to offer a new treatment option for patients who face limited choices. This progress is a promising step toward addressing the high unmet need in managing SLE, a chronic autoimmune disease that particularly affects women.
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Felzartamab - FDA Regulatory Timeline and Events
Felzartamab is a drug developed by Biogen for the following indication: For the Treatment of Antibody-Mediated Rejection in Kidney Transplant Recipients.
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:
- Felzartamab
- Announced Date:
- June 30, 2025
- Indication:
- For the Treatment of Antibody-Mediated Rejection in Kidney Transplant Recipients
Announcement
Biogen Inc. announced the initiation of dosing in the global clinical study, PROMINENT.
AI Summary
Biogen Inc. has begun dosing patients in the global Phase 3 clinical study, PROMINENT. This trial will compare the efficacy and safety of felzartamab, an investigational anti-CD38 monoclonal antibody, with tacrolimus in adults who have primary membranous nephropathy (PMN), a severe, immune-mediated kidney disease. Approximately 180 PMN patients will be enrolled in this study, which is expected to complete readouts in 2029. Felzartamab works by selectively depleting CD38+ plasma cells that produce harmful autoantibodies. By reducing these autoantibodies, the study hopes to achieve complete remission of proteinuria, a key indicator of PMN progression and a risk factor for kidney failure. This trial aims to address the unmet need for an approved, effective treatment in PMN, potentially transforming therapy for patients suffering from this challenging condition.
Read Announcement- Drug:
- Felzartamab
- Announced Date:
- March 11, 2025
- Indication:
- For the Treatment of Antibody-Mediated Rejection in Kidney Transplant Recipients
Announcement
Biogen Inc announced the initiation of dosing in the global clinical study, TRANSCEND.
AI Summary
Biogen Inc has begun dosing patients in its global Phase 3 TRANSCEND study. This trial will compare the investigational drug felzartamab with a placebo in approximately 120 adult kidney transplant recipients diagnosed with late antibody-mediated rejection (AMR), a common cause of transplant failure. TRANSCEND is a 52-week, double-blind, placebo-controlled study designed to evaluate both the efficacy and safety of felzartamab. Early results from a Phase 2 study showed promising first-in-class potential, which has led to the initiation of this larger trial. Biogen hopes that if felzartamab is proven to be effective, it could become a meaningful treatment option for patients suffering from late AMR, ultimately addressing the urgent need for better therapies in kidney transplant care.
Read Announcement- Drug:
- Felzartamab
- Announced Date:
- October 9, 2024
- Indication:
- For the Treatment of Antibody-Mediated Rejection in Kidney Transplant Recipients
Announcement
Biogen Inc. announced that felzartamab, an investigational anti-CD38 monoclonal antibody, has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for the treatment of late antibody-mediated rejection (AMR) without T-cell mediated rejection in kidney transplant patients.
AI Summary
Biogen Inc. announced that its investigational anti-CD38 monoclonal antibody, felzartamab, has received Breakthrough Therapy Designation (BTD) from the U.S. FDA. This designation is intended to accelerate the development and review process for drugs aimed at serious or life-threatening conditions. Felzartamab is being developed to treat late antibody-mediated rejection (AMR) without T-cell mediated rejection in kidney transplant patients—a condition that significantly contributes to transplant failure and represents an unmet medical need. The FDA granted this designation based on promising clinical results that showed the drug’s potential to effectively combat AMR. This status will allow Biogen to work more closely with the FDA and to move efficiently into Phase 3 trials, offering hope of a novel treatment option for kidney transplant recipients facing limited alternatives.
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Lecanemab (BAN2401) - FDA Regulatory Timeline and Events
Lecanemab (BAN2401) is a drug developed by Biogen for the following indication: Anti-amyloid beta (Aβ) protofibril antibody for the treatment of Alzheimer's disease (AD).
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:
- Lecanemab (BAN2401)
- Announced Date:
- January 31, 2025
- Indication:
- Anti-amyloid beta (Aβ) protofibril antibody for the treatment of Alzheimer's disease (AD)
Announcement
Eisai Co and Biogen Inc announced an update on the ongoing regulatory review of the Marketing Authorization Application for lecanemab as treatment for early AD (mild cognitive impairment due to Alzheimer's disease (AD) and mild AD) in the European Union.
AI Summary
Eisai Co. and Biogen Inc. provided an update on the regulatory review of their Marketing Authorization Application for lecanemab in the European Union. The treatment, aimed at early Alzheimer’s disease—including mild cognitive impairment and mild AD—received a positive opinion from the Committee for Medicinal Products for Human Use in November 2024. However, the European Commission has requested that the CHMP review new safety information that has become available since that opinion was issued, as well as clarify the language on risk minimization measures. These issues are scheduled to be discussed in the upcoming CHMP meeting in February 2025. Both companies remain confident that the additional safety details can be addressed using the existing data and are working closely with authorities to secure approval and deliver lecanemab to patients as soon as possible within the EU.
Read Announcement- Drug:
- Lecanemab (BAN2401)
- Announced Date:
- July 26, 2024
- Indication:
- Anti-amyloid beta (Aβ) protofibril antibody for the treatment of Alzheimer's disease (AD)
Announcement
Eisai Co., and Biogen Inc. announced that that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the Marketing Authorization Approval (MAA) for the humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody lecanemab as treatment for early AD (mild cognitive impairment due to Alzheimer's disease (AD) and mild AD).1
AI Summary
Eisai Co. and Biogen Inc. announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion on the marketing authorization application for lecanemab. This humanized monoclonal antibody targets soluble aggregated amyloid-beta and is aimed at treating early Alzheimer’s disease, including mild cognitive impairment due to AD and mild AD. The companies expressed disappointment with the decision, emphasizing the significant need for new, effective treatments that address disease progression in Alzheimer’s patients. Despite the setback, Eisai plans to request a re-examination of the CHMP’s opinion and will collaborate with relevant authorities to bring the treatment to eligible patients in the European Union as soon as possible. This decision marks a challenging turn for lecanemab, which is already approved in several other countries.
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LEQEMBI® (lecanemab-irmb) - FDA Regulatory Timeline and Events
LEQEMBI® (lecanemab-irmb) is a drug developed by Biogen for the following indication: Treatment of Alzheimer's 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:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- February 28, 2025
- Indication:
- Treatment of Alzheimer's Disease
Announcement
BioArctic AB announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has reaffirmed its positive opinion for the anti-Aβ monoclonal antibody lecanemab (Leqembi®), adopted in November 2024.
AI Summary
BioArctic AB, in partnership with Eisai, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has reaffirmed its positive opinion for the anti-Aβ monoclonal antibody lecanemab (Leqembi®), originally adopted in November 2024. The CHMP reviewed additional safety data requested by the European Commission and concluded that the initial positive opinion remains unchanged. Following this reaffirmation, the European Commission will now resume its decision-making process to grant marketing authorization for lecanemab across the European Union and associated countries, including Iceland, Liechtenstein, and Norway.
Lecanemab is designed to target and reduce harmful amyloid-beta aggregates that contribute to early Alzheimer’s disease. This continued regulatory support is an important step toward making lecanemab available to patients in need.
Read Announcement- Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- January 26, 2025
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Eisai Co., Ltd. and Biogen Inc announced that the U.S. Food and Drug Administration (FDA) has approved the Supplemental Biologics License Application (sBLA) for once every four weeks lecanemab-irmb (U.S. brand name: LEQEMBI®) intravenous (IV) maintenance dosing. LEQEMBI is indicated for the treatment of Alzheimer's disease (AD) in patients with mild cognitive impairment (MCI) or mild dementia stage of disease (collectively referred to as early AD) in the U.S.
AI Summary
Eisai Co., Ltd. and Biogen Inc. announced that the U.S. FDA approved a Supplemental Biologics License Application (sBLA) for LEQEMBI® (lecanemab-irmb) using a once‐every‐four-weeks intravenous maintenance dosing regimen. This approval targets patients with early Alzheimer’s disease, including those with mild cognitive impairment or mild dementia. The new dosing schedule comes after an 18-month phase where patients initially receive treatment every two weeks, with a transition option to a 10 mg/kg dosing every four weeks. Modeling data from Phase 2 and Phase 3 studies suggest that switching to the four-week maintenance dose will continue to deliver clinical benefits and help slow the progression of Alzheimer’s disease. This updated dosing regimen may offer an easier treatment schedule for patients and their caregivers.
Read Announcement - Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- January 13, 2025
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Eisai Co., Ltd. and Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted Eisai's Biologics License Application (BLA) for lecanemab-irmb (U.S. brand name: LEQEMBI®) subcutaneous autoinjector (SC-AI) for weekly maintenance dosing.
AI Summary
Eisai Co., Ltd. and Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted Eisai’s Biologics License Application (BLA) for its lecanemab-irmb subcutaneous autoinjector (SC-AI), marketed as LEQEMBI®. This new formulation is designed for weekly maintenance dosing in patients with early Alzheimer’s disease, specifically those with mild cognitive impairment or mild dementia. The acceptance is based on data from the Clarity AD open-label extension study and modeling analyses. If approved, LEQEMBI’s subcutaneous option would be the only Alzheimer’s treatment that patients could self-administer at home using an autoinjector, potentially reducing the need for frequent hospital visits. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date for August 31, 2025, which signals when a decision on this innovative treatment method might be expected.
Read Announcement- Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- August 22, 2024
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Eisai Co., Ltd. and Biogen Inc. announced that the humanized amyloid-beta (Aβ) monoclonal antibody "Leqembi®" (brand name, generic name: lecanemab) has been granted a Marketing Authorization by the Medicines and Healthcare products Regulatory Agency (MHRA) in Great Britain.
AI Summary
Eisai Co., Ltd. and Biogen Inc. announced that their humanized amyloid-beta monoclonal antibody, Leqembi® (lecanemab), has received Marketing Authorization from Great Britain’s Medicines and Healthcare products Regulatory Agency (MHRA). This approval marks the first authorization in Europe for a treatment that targets an underlying cause of Alzheimer’s disease. Leqembi is indicated for adult patients with mild cognitive impairment and mild dementia due to Alzheimer’s, specifically for those who are apolipoprotein E ε4 heterozygotes or non-carriers.
The decision was based on strong Phase 3 clinical trial results that demonstrated the treatment’s ability to slow cognitive decline. Eisai and Biogen are working together to ensure that eligible patients gain timely access to this innovative therapy, representing a significant advancement in the management of early Alzheimer’s disease in Europe.
Read Announcement- Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- July 30, 2024
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Eisai Co., Ltd. announced today that the latest findings for lecanemab-irmb (U.S. brand name: LEQEMBI®), an anti-amyloid beta (Aβ) protofibril* antibody for the treatment of early Alzheimer's disease (AD), were presented at the Alzheimer's Association International Conference (AAIC) 2024, held in Philadelphia, USA, and virtually. and Biogen Inc
AI Summary
Eisai Co., Ltd. and Biogen Inc. announced new findings at the Alzheimer’s Association International Conference (AAIC) 2024, held both in Philadelphia and online. The data focused on lecanemab‐irmb (U.S. brand name: LEQEMBI®), an anti-amyloid beta (Aβ) protofibril antibody developed for treating early Alzheimer’s disease. Lecanemab-irmb is designed to clear highly toxic protofibrils—damaging particles that continue to hurt brain cells even after the removal of amyloid plaques—thereby supporting neuronal function and slowing the spread of tau, a protein linked to disease progression.
Eisai leads the global development and regulatory submissions for lecanemab-irmb, while Biogen collaborates in its co-commercialization and promotion. The presentation at AAIC 2024 highlighted the potential of early intervention with this dual-acting treatment to improve outcomes for patients with early Alzheimer’s disease.
Read Announcement- Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- June 9, 2024
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Biogen Inc and Eisai Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has accepted Eisai's Supplemental Biologics License Application (sBLA) for monthly lecanemab-irmb (U.S. brand name: LEQEMBI®) intravenous (IV) maintenance dosing.
AI Summary
Biogen Inc. and Eisai Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has accepted Eisai’s Supplemental Biologics License Application (sBLA) for monthly lecanemab-irmb (U.S. brand name: LEQEMBI®) intravenous maintenance dosing. Under this proposed regimen, patients who complete the initial biweekly dosing phase would switch to a monthly infusion, which helps maintain effective drug levels to clear toxic protofibrils implicated in Alzheimer’s disease.
The acceptance is based on modeling from Phase 2 and Phase 3 studies, supporting the sustained therapeutic benefits of LEQEMBI in early Alzheimer’s patients. A Prescription Drug User Fee Act action date is set for January 25, 2025. This development demonstrates continued collaboration between Eisai and Biogen to enhance treatment options for Alzheimer’s disease.
Read Announcement- Drug:
- LEQEMBI® (lecanemab-irmb)
- Announced Date:
- May 14, 2024
- Indication:
- Treatment of Alzheimer's Disease
Announcement
Eisai Co., Ltd and Biogen Inc. announced that that Eisai has initiated the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lecanemab-irmb (U.S. brand name: LEQEMBI®) subcutaneous autoinjector for weekly maintenance dosing after it was granted Fast Track designation by the FDA.
AI Summary
Eisai Co., Ltd. and Biogen Inc. have announced an important advancement in their efforts to bring innovative treatments to patients. Eisai has started the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its lecanemab-irmb subcutaneous autoinjector. This device, known in the U.S. as LEQEMBI®, is designed for weekly maintenance dosing.
The submission follows the FDA’s Fast Track designation, a status that can speed up the review process for promising therapies. By using a rolling submission process, the FDA can evaluate sections of the application as they become available, which may help bring the treatment to market more quickly. This step reflects the companies’ commitment to accelerating access to this potentially beneficial therapy.
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nusinersen - FDA Regulatory Timeline and Events
nusinersen is a drug developed by Biogen for the following indication: In Treatment of spinal muscular atrophy (SMA).
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:
- nusinersen
- Announced Date:
- January 23, 2025
- Indication:
- In Treatment of spinal muscular atrophy (SMA).
Announcement
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA)
AI Summary
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for a higher dose regimen of nusinersen. This new regimen is designed to treat spinal muscular atrophy (SMA) more effectively. It includes a more rapid loading phase with two 50 mg doses given 14 days apart, followed by maintenance doses of 28 mg every four months. This differs from the current approved regimen of 12 mg, which is commercialized under the name SPINRAZA in over 71 countries.
The company believes that the higher dose regimen could offer significant benefits for patients and their families and is a reflection of Biogen’s commitment to advancing new treatment options for SMA. With the sNDA now under review, Biogen looks forward to progressing in providing improved care and potential clinical benefits to the SMA community.
Read Announcement- Drug:
- nusinersen
- Announced Date:
- January 23, 2025
- Indication:
- In Treatment of spinal muscular atrophy (SMA).
Announcement
Biogen Inc. announced that the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA).
AI Summary
Biogen announced that the European Medicines Agency (EMA) has validated its application for a higher dose regimen of nusinersen for treating spinal muscular atrophy (SMA). This new treatment plan includes a faster loading phase with two 50 mg doses administered 14 days apart, followed by a higher maintenance dose of 28 mg every four months, in contrast to the current 12 mg dose used in SPINRAZA®. Biogen expects that this enhanced regimen could lead to better patient outcomes by delivering higher doses early in treatment while maintaining a similar safety profile.
The EMA validation marks an important step in Biogen’s commitment to improving SMA therapies. With promising results from recent clinical studies, the company is optimistic that the higher dose regimen will provide meaningful benefits, potentially improving the quality of life for individuals and families affected by SMA.
Read Announcement- Drug:
- nusinersen
- Announced Date:
- October 8, 2024
- Indication:
- In Treatment of spinal muscular atrophy (SMA).
Announcement
Biogen Inc. announced detailed results from Part B and Part C of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of an investigational higher dose regimen of nusinersen in spinal muscular atrophy (SMA), showing benefits in both individuals previously treated and treatment-naïve to nusinersen with infantile-onset or later-onset SMA.
AI Summary
Biogen Inc. recently announced detailed results from Parts B and C of the Phase 2/3 DEVOTE study. This study evaluated a higher dose nusinersen regimen for treating spinal muscular atrophy (SMA) in both individuals who were treatment-naïve and those who had previously received nusinersen. The investigational regimen features a faster loading phase with two 50 mg doses given 14 days apart, followed by regular maintenance doses of 28 mg every four months, compared to the approved regimen.
The study results indicate that the higher dose not only improves motor function in infants and older patients but also slows neurodegeneration more rapidly by lowering neurofilament levels, a marker of nerve cell damage. Based on these promising findings, Biogen plans to submit regulatory applications worldwide in hopes of addressing unmet needs in the SMA community.
Read Announcement- Drug:
- nusinersen
- Announced Date:
- September 4, 2024
- Indication:
- In Treatment of spinal muscular atrophy (SMA).
Announcement
Biogen Inc. announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a higher dose regimen of nusinersen in treatment-naïve, symptomatic infants with spinal muscular atrophy (SMA).
AI Summary
Biogen announced encouraging topline results from the pivotal Part B cohort of its Phase 2/3 DEVOTE study, which evaluated a higher dose regimen of nusinersen in treatment-naïve, symptomatic infants with spinal muscular atrophy (SMA). The study met its primary endpoint at six months, demonstrating a statistically significant improvement in motor function compared to a matched, untreated sham control group. This investigational regimen features a more rapid loading phase with two 50 mg doses administered 14 days apart, followed by a higher maintenance dose of 28 mg every four months, in contrast to the approved 12 mg dose.
The results also showed favorable trends in key biomarkers and secondary endpoints, suggesting that the higher dose regimen may slow neurodegeneration more effectively. These promising findings support Biogen’s plan to pursue regulatory approval for this enhanced dosing strategy for infants with SMA.
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salanersen - FDA Regulatory Timeline and Events
salanersen is a drug developed by Biogen for the following indication: For the treatment of spinal muscular atrophy (SMA).
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:
- salanersen
- Announced Date:
- June 25, 2025
- Indication:
- For the treatment of spinal muscular atrophy (SMA).
Announcement
Biogen Inc. announced topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for the treatment of spinal muscular atrophy (SMA).
AI Summary
Biogen Inc. announced promising topline results from its Phase 1 study of salanersen (BIIB115/ION306), a new antisense oligonucleotide aimed at treating spinal muscular atrophy (SMA). The interim data showed that children with SMA who previously received gene therapy experienced a significant slowing of neurodegeneration, as measured by a reduction in neurofilament levels, along with meaningful improvements in motor function. Salanersen, which works similarly to SPINRAZA but is designed for enhanced potency and the convenience of once‐yearly dosing, was tested at both 40 mg and 80 mg doses and was generally well tolerated. Encouraged by these early results, Biogen is collaborating with regulators to plan Phase 3 registrational studies to further evaluate the treatment’s benefits in both previously treated and treatment‐naïve patients.
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SPINRAZA (Nusinersen) - FDA Regulatory Timeline and Events
SPINRAZA (Nusinersen) is a drug developed by Biogen for the following indication: Spinal muscular atrophy (SMA).
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:
- SPINRAZA (Nusinersen)
- Announced Date:
- June 25, 2025
- Indication:
- Spinal muscular atrophy (SMA)
Announcement
Ionis Pharmaceuticals, Inc. announced that its partner, Biogen, shared positive topline results from the Phase 1 study of salanersen (ION306/BIIB115), an investigational antisense oligonucleotide (ASO) being developed for the potential treatment of spinal muscular atrophy (SMA).
AI Summary
Ionis Pharmaceuticals announced positive topline results from the Phase 1 study of salanersen (ION306/BIIB115), an investigational antisense oligonucleotide being developed with partner Biogen for treating spinal muscular atrophy (SMA). The study showed that children with SMA who had previously received gene therapy experienced a substantial slowing of neurodegeneration and significant improvements in motor function. Both tested doses – 40 mg and 80 mg, given once-yearly – were generally well-tolerated, and reductions in neurofilament levels indicated slowed disease progression. Biogen plans to engage with regulators to move salanersen into registrational stage studies. Ionis highlighted that salanersen was created using their novel antisense chemistry, which may offer higher potency and the benefit of long-interval dosing, potentially offering improved treatment options for the SMA community.
Read Announcement- Drug:
- SPINRAZA (Nusinersen)
- Announced Date:
- July 31, 2024
- Indication:
- Spinal muscular atrophy (SMA)
Announcement
Alcyone Therapeutics Inc. announced today that the U.S. Food and Drug Administration (FDA) has provided approval to continue enrollment of the PIERRE study (https://clinicaltrials.gov/ct2/show/NCT05866419) to evaluate the safety and effectiveness of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system for chronic intrathecal access, CSF aspiration, and delivery of SPINRAZA® (nusinersen) in SMA patients as an alternative to repeat lumbar puncture (LP).
AI Summary
Alcyone Therapeutics Inc. announced that the FDA has approved the continuation of enrollment for the PIERRE study, which is designed to evaluate the safety and effectiveness of the ThecaFlex DRx™ subcutaneous port and intrathecal catheter system. This innovative device aims to provide chronic intrathecal access for cerebrospinal fluid (CSF) aspiration and the delivery of SPINRAZA® (nusinersen) in patients with spinal muscular atrophy (SMA), offering an alternative to the traditional repeat lumbar puncture. In the first stage of the trial, 10 SMA patients successfully underwent ThecaFlex implantation with no device-related adverse events observed within 30 days. Based on the positive initial clinical data, the FDA has allowed an additional 80 patients to be enrolled across up to 30 centers in the United States and Europe, marking an important step toward a less invasive treatment method for SMA patients.
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TOFIDENCE - FDA Regulatory Timeline and Events
TOFIDENCE is a drug developed by Biogen for the following indication: For the treatment of moderately to severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.
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:
- TOFIDENCE
- Announced Date:
- September 11, 2024
- Indication:
- For the treatment of moderately to severely active rheumatoid arthritis, polyarticular juvenile idiopathic arthritis and systemic juvenile idiopathic arthritis.
Announcement
Bio-Thera Solutions, Ltd. announced, in collaboration with Biogen, the publication in the journal Arthritis Research & Therapy of results from Treatment Period 2 (TP2; study weeks 24-48) of a Phase 3 clinical study evaluating BAT1806/BIIB800, an approved biosimilar to Actemra®/RoActemra®3 (tocilizumab). BAT1806/BIIB800 is currently commercialized under the brand name TOFIDENCE™4.
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