REGENXBIO (RGNX) has upcoming FDA regulatory milestones for RGX-202 and surabgene lomparvovec. The table below outlines estimated target dates and event types for these pending regulatory actions.
This section highlights FDA-related milestones and regulatory updates for drugs developed by REGENXBIO (RGNX).
Over the past two years, REGENXBIO has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
RGX-202, RGX-121, ABBV-RGX-314, and surabgene. 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.
RGX-202 FDA Regulatory Timeline and Events
RGX-202 is a drug developed by REGENXBIO for the following indication: Duchenne Muscular Dystrophy.
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:
- RGX-202
- Announced Date:
- May 7, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced presentations at the 2026 American Society of Gene & Cell Therapy Annual Meeting ("ASGCT 2026") taking place May 11-15, 2026, in Boston, Massachusetts.
AI Summary
REGENXBIO Inc. said it will present new data and research at the 2026 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, held May 11–15, 2026, in Boston, Massachusetts. The company plans to share updates on its gene therapy programs and study results with the scientific community during the meeting. Presentations will cover REGENXBIO’s ongoing work in AAV-based therapeutics and related preclinical and clinical findings.
All slides and posters presented at ASGCT 2026 will be posted on REGENXBIO’s Publications page at www.regenxbio.com. For media or investor inquiries, the company provided contacts: Dana Cormack, Corporate Communications, and George E. MacDougall, Investor Relations. The announcement directs interested parties to the company website for the full set of materials after the meeting.Read Announcement
- Drug:
- RGX-202
- Announced Date:
- March 11, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy.
AI Summary
REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, its gene therapy candidate for Duchenne muscular dystrophy (DMD). The company reported that early results show encouraging signs of benefit and an acceptable safety profile in treated patients, based on the data collected so far.
RGX-202 is designed to deliver a therapeutic gene to muscle tissue to address the underlying cause of DMD. The interim findings suggest the therapy may improve measures related to muscle health and function, supporting continued enrollment and further study in the trial.
While these are early results and more data are needed, REGENXBIO says the outcomes support advancing development. If later studies confirm these signals, RGX-202 could become an important new treatment option for people living with Duchenne muscular dystrophy. More details are expected as the trial progresses.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- January 11, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc highlighted progress and upcoming anticipated milestones across its pipeline of AAV gene therapies for rare and retinal diseases.
AI Summary
REGENXBIO highlighted progress and upcoming milestones across its pipeline of AAV gene therapies for rare and retinal diseases. New 18‑month functional data from the RGX‑202 Duchenne trial showed durable benefit at the pivotal dose, with patients improving an average of 7.4 points on the NSAA versus expected disease trajectory. The company plans to share more Phase I/II safety, biomarker, and functional data in March 2026 and expects pivotal topline results in early Q2 2026, followed by a mid‑year BLA filing. Enrollment in the pivotal study is complete and the confirmatory trial is continuing, with most patients expected to be dosed by the BLA submission.
REGENXBIO also reported progress on clemidsogene lanparvovec for MPS II and surabgene lomparvovec for wet AMD and diabetic retinopathy, backed by partners and commercial planning. In‑house manufacturing at its Rockville center is scaling supply, process qualification lots for RGX‑202 are complete, and new capsids are advancing toward IND readiness for additional retinal programs. The company expects multiple pivotal readouts in 2026 to support potential launches in 2026–2028.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- October 30, 2025
- Target Action Date:
- Q2 2026
- Estimated Target Date Range:
- April 1, 2026 - June 30, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced that Topline pivotal data now expected in early Q2 2026
AI Summary
REGENXBIO’s AFFINITY DUCHENNE® trial of RGX-202 has completed enrollment of 30 participants in its pivotal phase. Early Phase I/II data showed microdystrophin levels ranging from 20% to 122% at the pivotal dose, with no serious adverse events reported. Treated patients also demonstrated consistent functional improvements compared to natural history controls.
The company continues to recruit ambulatory participants aged one year and above for the confirmatory portion of the study. REGENXBIO has manufactured its first batches of RGX-202 for commercial supply at its Rockville, Maryland, Manufacturing Innovation Center. Using the NAVXpress® process, the facility can produce up to 2,500 doses per year.
Topline pivotal data are now expected in early Q2 2026, with a Biologics License Application planned for mid-2026. REGENXBIO aims for a commercial launch in 2027, offering a potential one-time gene therapy option for Duchenne muscular dystrophy.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- October 30, 2025
- Target Action Date:
- H1 2026
- Estimated Target Date Range:
- January 1, 2026 - June 30, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced that BLA submission in mid-2026
AI Summary
REGENXBIO Inc. has finished enrolling patients in the pivotal Phase I/II/III AFFINITY DUCHENNE® trial for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy. The company also produced the first batches intended for commercial use at its in-house Manufacturing Innovation Center, which can make up to 2,500 doses per year.
Topline data from the pivotal trial are expected in early Q2 2026. If results support safety and efficacy, REGENXBIO plans to submit a Biologics License Application (BLA) to the FDA in mid-2026. The submission would enable consideration for accelerated approval based on microdystrophin expression and functional improvements observed so far.
Meanwhile, REGENXBIO continues enrolling ambulatory participants aged one year and older in the confirmatory study. Positive trial results and timely regulatory review could bring RGX-202 closer to patients in need of new, durable treatment options.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- October 30, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced the completion of enrollment in the AFFINITY DUCHENNE® pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply.
AI Summary
REGENXBIO Inc. announced it has finished enrolling 30 participants in its pivotal AFFINITY DUCHENNE® trial of RGX-202, a gene therapy for Duchenne muscular dystrophy. The trial will measure microdystrophin expression and changes in timed function tests to support an accelerated approval pathway.
Earlier Phase I/II data showed microdystrophin levels ranging from 20% to 122% at the pivotal dose and clear functional improvements versus natural history controls. No serious adverse events or safety concerns were reported.
REGENXBIO’s in-house Manufacturing Innovation Center has also produced the first batches of RGX-202 intended for commercial supply. Using its NAVXpress® process, the center can manufacture up to 2,500 doses per year with industry-leading product purity.
Topline pivotal data are now expected in early Q2 2026, with a Biologics License Application planned for mid-2026. If approved, commercial launch could begin in 2027.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- September 29, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc announced Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society taking place in Vienna, Austria, October 7-11, 2025.
AI Summary
REGENXBIO Inc. announced that its Chief Medical Officer, Steve Pakola, M.D., will present at the International Congress of the World Muscle Society in Vienna, Austria, from October 7–11, 2025. His podium talk will share fresh analysis of 12-month functional data from the Phase I/II trial of RGX-202 in Duchenne muscular dystrophy patients. Using the North Star Ambulatory Assessment and the cTAP disease progression model, the presentation highlights individual patient improvements over baseline.
RGX-202 showed a favorable safety profile, with no serious adverse events or events of special interest. Participants receiving the pivotal dose outperformed matched external natural history controls on all functional measures. These findings support RGX-202’s potential as a differentiated, best-in-class gene therapy for Duchenne muscular dystrophy.
The session “Short Oral Presentations 1: Updates on SMA and DMD Trials,” including an accompanying poster (P425), takes place on October 8 at 3:30 CET. The presentation will be available on REGENXBIO’s website.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- July 10, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) domain to a microdystrophin construct without the CT domain.
AI Summary
REGENXBIO Inc. recently published preclinical research that compared two microdystrophin gene therapy constructs—one with the C-terminal (CT) domain and one without. The study, conducted in mice lacking dystrophin, found that the construct including the CT domain led to higher levels of microdystrophin protein, better recruitment of associated proteins, increased muscle force, and improved resistance against muscle damage.
This research supports the potential of REGENXBIO’s investigational gene therapy, RGX-202, for treating Duchenne Muscular Dystrophy. By including the CT domain—a critical part of the naturally occurring dystrophin protein—the therapy may offer improved muscle protection and longer-lasting effects compared to other designs. The findings suggest that the enhanced design of RGX-202 could significantly benefit patients by preserving muscle health and function.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- June 5, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial.
AI Summary
REGENXBIO Inc. recently shared positive interim data from the Phase I/II AFFINITY DUCHENNE trial for its investigational gene therapy RGX-202, aimed at treating Duchenne muscular dystrophy (DMD). The trial showed that patients receiving dose level 2 of RGX-202 experienced functional improvements, as measured by the North Star Ambulatory Assessment and timed function tests, exceeding performance compared to external natural history controls. This suggests that RGX-202 may positively change the disease progression for DMD patients.
Additionally, biomarker data showed robust microdystrophin expression across all treated ages. Notably, one 2-year-old patient reached an expression level of 118.6% compared to control levels. The therapy was well tolerated with no serious adverse events, supporting its potential as a transformative treatment for DMD. These promising results are expected to bolster the planned Biologics License Application submission by mid-2026.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- June 2, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation investigational gene therapy for the treatment of Duchenne muscular dystrophy.
AI Summary
REGENXBIO Inc. announced it will host a live webcast on Thursday, June 5, 2025, at 8:00 a.m. EDT to share new interim functional data from its Phase I/II AFFINITY DUCHENNE® trial of RGX-202. RGX-202 is the company’s next-generation investigational gene therapy aimed at treating Duchenne muscular dystrophy. During the webcast, Dr. Aravindhan Veerapandiyan, the principal investigator of the trial at Arkansas Children’s Hospital, will present the latest updates and insights on the therapy’s performance. Interested parties can access the live webcast through REGENXBIO’s website, with an archived replay available for about 30 days after the event. This update highlights the ongoing progress in gene therapy research and REGENXBIO’s commitment to advancing new treatment options for patients with Duchenne muscular dystrophy.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- March 19, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy (Duchenne).
AI Summary
REGENXBIO announced positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial for RGX-202, a promising gene therapy for Duchenne muscular dystrophy. The data, presented at the 2025 MDA Clinical & Scientific Conference, came from an age 1-3 cohort. A notable finding included a 3-year-old patient who demonstrated microdystrophin expression levels at 122.3% compared to control. These results add to those seen in older patients and show consistent, robust microdystrophin production and proper localization to the sarcolemma in all treated ages. Additionally, the trial showed encouraging safety outcomes with no serious adverse events or adverse events of special interest. The ongoing study supports RGX-202’s potential to improve functional outcomes for a wide range of patients with Duchenne muscular dystrophy, and enrollment continues as the company advances toward a Biologics License Application submission by mid-2026.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- March 10, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202 for the treatment of Duchenne muscular dystrophy will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX, March 16-19, 2025.
AI Summary
REGENXBIO Inc. announced that new interim biomarker data from the Phase I/II portion of its AFFINITY DUCHENNE® trial, studying RGX-202 for Duchenne muscular dystrophy, will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. This gene therapy research aims to offer a new treatment option for patients suffering from the debilitating condition.
The data presentation is scheduled to take place in Dallas, TX, during the conference held from March 16 to 19, 2025. A key session on Wednesday, March 19, 2025 at 8:15 a.m. CTP will focus on the interim clinical findings, while other sessions will include pre-clinical research insights. Further details and presentation materials will be available on REGENXBIO’s website.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- November 18, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO announced that AFFINITY DUCHENNE®, the multi-center, open-label trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy (Duchenne), has advanced to pivotal stage and dosed its first patient.
AI Summary
REGENXBIO Inc. announced that its AFFINITY DUCHENNE® trial for RGX-202—a promising gene therapy for Duchenne muscular dystrophy—has moved into the pivotal stage and dosed its first patient. This multi-center, open-label study is enrolling ambulatory patients aged one and older to evaluate the therapy’s effectiveness and safety. Positive data from earlier Phase I/II studies showed consistent microdystrophin expression in muscles and encouraging functional improvements, with patients exhibiting stable or better performance on key mobility tests. With a favorable safety profile reported at both dose levels, these results have supported discussions with the FDA. The company plans to file a Biologics License Application in 2026 under an accelerated approval pathway, marking an important step toward offering a potentially best-in-class treatment for boys affected by this devastating disease.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- November 18, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO announced new, positive efficacy and safety data from the Phase I/II portion of the study, including the first functional data.
AI Summary
REGENXBIO announced positive new data from the Phase I/II portion of its study for RGX-202, a gene therapy aimed at treating Duchenne muscular dystrophy. The study delivered the first functional data showing that patients treated with RGX-202 experienced stable or improved muscle function. Improvements were observed using tests like the North Star Ambulatory Assessment (NSAA) and timed function tests at both 12 and nine months for different dose levels. Biomarker results further supported the findings by showing a strong and consistent expression of the microdystrophin protein in treated muscles. Importantly, no serious adverse events were reported, underlining a favorable safety profile. These promising efficacy and safety results highlight RGX-202’s potential to positively change the disease trajectory and offer new hope to patients with Duchenne muscular dystrophy.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- June 24, 2024
- Target Action Date:
- Q3 2024
- Estimated Target Date Range:
- July 1, 2024 - September 30, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced that Remains on track to initiate pivotal trial in late Q3 to early Q4 2024
AI Summary
REGENXBIO Inc. announced that its pivotal trial for RGX-202 in patients with Duchenne muscular dystrophy remains on schedule to start in late Q3 to early Q4 2024. This trial will assess the investigational one-time AAV Therapeutic designed with a novel microdystrophin that incorporates the C-Terminal domain, a key component that makes it closely mimic naturally occurring dystrophin. The forthcoming trial is a major part of the company’s strategy to collect essential data that could support a Biologics License Application using an accelerated approval pathway, addressing the high unmet need for effective Duchenne treatments. In addition, REGENXBIO is actively enrolling patients and plans to finalize the pivotal trial design during an end-of-Phase II meeting with the FDA in late July. This development reinforces the company’s commitment to advancing innovative therapies for Duchenne patients.
Read Announcement- Drug:
- RGX-202
- Announced Date:
- June 24, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
REGENXBIO Inc. announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial to evaluate the safety and efficacy of RGX-202 in boys with Duchenne muscular dystrophy (Duchenne).
AI Summary
REGENXBIO Inc. has started enrolling patients aged 1 to 3 years in its Phase I/II AFFINITY DUCHENNE trial. The study aims to evaluate the safety and efficacy of RGX-202, an investigational one-time gene therapy for boys with Duchenne muscular dystrophy. RGX-202 is designed to deliver a novel microdystrophin that more closely mimics the naturally occurring protein, thanks to the inclusion of the C-Terminal domain. This unique design is expected to help preserve muscle function and offer a promising treatment option for a younger group of patients. The data collected from this cohort will be integrated into a larger pivotal trial dataset, supporting a faster path toward a Biologics License Application and potential broad label approval. The trial continues REGENXBIO’s efforts to bring innovative treatment solutions to the Duchenne community.
Read Announcement
RGX-121 FDA Regulatory Timeline and Events
RGX-121 is a drug developed by REGENXBIO for the following indication: MPS II (Hunter Syndrome).
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:
- RGX-121
- Announced Date:
- February 9, 2026
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec) for the treatment of Mucopolysaccharidosis II (MPS II), an ultra-rare neurodegenerative disease also known as Hunter syndrome.
AI Summary
REGENXBIO announced the U.S. Food and Drug Administration has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for RGX-121 (clemidsogene lanparvovec), an investigational gene therapy for Mucopolysaccharidosis II (MPS II or Hunter syndrome). The CRL means the FDA will not approve the application in its current form.
The company plans to request a Type A meeting with the FDA to discuss the CRL and a clear path forward. REGENXBIO said it will provide additional evidence from global MPS II experts to better define the neuronopathic patient population and submit longer-term clinical data to strengthen support for effectiveness. The company intends to move as quickly as possible toward a BLA resubmission.
The original BLA was supported by positive biomarker, functional, and safety data from the CAMPS I/II/III trial through 12 months, and RGX-121 was reported to be well tolerated across those trial phases.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- January 28, 2026
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I, also known as Hurler syndrome, following preliminary analysis of a single case of neoplasm (intraventricular CNS tumor) in a participant treated in its Phase I/II study.
AI Summary
REGENXBIO said the U.S. Food and Drug Administration placed a clinical hold on its investigational gene therapy RGX-111 for MPS I after a preliminary analysis of a single intraventricular CNS tumor in a Phase I/II participant. The tumor was discovered on a routine brain MRI in an otherwise asymptomatic five-year-old who received intracisternal RGX-111 four years earlier. REGENXBIO is awaiting the full clinical hold letter and additional details from the FDA.
Genetic testing of the removed tumor detected integration of the AAV vector genome with overexpression of the proto-oncogene PLAG1, which can be prone to chromosomal rearrangements. The company says the investigation is ongoing and causality has not been established. The child remains asymptomatic and has shown developmental progress. No neoplasms have been reported in nine other RGX-111 participants or in 32 participants treated with RGX-121.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- September 5, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced new, positive data from the Phase I/II/III CAMPSIITE® trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025.
AI Summary
At ICIEM 2025, REGENXBIO presented new results from its Phase I/II/III CAMPSIITE trial of RGX-121 (clemidsogene lanparvovec) for boys with MPS II (Hunter syndrome). Twelve-month pivotal data showed an 82% median reduction in cerebrospinal fluid heparan sulfate D2S6, a key biomarker of MPS II brain disease, sustained through one year. These findings align with earlier pivotal results, which met the primary endpoint of CSF HS D2S6 reduction at week 16 with high statistical significance.
Participants in the pivotal phase also demonstrated continued neurodevelopmental skill acquisition or stability, measured by the Bayley Scales of Infant and Toddler Development (BSID-III), through one year. New analyses revealed a strong correlation between early biomarker reductions and neurocognitive outcomes at 12 months, supporting HS D2S6 as a surrogate endpoint likely to predict clinical benefit.
If approved, RGX-121 would be the first one-time gene therapy designed to directly address the underlying genetic cause of Hunter syndrome, offering the potential to change its trajectory in affected children.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- August 18, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that the U.S. Food and Drug Administration (FDA) extended its review timeline of the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
AI Summary
REGENXBIO Inc. said the U.S. Food and Drug Administration has extended its review of the Biologics License Application for clemidsogene lanparvovec (RGX-121), its one-time gene therapy for Hunter syndrome. The Prescription Drug User Fee Act goal date moved from November 9, 2025, to February 8, 2026.
The extension follows REGENXBIO’s submission of longer-term clinical results from all 13 patients in its pivotal study. These 12-month data align with previously shared biomarker and neurodevelopmental findings, supporting the treatment’s potential benefits.
REGENXBIO plans to share the updated pivotal data at the International Congress of Inborn Errors of Metabolism in September 2025. If approved, the company says commercial launch plans remain on track to bring the first therapy that targets the root genetic cause of Hunter syndrome to patients.
RGX-121 holds Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- May 14, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
NS Pharma, Inc announced that that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc.
AI Summary
NS Pharma, Inc. announced that the U.S. Food and Drug Administration has accepted for review the Biologics License Application submitted by REGENXBIO Inc. The application is for RGX-121, a potential first-in-class gene therapy designed to treat Mucopolysaccharidosis II (MPS II), a rare disease affecting the central nervous system. This milestone means that the FDA will now evaluate whether the therapy is safe and effective for patients suffering from this challenging condition.
The FDA has granted a Priority Review for the application, with a target action date set for November 9, 2025. NS Pharma is optimistic that this decision will accelerate the development of RGX-121, potentially offering a life-changing treatment option for individuals and families impacted by MPS II. The collaboration between NS Pharma and REGENXBIO showcases their commitment to bringing innovative therapies closer to patients in need.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- May 13, 2025
- Target Action Date:
- November 9, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025.
AI Summary
REGENXBIO Inc. announced an important regulatory milestone as the U.S. Food and Drug Administration granted a Priority Review for its Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121). The FDA set a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025. This decision speeds up the review process for RGX-121, a potential one-time gene therapy treatment for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
RGX-121 is being developed with the hope of addressing both the neurodevelopmental and systemic aspects of Hunter syndrome. REGENXBIO is optimistic that the positive biomarker data and long-term outcomes seen so far will lead to a treatment that could change the current approach for managing MPS II, which currently relies on regular enzyme replacement therapy. This FDA milestone is a key step toward bringing a promising new therapy to patients and their families.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- May 13, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
AI Summary
REGENXBIO Inc. announced that the U.S. Food and Drug Administration has accepted its Biologics License Application for clemidsogene lanparvovec (RGX-121) for accelerated approval to treat Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The FDA granted Priority Review with a target action date of November 9, 2025. This development is a major step forward for a one-time gene therapy designed to deliver the iduronate-2-sulfatase (IDS) gene directly into the central nervous system, potentially addressing both the neurodevelopmental and systemic challenges of the disorder.
REGENXBIO believes RGX-121 could offer a transformative treatment option for patients and families by reducing the ongoing burden associated with current therapies. The acceptance of this application highlights the company’s commitment to advancing innovative gene therapies aimed at improving treatment outcomes for those affected by Hunter syndrome.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- March 4, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc announced the closing of its previously announced strategic partnership with Nippon Shinyaku.
AI Summary
REGENXBIO Inc. has closed its strategic partnership with Nippon Shinyaku. The agreement will focus on the development and commercialization of two gene therapy candidates: RGX-121 for treating Mucopolysaccharidosis II (Hunter syndrome) and RGX-111 for Mucopolysaccharidosis I (Hurler syndrome) in the United States and Asia. These therapies aim to offer new treatment options to patients with these rare genetic disorders, providing hope for improved long-term outcomes.
Curran M. Simpson, President and CEO of REGENXBIO, expressed optimism about the partnership, stating that RGX-121 and RGX-111 could be transformative for patients in need. He emphasized that the collaboration with Nippon Shinyaku would strengthen the company’s progress on these programs and support its mission to bring innovative gene therapies to the MPS community.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- January 30, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 21st Annual WORLDSymposium™ 2025, taking place in San Diego, CA February 3-7, 2025.
AI Summary
REGENXBIO Inc. announced that data from its RGX-121 (clemidsogene lanparvovec) program for treating mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be presented at the 21st Annual WORLDSymposium™ 2025. The symposium is scheduled to take place in San Diego, CA from February 3 to 7, 2025. During the event, REGENXBIO will share key findings from their clinical studies, including an encore presentation of topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial. One of the presentations will focus on an audiology assessment of patients, while another will provide an update on the overall clinical study and progress of RGX-121. This data is expected to offer important insights into the potential of gene therapy for improving the treatment of MPS II and enhancing patient care.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- January 14, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced a strategic partnership for the development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome.
AI Summary
REGENXBIO Inc. and Nippon Shinyaku have formed a strategic partnership focused on developing and commercializing gene therapies for rare genetic disorders. The collaboration centers on RGX-121 for treating Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for treating Mucopolysaccharidosis I (MPS I), or Hurler syndrome. Under the agreement, REGENXBIO will receive $110 million upfront, with a potential $700 million in milestone payments, as well as meaningful double-digit royalties on net sales in the U.S. and Asia. REGENXBIO will lead manufacturing and clinical development, while Nippon Shinyaku will drive commercialization efforts in these regions. RGX-121 is positioned to become the first gene therapy for MPS II, with potential FDA approval as early as 2025, and RGX-111 has shown promising results in early trials. This partnership leverages both companies’ expertise to bring transformative treatments to patients with these rare conditions.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- September 3, 2024
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc announced positive results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome.
AI Summary
REGENXBIO Inc. announced positive results from its Phase I/II/III CAMPSIITE® trial of RGX-121, a gene therapy aimed at treating Mucopolysaccharidosis Type II (Hunter syndrome). The trial’s pivotal dose level demonstrated an 85% median reduction in cerebrospinal fluid levels of HS D2S6, a key biomarker linked to brain disease in MPS II. These reductions were maintained for up to two years, suggesting a lasting effect on the underlying neurological issues of the disorder.
In the study, 80% of patients who received the pivotal dose either discontinued standard intravenous enzyme replacement therapy or remained treatment-naïve, reflecting improved neurodevelopmental outcomes when compared to natural history data. The promising results support RGX-121 as a potential one-time treatment that could change the course of Hunter syndrome. A rolling Biologics License Application is expected to be filed in Q3 2024.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- August 27, 2024
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc announced data from its RGX-121 program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the SSIEM 2024 Annual Symposium, taking place in Porto, Portugal from September 3-6, 2024.
AI Summary
REGENXBIO Inc. has announced that it will present data from its RGX-121 program designed to treat mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, at the upcoming SSIEM 2024 Annual Symposium. This event is scheduled to be held in Porto, Portugal, from September 3-6, 2024. At the symposium, the interim clinical update, titled “CAMPSIITE™ phase I/II/III: Interim clinical update of RGX-121, an investigational gene therapy for treatment of neuronopathic MPS II (PO-205),” will be presented on Wednesday, September 4, at 6:15 p.m. WEST.
The data is expected to offer insight into the progress of RGX-121 as a potential one-time gene therapy aimed at addressing the underlying issues of this rare disease. Researchers and clinicians attending the symposium will have the opportunity to learn more about the therapeutic approach and the clinical outcomes observed so far.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- June 18, 2024
- Target Action Date:
- H2 2025
- Estimated Target Date Range:
- July 1, 2025 - December 31, 2025
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that Confirmatory trial expected to begin in H2 2025
AI Summary
REGENXBIO Inc. announced a significant upcoming milestone for its gene therapy, RGX-121, for treating Mucopolysaccharidosis Type II (Hunter syndrome). The company revealed that it plans to initiate a confirmatory trial in the second half of 2025. This trial is designed to verify the predicted clinical benefit by evaluating key biomarker, neurocognitive, and systemic data. The study will support the ongoing efforts toward accelerated approval through the submission of a rolling Biologics License Application, expected to begin in the third quarter of 2024. REGENXBIO is confident in the comparability of its commercial bulk drug to the clinical trial material produced via its proprietary NAVXpress™ platform. This trial marks an important step as the company moves closer to establishing a new treatment option for patients suffering from this rare pediatric disease.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- June 18, 2024
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc announced it completed a successful Pre-Biologics License Application (BLA) meeting for RGX-121 for the treatment Mucopolysaccharidosis Type II (MPS II), where it finalized details of its BLA with the U.S. Food and Drug Administration (FDA).
AI Summary
REGENXBIO Inc. announced a successful Pre-BLA (Biologics License Application) meeting with the FDA for its gene therapy candidate, RGX-121, which is aimed at treating Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. During the meeting, the company and the FDA finalized key details for the upcoming rolling BLA submission that is planned to begin in the third quarter of 2024. The discussion focused on utilizing cerebrospinal fluid levels of the biomarker heparan sulfate D2S6 as a surrogate endpoint, supporting the accelerated approval pathway. FDA officials also confirmed that the commercial bulk drug is comparable to the clinical trial material. The meeting addressed important elements such as manufacturing and non-clinical aspects, which set a solid foundation for advancing the confirmatory trial expected to start in the second half of 2025.
Read Announcement- Drug:
- RGX-121
- Announced Date:
- June 18, 2024
- Indication:
- MPS II (Hunter Syndrome)
Announcement
REGENXBIO Inc. announced that Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024
AI Summary
REGENXBIO Inc. announced plans to begin submitting a rolling Biologics License Application (BLA) for its gene therapy RGX-121 in the third quarter of 2024 using the FDA’s accelerated approval pathway. This submission will include positive data on key biomarkers, neurocognitive outcomes, and systemic effects that support its potential clinical benefits in treating Hunter syndrome.
The FDA and REGENXBIO are aligned on the core elements of the BLA, including the use of cerebrospinal fluid heparan sulfate as a surrogate endpoint. In addition, both the commercial bulk drug and clinical trial material manufactured via REGENXBIO’s NAVXpress™ platform have been confirmed to be comparable. Following the BLA submission, an FDA inspection of the manufacturing facility is expected in the first half of 2025, with a confirmatory clinical trial set to begin enrollment in the latter half of 2025.
Read Announcement
ABBV-RGX-314 FDA Regulatory Events
ABBV-RGX-314 is a drug developed by REGENXBIO for the following indication: In patients with 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:
- ABBV-RGX-314
- Announced Date:
- October 9, 2025
- Indication:
- In patients with wet AMD
Announcement
REGENXBIO Inc. announced that it will present interim data from the Phase II ALTITUDE® trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the American Academy of Ophthalmology 2025 Annual Meeting.
AI Summary
REGENXBIO Inc. will share interim results from its Phase II ALTITUDE® trial at the American Academy of Ophthalmology 2025 Annual Meeting. These data look at suprachoroidal delivery of surabgene lomparvovec for people with diabetic retinopathy.
Surabgene lomparvovec, also called ABBV-RGX-314 or sura-vec, is a one-time gene therapy made with AbbVie. The treatment uses an AAV8 vector to block vascular endothelial growth factor, which causes leaky blood vessels and fluid buildup in the retina.
Highlights 2-year results in non-proliferative diabetic retinopathy. Dr. Charles C. Wykoff from Retina Consultants of Texas will present on October 17 at 4:46 p.m. ET during the “Late Breaking Developments, Part I” session.
This study could lead to a first-in-class treatment for diabetic retinopathy. The gene therapy is under investigation and not yet approved. REGENXBIO aims to offer patients a long-lasting option with one dose.
Read Announcement- Drug:
- ABBV-RGX-314
- Announced Date:
- January 13, 2025
- Indication:
- In patients with wet AMD
Announcement
AbbVie and REGENXBIO Inc. announced updates to the ABBV-RGX-314 clinical program.
AI Summary
AbbVie and REGENXBIO Inc. have updated the clinical program for their investigational gene therapy, ABBV-RGX-314. This program is exploring new treatment methods for two serious retinal diseases: wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR). For wet AMD, pivotal trial data on the safety and efficacy of the subretinal delivery of ABBV-RGX-314 are expected in 2026. This innovative approach could potentially reduce the need for frequent injections that many patients currently depend on.
For diabetic retinopathy, the companies plan a Phase 3 clinical program that will use the SCS Microinjector® for suprachoroidal delivery. This method aims to ease treatment burdens by providing a one-time therapy option. Both companies are optimistic that these advancements could lead to significant improvements in managing these vision-threatening conditions.
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Surabgene lomparvovec FDA Regulatory Events
Surabgene lomparvovec is a drug developed by REGENXBIO for the following indication: for 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:
- surabgene lomparvovec
- Announced Date:
- October 6, 2025
- Target Action Date:
- Q4 2026
- Estimated Target Date Range:
- October 1, 2026 - December 31, 2026
- Indication:
- for Wet AMD
Announcement
REGENXBIO Inc. announced that Topline pivotal data expected in Q4 2026
AI Summary
REGENXBIO Inc. completed enrollment in its ATMOSPHERE® and ASCENT® pivotal trials, marking the largest global gene therapy program ever. Over 1,200 participants are taking part to evaluate surabgene lomparvovec for wet age-related macular degeneration.
ATMOSPHERE, conducted in the U.S., compares sura-vec against ranibizumab, while ASCENT tests it versus aflibercept in the U.S. and 13 other countries. Both track vision changes, retinal thickness, safety, and injection needs after one year.
Earlier studies showed surabgene lomparvovec was well tolerated, with stable or improved vision and fewer injections for up to four years. These encouraging results support hopes that a one-time gene therapy could ease the burden of frequent eye treatments.
REGENXBIO expects topline pivotal data in the fourth quarter of 2026. If positive, these results may lead to global regulatory filings and make surabgene lomparvovec the first gene therapy for wet AMD, offering a lasting alternative to repeat injections.
Read Announcement- Drug:
- surabgene lomparvovec
- Announced Date:
- October 6, 2025
- Indication:
- for Wet AMD
Announcement
REGENXBIO Inc. nnounced the completion of enrollment in the ATMOSPHERE® and ASCENT® pivotal studies evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration (wet AMD) using subretinal delivery.
AI Summary
REGENXBIO Inc. announced completion of enrollment of over 1,200 participants in its two pivotal ATMOSPHERE® and ASCENT® trials of surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration. This represents the largest global gene therapy program to date.
ATMOSPHERE® in the U.S. compares subretinal sura-vec to ranibizumab. ASCENT® in the U.S. and 13 countries compares it to aflibercept. Both are randomized, active-controlled trials that aim to show non-inferior gains in best-corrected visual acuity at 54 weeks (ATMOSPHERE®) and one year (ASCENT®). Secondary measures include safety, retinal thickness changes, and the need for anti-VEGF injections. Topline data are expected in the fourth quarter of 2026.
Earlier trials found subretinal sura-vec was well tolerated, durable for four years, and reduced injection burden. If approved, it could become the first one-time gene therapy for wet AMD, offering patients an alternative to frequent eye injections.
Read Announcement
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