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
ABBV-RGX-314, RGX-121, and RGX-202. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ABBV-RGX-314 - FDA Regulatory Timeline and 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:
- 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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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:
- 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
- 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:
- May 13, 2025
- Estimated Event Date Range:
- November 9, 2025 - November 9, 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:
- 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
- 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- Drug:
- RGX-121
- Announced Date:
- June 18, 2024
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- 2025-H2
- 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.
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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:
- 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 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:
- 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:
- 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- Drug:
- RGX-202
- Announced Date:
- June 24, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 - 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
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