This section highlights FDA-related milestones and regulatory updates for drugs developed by Sarepta Therapeutics (SRPT).
Over the past two years, Sarepta Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ELEVIDYS, SRP-9001-101, SRP-9001-301, and SRP-9003. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ELEVIDYS (delandistrogene moxeparvovec-rokl) - FDA Regulatory Timeline and Events
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a drug developed by Sarepta Therapeutics for the following indication: Designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
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:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- June 15, 2025
- Indication:
- Designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Announcement
Sarepta Therapeutics, Inc. today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients.
AI Summary
Sarepta Therapeutics today provided a safety update on ELEVIDYS, the only approved gene therapy for treating Duchenne muscular dystrophy, focusing on improving safety for non-ambulatory patients. In response to a second fatal case of acute liver failure in this group, the company is developing an enhanced immunosuppressive regimen that includes sirolimus. This enhanced protocol is being designed in consultation with a panel of clinical experts and regulators to better manage liver enzyme elevations and reduce risks.
As a result, shipments of ELEVIDYS for non-ambulatory patients have been temporarily suspended until the new regimen is approved. Additionally, the ENVISION clinical study has been paused to allow for a protocol amendment incorporating extra immunosuppression. Sarepta emphasizes that for ambulatory patients, current treatments remain unchanged while safety improvements for non-ambulatory patients are pursued under FDA guidance.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- May 21, 2025
- Indication:
- Designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Announcement
Sarepta Therapeutics, Inc. shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy.
AI Summary
Sarepta Therapeutics, Inc. recently provided an important update on ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy (DMD). The update noted that the Medicines & Healthcare products Regulatory Agency (MHRA) in the United Kingdom advised that dosing in the ongoing global ENVISION study can proceed without interruption. ENVISION is a Phase 3, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in both non-ambulatory and older ambulatory individuals with DMD.
ELEVIDYS is a single-dose, AAV-based gene transfer therapy that targets the underlying genetic defect in DMD by delivering a transgene to produce micro-dystrophin in skeletal muscle. In the United States, it is approved for DMD patients aged 4 years and older, with specific indications for both ambulatory and non-ambulatory patients, marking a significant advancement in precision genetic medicine for rare diseases.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- May 16, 2025
- Indication:
- Designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Announcement
Sarepta Therapeutics, Inc today presented new data from Part 2 of the EMBARK study that continue to support the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy.
AI Summary
Sarepta Therapeutics recently presented encouraging data from Part 2 of the EMBARK study, which supports the clinical benefits of ELEVIDYS (delandistrogene moxeparvovec-rokl) for Duchenne muscular dystrophy. In one cohort of young patients treated at age 2, the therapy achieved an average protein expression of 93.87% as measured by western blot, along with 79.9% dystrophin positive fibers. These results were confirmed from biopsies taken 12 weeks after treatment.
The safety profile was consistent with previous ELEVIDYS studies and real-world experiences. Reported side effects included nausea and vomiting, while elevated liver enzymes in a couple of patients were successfully managed with steroid treatment. Such encouraging biomarker outcomes in younger patients may support Sarepta’s plans to seek FDA discussions on expanding ELEVIDYS’ labeled use to include even younger children with Duchenne muscular dystrophy.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- June 20, 2024
- Indication:
- Designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
Announcement
Sarepta Therapeutics, Inc. announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.
AI Summary
Sarepta Therapeutics, Inc. announced that the FDA has expanded the indicated use of ELEVIDYS (delandistrogene moxeparvovec-rokl) for individuals with Duchenne muscular dystrophy (DMD) who are at least 4 years old and have a confirmed DMD gene mutation. This approval now includes both ambulatory and non-ambulatory patients. The FDA granted traditional approval for ambulatory patients based on demonstrated functional benefits, while non-ambulatory patients received accelerated approval. For these non-ambulatory patients, continued approval may depend on future clinical trials that verify the drug’s clinical benefits. This expansion is an important milestone for those living with DMD, offering a new gene therapy option to treat the underlying cause of the disease and potentially help more patients maintain muscle function and improve their quality of life.
Read Announcement
SRP-9001-101 - FDA Regulatory Timeline and Events
SRP-9001-101 is a drug developed by Sarepta Therapeutics 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:
- SRP-9001-101
- Announced Date:
- September 26, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Sarepta Therapeutics, Inc. the leader in precision genetic medicine for rare diseases, will present at the 29th Annual Congress of the World Muscle Society 2024 Congress (WMS 2024), taking place Oct. 8-12, 2024, in Prague, Czechia.
AI Summary
Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, will present at the 29th Annual Congress of the World Muscle Society (WMS 2024) in Prague, Czechia, from October 8-12, 2024. At this well-known event, Sarepta will share new safety and efficacy data from several studies related to its delandistrogene moxeparvovec clinical development program. The presentations will include early insights from the EMBARK study, offering the first look at skeletal muscle and cardiac MRI outcomes, as well as a detailed safety analysis across multiple trials. Additionally, almost five-year functional data from Study SRP-9001-101 will be discussed, marking the longest-term gene therapy data in Duchenne muscular dystrophy to date. These findings underscore Sarepta’s commitment to advancing gene therapy research and their continued leadership in developing treatments for muscular dystrophies.
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SRP-9001-301 - FDA Regulatory Timeline and Events
SRP-9001-301 is a drug developed by Sarepta Therapeutics for the following indication: In Ambulatory Individuals with 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:
- SRP-9001-301
- Announced Date:
- January 27, 2025
- Indication:
- In Ambulatory Individuals with Duchenne Muscular Dystrophy
Announcement
Sarepta Therapeutics, Inc announced positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy.
AI Summary
Sarepta Therapeutics announced positive results from Part 2 of its Phase 3 EMBARK study for ELEVIDYS, the only gene therapy approved for patients with Duchenne muscular dystrophy. In this well-controlled, international study, patients who switched from placebo to ELEVIDYS showed significant improvement in motor function as measured by the North Star Ambulatory Assessment, Time to Rise, and 10-meter walk/run tests. Despite these patients being older on average than those in Part 1, the benefits were clear when compared to a matched external control group. The study also reinforced earlier findings from Part 1, where treated patients maintained muscle function improvements and showed minimal progression of muscle damage on MRI scans at the two-year mark. Additionally, the safety profile of ELEVIDYS remained consistent, offering continued hope in managing the disease's progression.
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SRP-9003 - FDA Regulatory Timeline and Events
SRP-9003 is a drug developed by Sarepta Therapeutics for the following indication: For the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4.
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:
- SRP-9003
- Announced Date:
- June 4, 2025
- Indication:
- For the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4
Announcement
Sarepta Therapeutics, Inc. announced that the rAAVrh74 viral vector used in the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy type 2E/R4, has been granted platform technology designation by the U.S. Food & Drug Administration.
AI Summary
Sarepta Therapeutics recently announced that the rAAVrh74 viral vector used in their investigational gene therapy SRP-9003 has been granted platform technology designation by the U.S. Food & Drug Administration. This designation recognizes the vector’s reproducibility and adaptability, qualities that make it a promising tool for developing multiple therapeutic programs. According to Dr. Louise Rodino-Klapac, this milestone is significant because it is one of the first gene therapy programs to receive such recognition, and it highlights the consistency of data seen across different clinical trials.
The platform designation is designed to streamline the research, manufacturing, and regulatory review processes for future drug applications. By leveraging previously generated data, sponsors may use the platform to support new investigational treatments, potentially accelerating the development of transformative therapies for patients with limb-girdle muscular dystrophy type 2E/R4.
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