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, AMONDYS, SRP-1001, SRP-1005-101, SRP-9001-301, and SRP-9003. 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.
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 under review 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:
- September 30, 2026
- 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 at the 31st Annual Congress of the World Muscle Society (WMS) that provide evidence of clinically meaningful functional benefit of ELEVIDYS (delandistrogene moxeparvovec) in older ambulatory patients with Duchenne muscular dystrophy.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- March 16, 2026
- 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 screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103).
AI Summary
Sarepta Therapeutics announced that screening and enrollment are underway for Cohort 8 of the ENDEAVOR study (Study 9001-103). Approximately 25 non-ambulatory participants will receive sirolimus as part of an enhanced immunosuppressive regimen. The regimen is intended to reduce the risk of acute liver injury (ALI) and acute liver failure (ALF) that can be associated with AAV gene therapy in non-ambulatory patients, by more strongly controlling immune responses around treatment.
ENDEAVOR is an open-label Phase 1b study evaluating the expression and safety of ELEVIDYS (delandistrogene moxeparvovec) in multiple cohorts of males with Duchenne muscular dystrophy. The study has enrolled participants across seven cohorts and has included both ambulatory and non-ambulatory individuals. The primary endpoint is change from baseline in micro‑dystrophin protein measured by western blot at 12 weeks, with secondary measures including percent dystrophin-positive fibers at 12 weeks.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- January 23, 2026
- Target Action Date:
- January 26, 2026
- 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 that on Monday, Jan. 26, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present 3-year topline functional results from patients treated in Part 1 of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment.
AI Summary
Sarepta Therapeutics announced it will host a webcast and conference call on Monday, Jan. 26, 2026 at 8:30 a.m. Eastern to present 3‑year topline functional results from patients in Part 1 of EMBARK (Study 9001‑301). The company will share outcomes from this part of the trial and discuss what the results mean for patients and research.
EMBARK is a global, randomized, placebo‑controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec‑rokl) in ambulatory individuals with Duchenne muscular dystrophy who were four to seven years old at the time they received treatment. The Part 1 data reflect three years of functional follow‑up in treated patients.
The live webcast will be available on Sarepta’s investor relations site: https://investorrelations.sarepta.com/events-presentations. A replay will be archived there for one year.
Phone participants must register via the online form; registrants receive an email with the dial‑in number and a personal PIN to access the call.Read Announcement
- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- November 14, 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. announced an update to the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD).
AI Summary
Sarepta Therapeutics updated the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD). Key label changes include a boxed warning about the risk of acute serious liver injury (ALI) and acute liver failure (ALF), and removal of the non‑ambulatory indication. Sarepta plans a study of an enhanced sirolimus immunosuppressive regimen to address ALI/ALF with the goal of resuming dosing for non‑ambulatory patients.
The label also adds expanded prescriber guidance: a modified oral corticosteroid regimen before and after infusion, and enhanced monitoring with weekly liver checks for three months post‑infusion. A new warning notes increased susceptibility to serious infections from immunosuppression. ELEVIDYS is a single‑dose AAV‑based IV gene therapy indicated for ambulatory patients 4 years and older with a confirmed DMD gene mutation and is not recommended for patients with significant liver impairment, recent vaccination, or active/recent infections.
More than 1,100 patients have received ELEVIDYS in clinical and real‑world settings. The updated label stresses close monitoring, proximity to medical care after infusion, and specialist consultation if liver injury is suspected.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- October 3, 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 will present at the 30th Annual Congress of the World Muscle Society (WMS) 2025 Congress, taking place Oct. 7-11, 2025, in Vienna, Austria.
AI Summary
Sarepta Therapeutics will take part in the 30th Annual Congress of the World Muscle Society, held October 7–11, 2025, in Vienna, Austria. The company will share new data on gene therapy and exon-skipping approaches for neuromuscular diseases, underlining its leadership in precision genetic medicine.
Presentations include results from the delandistrogene moxeparvovec clinical program, showcasing micro-dystrophin expression and safety in young boys with Duchenne muscular dystrophy. A real-world evidence study on pulmonary function in advanced-stage patients treated with casimersen will also be featured. Additionally, Sarepta will report on the EMERGENE phase 3 trial of bidridistrogene xeboparvovec in limb-girdle muscular dystrophy type 2E and an investigator-initiated abstract on using sirolimus prophylaxis to reduce potential liver injury in gene therapy recipients.
These findings aim to deepen understanding of safety, efficacy and long-term benefits, with the goal of advancing patient care and treatment options for those living with rare neuromuscular conditions.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- July 28, 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 announced that the U.S. Food and Drug Administration (FDA) notified Sarepta that it may lift its voluntary pause on shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory patients with Duchenne.
AI Summary
Sarepta Therapeutics announced that the U.S. Food and Drug Administration has notified the company it may lift its voluntary pause on shipments of ELEVIDYS for ambulatory patients with Duchenne muscular dystrophy. Sarepta commended the FDA for its rapid and comprehensive review of available safety data, including the case of an 8-year-old patient in Brazil whose death was deemed unrelated to the therapy.
Shipments of ELEVIDYS will resume imminently to treatment sites across the United States. Sarepta emphasized its shared commitment with the FDA to ensure patients receive timely access to this gene therapy, which addresses the underlying genetic cause of Duchenne.
Looking ahead, Sarepta and the FDA will continue discussions on updating ELEVIDYS’s safety labeling and defining a risk-mitigation strategy for non-ambulatory patients. The company remains focused on advancing patient care and working closely with regulators throughout this process.
Read Announcement- Drug:
- ELEVIDYS (delandistrogene moxeparvovec-rokl)
- Announced Date:
- July 25, 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 the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Sarepta Therapeutics, Inc., a leader in precision genetic medicine for rare diseases, announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory children ages three to seven with Duchenne muscular dystrophy (DMD). The CHMP highlighted concerns about safety data, trial design, and the durability of benefit under the proposed dosing regimen.
Sarepta will review the feedback, supply additional clinical and long-term data, and address questions on safety and efficacy. The company is open to modifying trial protocols and adding follow-up measures to meet regulators’ standards.
It plans scientific advice meetings, post-authorization studies, and new pathways to bring ELEVIDYS to patients. Sarepta says it remains dedicated to the DMD community and transparent with regulators.
Read Announcement- 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
AMONDYS 45 FDA Regulatory Events
AMONDYS 45 is a drug developed by Sarepta Therapeutics for the following indication: treatment of Duchenne muscular dystrophy (DMD).
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:
- AMONDYS 45
- Announced Date:
- June 30, 2026
- Indication:
- treatment of Duchenne muscular dystrophy (DMD).
Announcement
Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Sarepta Therapeutics said the U.S. Food and Drug Administration has accepted for filing its supplemental New Drug Applications for AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen), both used to treat Duchenne muscular dystrophy. The company is seeking to move both drugs from accelerated approval to traditional approval.
The FDA has agreed to review the applications and set a target action date of February 28, 2027. Sarepta said the requests are supported by results from the ESSENCE confirmatory study, along with published real-world evidence and the drugs’ consistent safety profiles. The filings reflect the company’s effort to show that both exon-skipping therapies have enough evidence for full approval in patients with DMD.
Read Announcement- Drug:
- AMONDYS 45
- Announced Date:
- March 19, 2026
- Indication:
- treatment of Duchenne muscular dystrophy (DMD).
Announcement
Sarepta Therapeutics, Inc. provided an update on its ongoing regulatory interactions with the U.S. Food and Drug Administration (FDA) regarding AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Sarepta Therapeutics said it has been working with the U.S. Food and Drug Administration and, following FDA feedback, intends to submit supplemental new drug applications by the end of April 2026 asking to convert AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) from their current status to traditional approval. The company framed this step as part of ongoing regulatory interactions with FDA.
An updated analysis of the ESSENCE study—excluding data from 23 participants whose baseline 4‑step ascend velocity occurred during the COVID‑19 impact period—showed a least‑squares mean difference of 0.12 steps/second at week 96 (P=0.050). Sarepta reported no new safety signals in ESSENCE; most adverse events were mild (88%) or moderate (10.9%) and were comparable between treatment and placebo groups.
Both AMONDYS 45 and VYONDYS 53 are described by the company as having met full statutory standards for safety and effectiveness and are not considered investigational or experimental.Read Announcement
SRP-1001 FDA Regulatory Events
SRP-1001 is a drug developed by Sarepta Therapeutics for the following indication: Targeting FSHD1 and DM1.
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-1001
- Announced Date:
- March 25, 2026
- Indication:
- Targeting FSHD1 and DM1
Announcement
Sarepta Therapeutics, Inc today shared the first clinical results from two of its siRNA programs for neuromuscular diseases.
AI Summary
Sarepta Therapeutics announced it has shared the first clinical results from two early-stage siRNA programs aimed at neuromuscular diseases. SRP-1001 is an investigational siRNA designed to reduce DUX4 protein in skeletal muscle for patients with FSHD1. Its Study 1001-101 is a combined Phase 1/2, single- and multiple-ascending dose (SAD/MAD), randomized, placebo-controlled trial enrolling participants aged 16 to 70. SRP-1003 is an investigational siRNA for myotonic dystrophy type 1 (DM1) that targets and silences DMPK mRNA. Study SRP-1003-101 is a first-in-human Phase 1/2 SAD/MAD, randomized, placebo-controlled trial in adults aged 18 to 65.
Sarepta says these results support continued development of its next-generation siRNA platform, which focuses on chronically administered therapies for neurodegenerative and pulmonary diseases. The company is also advancing related preclinical programs for SCA1 and SCA3 under an exclusive license with Arrowhead Pharmaceuticals.
Read Announcement- Drug:
- SRP-1001
- Announced Date:
- March 24, 2026
- Indication:
- Targeting FSHD1 and DM1
Announcement
Sarepta Therapeutics, Inc. announced that on Wed., March 25, 2026, at 8:30 am Eastern Time, the Company will host a webcast and conference call to present the early clinical results from the Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1).
AI Summary
Sarepta Therapeutics, Inc. announced it will host a webcast and conference call on Wednesday, March 25, 2026, at 8:30 a.m. Eastern Time to present early clinical results. The company will review data from its Phase 1/2 ascending‑dose studies of two investigational therapies: SRP‑1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP‑1003 for myotonic dystrophy type 1 (DM1).
The presentation will cover initial findings across the ascending‑dose cohorts, including topline safety, tolerability, and early signs of activity. Sarepta intends to discuss how these early results will guide dose escalation and next steps in development for both programs. The webcast and call are aimed at clinicians, researchers, patients, and investors seeking an early look at the performance of SRP‑1001 and SRP‑1003. Details for accessing the live webcast and conference call were provided by the company.
Read Announcement
SRP-1005-101 FDA Regulatory Events
SRP-1005-101 is a drug developed by Sarepta Therapeutics for the following indication: Treatment for Huntington'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:
- SRP-1005-101
- Announced Date:
- February 4, 2026
- Indication:
- Treatment for Huntington's Disease
Announcement
Sarepta Therapeutics, Inc. announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted approval for its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT.
AI Summary
Sarepta Therapeutics announced that Medsafe, New Zealand’s Medicines and Medical Devices Safety Authority, has approved the clinical trial application (CTA) for Study SRP-1005-101, called INSIGHTT. This approval clears the way to begin the first-in-human trial of SRP-1005 in New Zealand. Sarepta expects the INSIGHTT study to start in the second quarter of 2026. The CTA approval is an important regulatory step that lets the company move from preclinical work into testing the drug in people.
SRP-1005 is part of Sarepta’s next-generation siRNA platform, which is aimed at developing chronically administered treatments for neurodegenerative and pulmonary diseases. The platform includes programs for facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy type 1 (DM1), spinocerebellar ataxia type 2 (SCA2), idiopathic pulmonary fibrosis (IPF), and Huntington’s disease (HD). Sarepta is also advancing preclinical work in SCA1 and SCA3 and has an exclusive collaboration with Arrowhead Pharmaceuticals on muscle and CNS discovery targets.
Read Announcement- Drug:
- SRP-1005-101
- Announced Date:
- January 7, 2026
- Target Action Date:
- Q2 2026
- Estimated Target Date Range:
- April 1, 2026 - June 30, 2026
- Indication:
- Treatment for Huntington's Disease
Announcement
Sarepta Therapeutics, Inc announced that Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026.
AI Summary
Sarepta announced it has submitted a clinical trial application (CTA) to Medsafe in New Zealand for Study SRP-1005-101, called INSIGHTT. Pending regulatory approval, the company expects to start the first-in-human trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapy aimed at treating Huntington’s disease.
INSIGHTT is a Phase 1, multi-center, dose-escalation study designed to evaluate safety and tolerability of subcutaneous dosing in about 24 participants. Subcutaneous delivery is intended to keep drug levels below transferrin saturation while allowing steady penetration across the blood–brain barrier.
SRP-1005 uses an advanced transferrin receptor 1 (TfR1) approach with a monovalent fragment antigen binding (fAb) to improve delivery to the central nervous system. Preclinical data show notable huntingtin protein knockdown in deep brain regions like the putamen and caudate and in cortical areas, supporting further clinical testing.
Read Announcement- Drug:
- SRP-1005-101
- Announced Date:
- January 7, 2026
- Indication:
- Treatment for Huntington's Disease
Announcement
Sarepta Therapeutics, Inc. announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority.
AI Summary
Sarepta Therapeutics announced it submitted a clinical trial application (CTA) for Study SRP‑1005‑101, called INSIGHTT, to Medsafe, New Zealand’s medicines and medical devices safety authority. Pending approval, the company expects to begin the first‑in‑human trial in the second quarter of 2026. SRP‑1005 is an investigational small interfering RNA (siRNA) therapy aimed at lowering the mutant huntingtin protein that causes Huntington’s disease.
INSIGHTT is a Phase 1, multi‑center, dose‑escalation study that will evaluate safety and tolerability of subcutaneous dosing in about 24 participants. The drug uses an advanced transferrin receptor 1 (TfR1) approach with a monovalent fragment antigen binding (fAb) to improve delivery into the central nervous system. Subcutaneous delivery is intended to allow steady brain penetration while avoiding transferrin saturation.
Preclinical data showed substantial protein knockdown in deep brain regions such as the putamen, caudate, and parts of the temporal and frontal cortex. Timelines and outcomes depend on regulatory approval and clinical results.
Read Announcement
SRP-9001-301 FDA Regulatory 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 26, 2026
- Indication:
- In Ambulatory Individuals with Duchenne Muscular Dystrophy
Announcement
Sarepta Therapeutics, Inc announced positive topline three-year functional results from Part 1-treated patients in EMBARK (Study SRP-9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment and at time of last assessment were on average over nine years of age.
AI Summary
Sarepta Therapeutics reported positive three‑year topline results from Part 1 of EMBARK, a global, randomized, placebo‑controlled Phase 3 study of ELEVIDYS in ambulatory children with Duchenne muscular dystrophy who were 4–7 years old at treatment and on average over 9 years old at last assessment. Fifty‑two treated patients were followed for three years and compared to a propensity‑weighted external control group.
At a mean age of about 9, ELEVIDYS‑treated patients maintained mean NSAA scores above baseline at Year 3 while the external control group declined. ELEVIDYS slowed disease progression by about 73% on Time to Rise and 70% on the 10‑meter walk/run versus controls. Reported least squares mean differences were NSAA +4.39 points (p=0.0002), TTR −6.05 seconds (p<0.0001), and 10MWR −2.70 seconds (p=0.0039). The treatment effect increased between Year 2 and Year 3.
No new treatment‑related safety signals were observed, and the safety profile remained consistent and manageable; further analyses are ongoing.
Read Announcement- 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.
Read Announcement
SRP-9003 FDA Regulatory 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.
Read Announcement
