Sarepta Therapeutics
Sarepta Therapeutics works on 7 rare diseases tracked on Trial Friend, including Becker Muscular Dystrophy, Duchenne Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy and 4 more, with 10 active clinical trials (3 recruiting) and 4 FDA-approved rare disease drugs.
Sarepta Therapeutics is a Cambridge, Massachusetts biotech that has spent most of the last decade trying to deliver gene therapy to patients with Duchenne muscular dystrophy. The company was originally founded in 1980 and trades on the NASDAQ under the ticker SRPT. Today its identity is built around the modern Sarepta of the 2010s onward, a company that has bet repeatedly that genetic medicine for neuromuscular diseases is worth pursuing even when the path through the FDA is hard.
The flagship product is Elevidys (delandistrogene moxeparvovec), the first gene therapy approved for Duchenne muscular dystrophy. Duchenne is a rare, progressive genetic disease that primarily affects boys, caused by mutations on the X chromosome. Patients are missing functional dystrophin, a protein their muscles need to repair themselves, so muscle tissue is gradually replaced by scar and fat over time. Most patients lose the ability to walk before adolescence and face premature death from cardiac and respiratory complications. Elevidys delivers a shortened working version of the dystrophin gene to muscle cells using a viral vector called AAV. The drug received FDA accelerated approval in 2023 for ambulatory boys aged 4 to 5, and the indication was expanded in 2024.
The Elevidys story has had a difficult more recent arc. In 2025, two non-ambulatory pediatric patients died from acute liver failure following Elevidys treatment. The FDA responded by approving a Boxed Warning on the drug's label and revising the approved indication to limit use to ambulatory patients aged 4 and older. Sarepta is now conducting a postmarketing observational safety study of approximately 200 patients to better characterize risk in the real-world treated population. The events have made Elevidys a difficult and personal decision for many families weighing risk against the natural course of the disease, and Sarepta has been in active dialogue with the FDA and the DMD patient community since.
Sarepta also markets three earlier antisense oligonucleotide therapies for DMD that work by skipping over specific mutated exons of the dystrophin gene so a partially functional protein can still be produced. These are Exondys 51, Vyondys 53, and Amondys 45, each designed for a different DMD mutation type. The pipeline beyond Elevidys includes multiple gene therapy programs for limb-girdle muscular dystrophy subtypes, and next-generation siRNA therapeutics aimed at facioscapulohumeral dystrophy and myotonic dystrophy.
Sarepta Therapeutics Drug Pipeline
Sarepta Therapeutics has 10 active clinical trials across 5 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Sarepta Therapeutics Clinical Trials (10)
Active and recruiting clinical trials sponsored by Sarepta Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Sarepta Therapeutics FDA-Approved Drugs (4)
Medications developed or marketed by Sarepta Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| CASIMERSEN Antisense Oligonucleotide [EPC] | AMONDYS 45 intravenous | Feb 25, 2021 | |
| ETEPLIRSEN Antisense Oligonucleotide [EPC] | Exondys 51 intravenous | Sep 19, 2016 | |
| GOLODIRSEN Antisense Oligonucleotide [EPC] | Vyondys 53 intravenous | Dec 12, 2019 | |
| DELANDISTROGENE MOXEPARVOVEC-ROKL | ELEVIDYS intravenous | — |
Sarepta Therapeutics Trial Locations
Sarepta Therapeutics clinical trials are running at 168 sites in 34 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (7)
Diseases targeted by Sarepta Therapeutics's clinical trial and drug development programs
Becker Muscular Dystrophy (BMD) is an X-linked inherited muscular dystrophy caused by mutations in the dystrophin gene that produce a partially functional dystrophin protein. BMD is milder than Duchen...
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscl...
Facioscapulohumeral Muscular Dystrophy (FSHD) is an inherited muscular dystrophy characterized by progressive weakness of the facial, shoulder, and upper arm muscles. The condition results from abnorm...
Huntington disease is an autosomal dominant neurodegenerative disorder caused by an expanded CAG trinucleotide repeat in the huntingtin gene. The progressive disease causes movement problems, cognitiv...
Limb-Girdle Muscular Dystrophy (LGMD) refers to a genetically heterogeneous group of muscular dystrophies characterized by progressive weakness of the hip and shoulder girdle muscles. Multiple genetic...
Myotonic Dystrophy (MD) is the most common muscular dystrophy in adults, characterized by progressive muscle weakness and myotonia—the inability of muscles to relax after contraction. The condition re...
Patient Resources
Organizations and resources related to Sarepta Therapeutics's rare disease focus areas
Frequently Asked Questions About Sarepta Therapeutics
Common questions about Sarepta Therapeutics's rare disease programs, clinical trials, and treatments.