On 22 June 2023, Sarepta Therapeutics announced that it had received marketing authorisation from the FDA for Elevidys (delandistrogene moxeparvovec-rokl or SRP-9001), the company’s microdystrophin gene therapy product for Duchenne muscular dystrophy, under the accelerated approval process.
- This authorisation applies to boys with DMD, aged between 4 and 5 years, who are walking, excluding those carrying deletions in exons 8 and/or 9 of the dystrophin gene (DMD). It does not concern Europe.
- The FDA’s decision to grant accelerated approval is based on the actual production of dystrophin in the muscles of treated patients, measured 3 months after administration of the product in a phase 2 trial.
- However, it remains conditional on Sarepta demonstrating clinical efficacy: the confirmatory phase 3 trial currently underway, EMBARK, should provide proof of this (results expected by 2024).
- Adverse reactions have been observed in patients: severe acute liver disorders, immune myositis, myocarditis, nausea, vomiting, increased liver enzymes, fever, reduced platelet count, etc.
Sarepta Therapeutics, press release, 22 June 2023 – Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy
