Elevidys, the first microdystrophin gene therapy authorised for DMD, subject to conditions

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