What restrictions now apply to Elevidys?
As of September 2026, Elevidys is approved only for ambulatory patients aged 4+ with confirmed DMD mutations (excluding exon 8/9 deletions), carries a boxed liver-failure warning, and is not authorised in Europe.
On July 28, 2025 the FDA recommended Sarepta resume shipments for ambulatory patients, while non-ambulatory patients remained on hold. A November 14, 2025 label update added a boxed warning for acute serious liver injury and acute liver failure and removed eligibility for non-ambulatory patients entirely. As of September 2026, Elevidys is approved only for ambulatory patients aged 4 and older with a confirmed DMD mutation, excluding those with exon 8 or 9 deletions. The European Medicines Agency refused EU authorisation. In practice, treatment centres must now monitor patients closely for liver injury after infusion. Sarepta had also been working on an enhanced immunosuppressive regimen including sirolimus aimed at making the therapy safer for non-ambulatory patients.