EMA halts Sarepta’s DMD gene therapy trial after patient death

3 April 2025

The European Medicines Agency has temporarily suspended enrollment and dosing in a Phase II trial of Sarepta Therapeutics' (Nasdaq: SRPT) gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), for Duchenne muscular dystrophy (DMD).

The decision follows the death of a 16-year-old patient from acute liver failure after receiving the treatment in an unrelated study. Shares in the company ticked down around 4% before trading on Thursday.

Sarepta, a biotech specialist based in the thriving life science hub around Cambridge, Massachusetts, has been working on the Phase II trial together with Swiss partner Roche (SIX: ROG), which holds rights to Elevidys outside the USA.

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