Sarepta says patient dies after treatment with gene therapy

Published: 2025-03-18 08:24:03 ET SRPT

March 18 (Reuters) - Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for rare muscular dystrophy.

Shares of the company fell more than 25% to $75.86 in premarket trading.

The gene therapy, branded as Elevidys, is approved in the U.S. for patients with Duchenne muscular dystrophy aged four and older.

Acute liver injury is a known possible side effect of the therapy and mentioned in its prescribing information, the company said.

Although it is not a new safety signal, acute liver failure leading to death represents a severity of acute liver injury not previously reported for the therapy. (Reporting by Sriparna Roy in Bengaluru; Editing by Shilpi Majumdar and Shinjini Ganguli)