Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the market

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.

Sarepta Therapeutics Inc. announced that the company plans to temporarily pause all shipments of its gene therapy to treat Duchenne muscular dystrophy, Elevidys, in a reversal of its prior stance.

Roche is following its partner Sarepta and halting shipments of the Duchenne gene therapy Elevidys in some countries, amid safety concerns.

Sarepta Therapeutics announces voluntary pause all shipments of Elevidys for Duchenne muscular dystrophy in US: Cambridge, Massachusetts Wednesday, July 23, 2025, 12:00 Hrs [IST]

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement:

Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker.

The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.

Sarepta Therapeutics said it will voluntarily “and temporarily” pause all shipments of Elevidys® (delandistrogene moxeparvovec-rokl), the marketed Duchenne muscular dystrophy (DMD) treatment linked to the deaths of two patients this year.