The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.

Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...

Sarepta Therapeutics (SRPT) said late Monday it will pause all shipments of its Duchenne muscular dystrophy gene therapy, ...

The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...

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

Sarepta Therapeutics said it will voluntarily “and temporarily” pause all shipments of Elevidys® (delandistrogene ...

Discovered and developed by Sarepta Therapeutics, Elevidys is a gene therapy that won accelerated approval in June 2023 and ...