CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the ...

Sarepta Therapeutics (SRPT) stock drops as the company faces potential FDA-mandated studies after safety concerns over its ...

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

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

Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...

European regulators on Friday said that the Duchenne muscular dystrophy gene therapy Elevidys should not be approved ...

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

Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...

Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company’s gene therapy for Duchenne muscular dystrophy.

Sarepta said it accepts the CHMP decision on Elevidys, while partner Roche said it will continue working with the EMA.

Roche Holding AG’s gene therapy Elevidys failed to get the backing of European regulators, a blow to the Swiss drugmaker and to Sarepta Therapeutics Inc., the embattled US biotech that developed the ...