Complete Response Letters recently issued by the FDA signal heightened scrutiny of trial design and reinforce the agency’s shifting regulatory expectations for sponsors and CROs.

This week's episode covers news about Sarepta, babies born following mitochondrial transfer, and AI identifying therapy ...

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

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

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...

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

Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) plunged 13% on Friday after a major regulatory blow in Europe. The European ...

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

Separately, Sareptea said last week that it would pause the development of most of its experimental gene therapies for a different type of muscular dystrophy. The stoppage came after one of the ...

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

Most Americans encounter the Federal Trade Commission only if they’ve been scammed: It handles identity theft, fraud, and ...

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