The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.

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

Wall Street analysts warn that if Sarepta Therapeutics doesn't resolve the Elevidys issues, it could jeopardize its ability to repay debt in 2027.

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

There were warnings to the FDA about Sarepta Therapeutics Inc. before US regulators asked the company to halt shipments of its gene therapy.

BMO Capital Markets pointed to FDA leadership, and CBER Director Vinay Prasad in particular, as potential factors in the ...

The Food and Drug Administration on Monday named a longtime pharmaceutical executive to run the agency's drug program, the latest in a string of leadership changes at the agency.

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...

Cell therapy developers are also set to face increased regulatory scrutiny since the appointment of Vinay Prasad as CBER chief in May.