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FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report
Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the company over a mounting string of controversies—to the FDA, Endpoints News and Bloomberg separately reported Thursday, citing an unnamed senior FDA official.
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
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.
Sarepta and Roche face a regulatory setback as the EMA's CHMP issues a negative opinion on Elevidys for Duchenne muscular ...
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...
Despite a new setback for Elevidys in Europe, Roche—which markets Sarepta’s gene therapy outside the U.S.—remains committed ...
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
3don MSN
After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy ...
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