News

BioPharma Dive1h
Sarepta woes mount as Duchenne gene therapy knocked back in Europe
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...
STAT2h
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
Sarepta fails to win EU backing for muscle disorder gene therapy
Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
BioSpace2h
Sarepta’s Future Increasingly Uncertain as FDA Eyes New Study for Elevidys
CBER is unanimously against Elevdiys’ return to the market without additional evidence, according to media reports citing an ...
Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the ...
Fierce Pharma19h
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 ...
EU panel declines backing Sarepta-Roche gene therapy for Duchenne
Friday said that the European Medicines Agency has issued a negative opinion on the conditional marketing authorization for Elevidys, intended for ambulatory patients aged three to seven with Duchenne ...
Sarepta stock falls after Elevidys fails to get backing by EU regulators
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 Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...