Tag Archives: dmd

Sarepta Plummets As FDA Slams Muscular Dystrophy Drug

Biotech Sarepta Therapeutics’ (SRPT) stock price was cut in half Friday after the FDA released highly critical briefing documents for an advisory committee evaluating Sarepta’s muscular dystrophy drug. The committee is scheduled to meet next Friday to review eteplirsen, a drug for Duchenne muscular dystrophy (DMD). Sarepta filed for approval of the drug last year after a long back-and-forth with the FDA over the appropriate amount of evidence in

BioMarin Muscular Dystrophy Drug Rejected By FDA

BioMarin Pharmaceutical (BMRN) said Thursday that the FDA had rejected its Duchenne muscular dystrophy (DMD) drug Kyndrisa, saying the evidence for its effectiveness was inadequate. The news was not a surprise, as Kyndrisa (also known as drisapersen) had failed its clinical trials, leaving BioMarin to argue that the totality of the evidence supported the drug’s efficacy even though it didn’t hit the preset endpoints. The FDA called an advisory

BioMarin, Intercept Pharma Drugs Face FDA Delays

BioMarin Pharmaceutical (BMRN) and Intercept Pharmaceuticals (ICPT) stocks were heading in opposite directions after both of their closely-watched drugs received delays in their FDA approval decisions. BioMarin’s Duchenne muscular dystrophy (DMD) treatment Kyndrisa (drisapersen) had a decision deadline, or PDUFA date, of Dec. 27, which meant that the news was widely expected to come by Christmas Eve. However, BioMarin said in a brief statement