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Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen

 
• By Mandy Jackson

The US FDA issued a complete response letter rejecting golodirsen based on risk of infection at intravenous infusion ports and preclinical renal toxicity.

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US FDA approval never is certain, but golodirsen's rejection was surprising. • Source: Shutterstock

Sarepta Therapeutics Inc. hoped to win approval for a second exon-skipping drug for Duchenne muscular dystrophy (DMD), golodirsen, on 19 August, but instead announced that it received a complete response letter (CRL) from the US Food and Drug Administration, which president and CEO Doug Ingram described as a surprising development based on recent communication with the agency.

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