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Golodirsen Rebuff: Did Exondys 51 Review Strife Influence US FDA's Decision?

 
• By Michael Cipriano

Sarepta cited two safety issues when announcing a complete response letter for its exon 53 skipping DMD drug golodirsen, but the US FDA's decision may be a result of the Exondys 51 approval from 2016, analysts suggest.

Rejected stamp

The US Food and Drug Administration's rejection of Sarepta Therapeutics Inc.' Duchenne muscular dystrophy (DMD) treatment golodirsen came as a shock to many, raising questions about whether the fallout from the Exondys 51 (eteplirsen) review factored into the agency's decision.

Sarepta announced 19 August that it received a complete response letter (CRL) for golodirsen, which is designed to treat DMD patients who have genetic mutations that are amenable to exon 53 skipping

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