Blow for Sarepta as FDA rejects Duchenne candidate
US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) saw its share plunge after it revealed that it has received a Complete Response Letter (CRL) from the US Food and Drug Administration regarding the New Drug Application (NDA) for Vyondys 53 (golodirsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.
The decision comes as something of a surprise, given that golodirsen is similar to Sarepta's already marketed drug Exondys 51 (eteplirsen), which was approved by the agency for use in another subset of Duchenne patients in September 2016.
The company’s shares fell nearly 4% by close of regular trading and slumped a further 13.3% to $104.31 after hours. Sarepta said it will immediately request a meeting with the FDA to determine next steps.
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