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No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy

Noises From US FDA Positive Ahead Of PDUFA

 
• By Andrew McConaghie

The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.

Sarepta building
Sarepta looks to be on the cusp of an historic approval for its Duchenne gene therapy, expected on 29 May. • Source: Shutterstock

Sarepta’s chances of gaining approval for the first ever Duchenne muscular dystrophy gene therapy and setting a new benchmark in the market look stronger following a decision by the US regulator not to hold an advisory committee to inform its review of the company's SRP-9001.

That means there are no extra hurdles for SRP-9001 (delandistrogene moxeparvovec) to clear before an expected US Food and Drug Administration decision on the one-time gene therapy on 29 May, which

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