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Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids

 
• By Sue Sutter

SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.

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Product quality differences resulting from a manufacturing change for SRP-9001 represents an efficacy and safety wild card, one panelist said. • Source: Shutterstock

The US Food and Drug Administration panel review of Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy treatment SRP-9001 (delandistrogene moxeparvovec) demonstrated the agency’s unwillingness to cap the proportion of empty capsids in an AAV-based gene therapy – a reluctance that aligns with a 2021 advisory committee recommendation.

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