No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy
Noises From US FDA Positive Ahead Of PDUFA
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.
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Former OTAT Director Wilson Bryan says Peter Marks’ order to file Sarepta’s DMD gene therapy application was mainly a public relations move, which does not seem to have worked in the FDA’s favor. Upcoming advisory committee could now involve as much scrutiny of the agency as the application itself.
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
The route to market for Sarepta’s gene therapy candidate SRP-9001 for Duchenne muscular dystrophy has just got more complex after a U-turn by the US FDA. Its accelerated approval will now be scrutinized by outside experts – but the company remains confident in its data.