Slim Adcomm Majority Boosts Sarepta’s Gene Therapy In Duchenne Muscular Dystrophy
Executive Summary
Accelerated approval seems imminent after a majority of the US FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted the risk-benefit balance supported it.
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As Sarepta again brings a product application from the brink of rejection to the cusp of approval, the one-month review delay creates more assurance the confirmatory EMBARK trial will be completed as well as providing more time for label negotiations with the US FDA.
Commercial Fate Of Sarepta’s DMD Gene Therapy Remains Tied To EMBARK
The company guided investors to expect a narrow initial label under a US FDA accelerated approval for SRP-9001, with expansion possible based on EMBARK.
The Power Of The Open Public Hearing
The US FDA took three high profile applications to an external advisory committee meeting during the week of 8 May – and offered a very skeptical read on the approvability of each. All three won positive votes from the committees, in no small part thanks to the messages delivered during the Open Public Hearings.