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.
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Company’s prediction that Duchenne muscular dystrophy patients likely would not be able to access SRP-9001 until four months after accelerated approval eased some US FDA panelists’ concerns about interference with the ongoing confirmatory trial.
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.
Chief operating officer Christina Rossi talked to Scrip about the company’s new blockbuster-sized commercial opportunity for Ayvakit and the company’s next phase: financial sustainability.