Sarepta's Eteplirsen Approved After Contentious Internal FDA Debate
CDER Director Woodcock grants accelerated approval after FDA Commissioner Califf refuses to overturn her finding that the drug's effect on dystrophin is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy.
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Fourteen user fee goal dates are on the October calendar, including three novel agents, according to the Pink Sheet US FDA Performance Tracker.
Sarepta Plots Multi-Study Label Expansion For Elevidys To Eventually Cover Nearly All DMD Population
Studies supporting label expansion already are under way, with more targeting those currently ineligible, including boys with pre-existing antibodies, set to begin later this year, after the initial accelerated approval only allowed use in four- and five-year old patients.
FDA clinical, clinical pharmacology and statistical review teams did not recommend approval of Elevidys, but the CBER director concluded that randomized data in a subgroup of patients ages four and five years old were ‘compelling,’ justifying accelerated approval; expansion beyond this age-restricted subgroup will hinge on data from the EMBARK confirmatory trial.