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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Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
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.
US FDA Neuroscience Office Director Billy Dunn Departs As Agency Faces New Review Challenges
Dunn is retiring after an 18-year career that included involvement in some high-profile and controversial drug approval decisions, not the least of which is Biogen’s Alzheimer’s treatment Aduhelm; deputy director Teresa Buracchio takes over the neurology office on an acting basis.
Rare Disease Patients: Placebo Controls, Sham Surgeries Not Appropriate For Gene Therapy Trials
Advocates also worry that long follow-up times could prevent participation in future clinical trials.