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.
You may also be interested in...
A look at drugs the agency approved despite an advisory committee vote not to do so and how they compare to Biogen’s Alzheimer’s disease drug aducanumab.
An FDA advisory committee panel were critical of the agency’s review package for Biogen's aducanumab, raising uncertainty about the prospects of approval.
Given inconsistent study results, robustness of the efficacy evidence is expected to be key focus of the 6 November meeting of the Peripheral and Central Nervous System Drugs Advisory Committee; review could put a December 2019 guidance on the ‘substantial evidence’ standard to the test and offer insight into the amount of flexibility the agency is willing to exercise for neurodegenerative diseases with high unmet need.