BioMarin May Have To Find Subgroups For Drisapersen Efficacy
This article was originally published in The Pink Sheet Daily
FDA advisory committee has concerns about efficacy, says more work needed to identify responders.
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Several members of the Peripheral and Central Nervous System Drugs Advisory Committee meeting looking at PTC' Therapeutics' proposed Duchenne muscular dystrophy treatment also looked at Sarepta's Exondys 51 and BioMarin's Kyndrisa, but most did not vote or comment favorably.
Cell and gene therapy product activities also have increased CBER’s workload significantly and strained staff, the agency tells industry representatives.
One of US FDA’s most important publications ever, the Orange Book celebrates 40 years as the definitive guide to approved drug products and therapeutic equivalents. Even as the color has receded from the book itself, orange still generates appreciation from regulators and outside attorneys.