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BioMarin May Have To Find Subgroups For Drisapersen Efficacy

 
• By Derrick Gingery

FDA advisory committee has concerns about efficacy, says more work needed to identify responders.

BioMarin Pharmaceutical Inc.’s drisapersen appears to show efficacy in some patients, but with lingering questions about the strength of available clinical data, the company may have to determine the subgroups that respond to the therapy in order to gain approval.

FDA's Peripheral and Central Nervous System Drugs Advisory Committee did not vote on an approval recommendation during their Nov. 24 meeting about the product, but comments indicated many doubts about...

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More from North America

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