The Peripheral and Central Nervous System Drugs Advisory Committee of the US Food and Drug Administration (FDA) met to discuss the data submitted to support USA-based BioMarin Pharmaceutical’s (Nasdaq: BMRN) New Drug Application for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.
The committee reviewed the Kyndrisa data package, which includes three randomized, placebo controlled trials with more than 300 patients and more than 500 patient years of exposure.
The panel of external advisers indicated that efficacy data from BioMarin on its experimental drug for treating the muscle wastage disorder was not persuasive enough. The FDA panel did not officially vote on an approval of the drug. However, 15 out of 17 members were of the opinion that the lack of statistical significance in BioMarin's late-stage study weakened findings from two earlier studies, according to a report by Reuters.
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