FDA Advisory Committee Discusses Clinical Data Package for BioMarin's Kyndrisa for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin Pharmaceutical Inc.

announced today that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) met to discuss the data submitted to support the New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) 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 committee discussed the overall strengths and weaknesses of the application but was not asked to vote on a recommendation for approval of Kyndrisa.  "I'd like to thank the patients, families and physicians who participated in Kyndrisa clinical trials and in today's panel discussion," said Jean-Jacques Bienaimé, chairman and chief executive officer of BioMarin.  "We look forward to continuing our efforts to bring Kyndrisa to the Duchenne community.  After today's meeting, our next step is to continue working with