BioMarin Receives Rare Pediatric Disease Designation From FDA for Drisapersen for the Potential Treatment of Duchenne Muscular Dystrophy

SAN RAFAEL, Calif., Aug. 19, 2015 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment. The FDA has previously granted drisapersen Orphan and Fast Track Status, Breakthrough Therapy Designation, as well as Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.
“BioMarin has a track record of efficiently developing therapies to treat rare and ultra-rare genetic diseases and we are committed to bringing drisapersen to children who desperately need an approved treatment option in the U.S. We are pleased that the FDA’s Office of Orphan Products Development has granted drisapersen this designation,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. “BioMarin was the first Company to receive a Rare Pediatric Disease Priority Review Voucher upon the approval of Vimizim®, in February of 2014. We are dedicated to developing important new therapeutics for patients, mostly children, with life threatening disorders, and this designation for drisapersen is a testament to BioMarin’s deep commitment to that goal.”

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