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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