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BioMarin completes NDA submission for drisapersen

April 28, 2015 1:23 AM UTC

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) completed the submission of a rolling NDA to FDA for drisapersen ( PRO051) to treat a genetically defined subset of patients with Duchenne muscular dystrophy (DMD).

Drisapersen, an antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene, has Fast Track and breakthrough therapy designations from FDA, and Orphan Drug designation in the U.S. and EU. BioMarin said that in about 13% of DMD patients, skipping of exon 51 restores the proper dystrophin reading frame. ...