Drisapersen regulatory update

BioMarin completed submission of a rolling NDA to FDA for drisapersen to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene has Fast Track, Orphan Drug and breakthrough therapy designation in the U.S. for DMD. BioMarin said that in about 13% of DMD patients, skipping of exon 51 restores the proper dystrophin reading frame. ...