Drisapersen regulatory update

FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will meet on Nov. 24 to discuss an NDA from BioMarin for drisapersen to treat Duchenne muscular dystrophy (DMD) with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping. The PDUFA date is Dec. 27. The antisense oligoribonucleotide induces exon 51 skipping on the dystrophin gene. ...