ARTICLE | Clinical News

Drisapersen regulatory update

October 27, 2014 7:00 AM UTC

Prosensa began submitting a rolling NDA to FDA seeking accelerated approval of drisapersen to treat Duchenne muscular dystrophy (DMD). The company plans to complete the submission by year end and submit an MAA to EMA for conditional approval of drisapersen in early 2015. The product -- an 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. It also has Orphan Drug designation in the EU, Australia and Japan. ...