BioMarin, Sarepta DMD submissions move forward
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) and Sarepta Therapeutics Inc. (NASDAQ:SRPT) each provided regulatory updates for their Duchenne muscular dystrophy programs on Monday. BioMarin said FDA accepted and granted Priority Review to an NDA for drisapersen to treat DMD amenable to exon 51 skipping. The PDUFA date is Dec. 27. BioMarin said FDA plans to hold an advisory committee meeting to discuss the NDA, but has not set a date.
On Friday, BioMarin said EMA accepted for review an MAA for drisapersen. The 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 gained the product through its acquisition of Prosensa Holding N.V. last quarter. The company lost $2.53 to $132.31 on Monday. ...