EMA to review drisapersen, Galafold, Alprolix
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said EMA accepted for review an MAA for drisapersen to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In April, BioMarin completed submission of a rolling NDA to FDA for the antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene. Drisapersen 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 (see BioCentury Extra, April 27). BioMarin slipped $0.52 to $134.84 on Friday.
Amicus Therapeutics Inc. (NASDAQ:FOLD) said EMA accepted for review its MAA for Galafold migalastat to treat Fabry's disease in patients with amenable genetic mutations. Last month, EMA's CHMP granted accelerated assessment to Galafold. Next half, Amicus plans to submit an NDA to FDA and seek accelerated approval for the small molecule that enhances alpha galactosidase A activity. Amicus rose $0.39 to $14.05 on Friday. ...