ARTICLE | Company News
BioMarin ending Kyndrisa development
June 1, 2016 1:19 AM UTC
BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) said it withdrew an MAA for Duchenne muscular dystrophy (DMD) candidate Kyndrisa drisapersen after EMA's CHMP "clearly indicated" that it intended to issue a negative opinion. BioMarin said it will discontinue development of Kyndrisa and three follow-on DMD candidates.
In January, FDA rejected an NDA for Kyndrisa, saying that the "standard of substantial evidence of effectiveness" was not met. BioMarin spokesperson Debra Charlesworth told BioCentury on Tuesday that CHMP and FDA had similar concerns over Kyndrisa's efficacy (see BioCentury Extra, Jan. 14). ...