BioCentury
ARTICLE | Clinical News

Kyndrisa drisapersen regulatory update

January 25, 2016 8:00 AM UTC

FDA issued a complete response letter to BioMarin for an NDA for Kyndrisa drisapersen to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. According to the company, FDA concluded the “standard of substantial evidence of effectiveness has not been met.”

FDA is requesting an additional trial. BioMarin will determine next steps after meeting with FDA and after a decision anticipated in June from EMA’s CHMP on an MAA for drisapersen. The product is an antisense oligoribonucleotide that induces exon skipping on the dystrophin gene. ...