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FDA rejects BioMarin's Kyndrisa

January 15, 2016 3:00 AM UTC

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said FDA issued a complete response letter 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."

BioMarin spokesperson ‎Debra Charlesworth said FDA asked for another trial. "We will need to meet with them to understand what that trial might look like," she said. 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. ...