FDA Denies NDA for Duchenne Muscular Dystrophy Drug Drisapersen

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FDA logo
After a lukewarm review, the FDA has officially denied the NDA for drisapersen.

The FDA has officially denied the New Drug Application (NDA) for drisapersen (Kyndrisa) for the treatment of Duchenne muscular dystrophy amendable to exon 51 skipping.

The Complete Response letter comes nearly 2 months after the Peripheral and Central Nervous Systems Drugs Advisory Committee concluded that there was insufficient and inconsistent clinical evidence supporting the drug’s effect on the rare neuromuscular disorder.

During the review, the FDA committee expressed concern over the drug’s weak mechanism of action, as well as its safety profile.

Drug manufacturer BioMarin Pharmaceutical plans to continue the ongoing extension studies for drisapersen and clinical trials for other exon-skipping oligonucleotides, including BMN 044, BMN 045, and BMN 053. Patients who are currently receiving drisapersen, BMN 044, BMN 045, and BMN 053 will remain on the therapy.

Drisapersen is currently under review by the European Medicines Agency, with a decision expected in the first half of 2016.


BioMarin Pharmaceutical Inc. press release