FDA delays approval of BioMarin's Kyndrisa, issuing CRL

The US Food and Drug Administration has issued a Complete Response Letter to BioMarin Pharmaceutical’s (Nasdaq: BMRN) New Drug Application for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping.

The FDA issues CRLs to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. The agency has concluded that the standard of substantial evidence of effectiveness has not been met. BioMarin is reviewing the Letter and will work with the FDA to determine the appropriate next steps regarding this application.

Duchenne affects approximatelyone1 in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood. There is currently no FDA-approved therapy designed specifically to treat Duchenne, noted BioMarin.