FDA issues Complete Response Letter for Kyndrisa (drisapersen) to treat Duchenne muscular dystrophy- Biomarin

BioMarin Pharmaceutical Inc.announced that the FDA issued a Complete Response letter to the Company's New Drug Application (NDA) for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping. The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA has concluded that the standard of substantial evidence of effectiveness has not been met. BioMarin is reviewing the Complete Response Letter and will work with the FDA to determine the appropriate next steps regarding this application.