BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company's New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.
amenable to exon
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.
Duchenne affects approximately 1 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.
The ongoing Kyndrisa extension studies will continue, as will the ongoing clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045 and BMN 053, while BioMarin is exploring next steps for this application. Patients currently receiving Kyndrisa, BMN 044, BMN 045 and BMN 053 will remain on therapy.
An application for marketing approval of Kyndrisa is also under review in the European Union. BioMarin anticipates that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will provide an opinion for the company's Marketing Authorization Application (MAA) for Kyndrisa for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping in the first half of 2016. If the CHMP opinion is positive, the MAA will be referred to the European Commission (EC). If the MAA is approved by the EC, BioMarin would receive marketing authorization for Kyndrisa in all EU Member States. The EC is expected to render a final decision for Kyndrisa in the second half of 2016.
Kyndrisa is an antisense oligonucleotide that induces exon skipping to provide a molecular patch for dystrophin transcripts produced by certain mutated dystrophin genes. Exons are the parts of a gene that contain the instructions for generating a protein. In applicable cases, skipping an exon near the mutation allows for the production of a truncated but functional dystrophin protein.
