FDA Issues Complete Response Letter to Kyndrisa
BioMarin announced that the Food and Drug Administration (FDA) has issued a Complete Response Letter to the New Drug Application (NDA) for Kyndrisa (drisapersen), for the treatment of Duchenne Muscular Dystrophy (DMD) amenable to exon 51 skipping.
The Complete Response Letter stated the FDA's conclusion that Kyndrisa failed to meet the standard of substantial evidence of effectiveness, denying the application's approval in its present form. The Company is reviewing the letter and will work with the FDA to determine the appropriate steps to take.
BioMarin will continue its ongoing Kyndrisa extension studies, as well as ongoing clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045 and BMN 053, while exploring the next steps for this application. Patients currently on Kyndrisa, BMN 044, BMN 045 and BMN 053 will continue to receive their treatments.
Kyndrisa, an antisense oligonucleotide, induces exon skipping to provide a molecular patch for dystrophin transcripts produced by certain mutated dystrophin genes.
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