BioMarin Pharmaceutical Inc. (BMRN) said that the U.S. Food and Drug Administration issued a Complete Response letter to the company's New Drug Application or NDA for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy 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. The FDA 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 about 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.
BioMarin noted that 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. The company also said that 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 added.
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