FDA Issues Complete Response Letter for BioMarin’s dirsapersen

BioMarin Pharmaceutical announced FDA issued a complete response letter to dirsapersen, disclosing that the NDA was not approved.

BioMarin Pharmaceutical announced FDA issued a complete response letter (CRL) to the company’s new drug application (NDA) for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy amendable to exon 51 skipping.

FDA issues CRL to indicate that the review cycle for an NDA is complete, and that the application is not ready for approval. According to a Jan. 14, 2016 press announcement from BioMarin, FDA concluded that the standard of substantial evidence of effectiveness has not been met.

This comes after FDA announced in December 2015, that the agency would need more time to review the NDA for drisapersen. The agency missed the December 27, 2015 action date and anticipated action in early January 2016. 

Although the application was not approved, the company says that ongoing Kyndrisa extension studies will continue, as will clinical trials for other exon-skipping oligonucleotides, BMN 044, BMN 045, and BMN 053. Patients currently receiving Kyndrisa, BMN 044, BMN 045, and BMN 053 will remain on therapy. A marketing authorization application for the drug remains under review in Europe.

The company is planning to work with FDA to determine the next steps regarding the application. 

Source: BioMarin