BioMarin Pharmaceutical Inc. has announced the withdrawal of its marketing authorization application from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The decision follows deliberations at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting, which resulted in an indication that the CHMP would issue a negative opinion on the application. In light of this decision and the U.S. Food and Drug Administration (FDA) Complete Response Letter in January, BioMarin will discontinue clinical and regulatory development of Kyndrisa, as well as three other follow-on products (BMN 044, BMN 045, and BMN 053) currently in Phase 2 clinical studies for different forms of DMD. BioMarin will continue to explore the development of next-generation oligonucleotides for the treatment of DMD. Now, the company will collaborate with medical physicians, patient groups, and regulatory authorities to develop a transition plan for those who are already receiving treatment with Kyndrisa or any of the three first-generation follow-on products.
