Biomarin withdraws MAA for Kyndrisa (drisapersen) from EMA as a treatment for Duchenne muscular dystrophy .

BioMarin Pharmaceutical Inc. announced that it has withdrawn its Kyndrisa (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

Based on discussions at the CHMP meeting and the Food and Drug Administration Complete Response Letter in January, BioMarin intends to discontinue clinical and regulatory development of Kyndrisa as well as the three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, currently in Phase II studies for distinct forms of Duchenne muscular dystrophy. Notwithstanding this outcome for Kyndrisa in Europe, the Company continues to expect to achieve non-GAAP break-even or better in 2017. BioMarin plans to work with physicians, patient groups, and regulatory authorities to develop a transition plan for those patients currently being treated with Kyndrisa, BMN 044, BMN 045 and BMN 053. The Company will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne muscular dystrophy.