Development of Drisapersen and Follow-On Compounds Discontinued

BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen.

The company recently withdrew its 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 that indicated the CHMP intended to issue a negative opinion.

Based on discussions at the CHMP meeting and the decision in January by the U.S. Food and Drug Administration that drisapersen was not ready for approval in the U.S. at that time, BioMarin made the decision to discontinue clinical and regulatory development of the drug. In addition, it will discontinue development of three other exon skipping drugs, BMN 044, BMN 045 and BMN 053, which have similar chemistry to drisapersen and are currently in phase 2 studies for different forms of Duchenne muscular dystrophy.

Drisapersen (brand name Kyndrisa) is an experimental exon skipping drug. It was being developed by BioMarin for the treatment of DMD in kids and adults who can be treated by skipping a section of genetic code in the dystdrophin gene called exon 51.

In a letter to the Duchenne community, the company said it is disappointed with this outcome and that it extends its sincere gratitude to every organization, caregiver, family and patient who has supported the development of drisapersen and the follow-on products over the past several years.

BioMarin plans to work with physicians, patient groups, and regulatory authorities to develop a transition plan for those patients currently being treated with drisapersen, BMN 044, BMN 045 and BMN 053. The Company says it will continue to explore the development of next generation oligonucleotides for the treatment of Duchenne muscular dystrophy.

“There are more than two dozen promising drugs under clinical development for treatment of DMD and MDA will continue to fund research in this area,” said Laura Hagerty, Ph.D., Scientific Program Officer for DMD at MDA.

For questions, please contact BioMarin Patient Advocacy (