BioMarin Acquires Prosensa For Up To $840M

California based drug maker BioMarin Pharmaceuticals has agreed to acquire Dutch biotech company, Prosensa, for nearly $840 million in a deal that BioMarin is hoping will enable the company to win regulatory approval for a treatment for Duchenne muscular dystrophy (DMD), a rare and fatal disease.

The Prosensa acquisition, which is BioMarin’s largest one to date, will provide the rare disease drug maker with worldwide rights to several orphan-drug candidates developed by Prosensa, including the DMD candidate, drisapersen. Drisapersen has received the U.S. FDA’s coveted Orphan, Fast Track, and Breakthrough Therapy designations. It is currently under rolling review as part of a New Drug Application (NDA) with the FDA. 

Under the terms of the agreement, BioMarin will buy Prosensa for approximately $680 million up-front. In addition, BioMarin will pay Prosensa an additional $80 million if drisapersen receives U.S. regulatory approval before May 2016, and another $80 million if it receives approval in Europe before February 2017.

Prosensa co-developed drisapersen with GSK, however this partnership ended last year following a setback in the clinical trials. Prosensa is still optimistic about the drug, as the FDA will consider approving the drug based on new analyses of the study data. Prosensa would still be required to provide additional data from two more clinical trials to support the drug’s benefits if it is approved. The company expects to complete the drug’s regulatory filing in the first quarter of 2015.

Duchenne muscular dystrophy is a genetic condition that causes muscle-weakening and muscle-wasting in children, especially in boys. Patients with DMD don’t usually live past 30. The fatal disorder affects nearly 15,000 to 20,000 people every year in the U.S. If approved, drisapersen is expected to be a blockbuster drug, with an estimated treatment cost of about $250,000 per patient per year, reports Reuters. BioMarin anticipates that the FDA will make a decision on drisapersen by the end of 2015.

Jean-Jacques Bienaimé, CEO of BioMarin, said, "BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs. We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition."

Mr. Bienaimé also says that Prosensa will benefit from BioMarin’s successful track record in developing and selling treatments for rare diseases. As the North Bay Journal reported last week, BioMarin has brought five "powerhouse" orphan drugs to the market in the past thirteen years.