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BioMarin to buy Prosensa in $840 million deal

pharmafile | November 25, 2014 | News story | Sales and Marketing |  BioMarin, Prosensa, drisapersen, dystrophy 

Rare-disease drug firm BioMarin has finalised its purchase of muscular disease specialist Prosensa in a deal worth over $840 million.

US-based BioMarin will pay $680 million upfront for the Dutch company, with two additional $80 million milestones payable for the approval of its Duchenne muscular dystrophy (DMD) drug drisapersen in the US and Europe.

“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,” says Jean-Jacques Bienaimé, chief executive of BioMarin.

He goes on: “We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible.

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Drisapersen is just one of a number of orphan drug candidates BioMarin will gain worldwide rights to as part of the acquisition. The DMD treatment is one of Prosensa’s more promising drugs, however, and is currently under rolling review as part of a New Drug Application (NDA) with the FDA. It will need to be approved in the US by 15 May 2016 (and in Europe by 15 February 2017) in order for Prosensa to receive the milestone payments.

DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500 live male births. It is caused by a gene mutation that causes progressive muscle weakness as a child and often results in death when the patient is in their late twenties. There is currently no cure for DMD. As the gene in question is found in the X-chromosome, it primarily affects boys.

Pat Furlong, president and founder of Parent Project Muscular Dystrophy, says: “BioMarin has a successful track record of developing new therapies for people with devastating disorders and for effectively collaborating with health authorities and patient communities. We look forward to working with BioMarin to bring new treatments to boys with Duchenne and other forms of muscular dystrophy.”

Hans Schikan, chief executive of Prosensa, adds: “This transaction will enhance Prosensa’s mission by bringing innovative therapies to patients across the world as quickly and efficiently as possible.”

George Underwood

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