Rare Disease Report

BioMarin Enters the Duchenne Race

NOVEMBER 24, 2014
James Radke

BioMarin Pharmaceuticals announced it plans to buy Prosensa Holding NV for approximately $840 million ($17.75 for all shares). In addition, BioMarin will offer two milestone payments of $80 million each following U.S. and European approvals for drisapersen, Prosensa's leading pipeline drug for treating Duchenne muscular dystrophy. The milestone payments are contingent on drisapersen being approved in the U.S. by May 15, 2016 and in the EU by February 15, 2017.

In a press release, Jean-Jacques Bienaimé, Chief Executive Officer 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 will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible. Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017."

Pat Furlong, President and Founder of Parent Project Muscular Dystrophy said,

"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."

The deal will give BioMarin a strong foothold in the Duchenne muscular dystrophy  market.  Prosensa is developing several exon skipping drugs, most notably for different mutations that lead to Duchenne muscular dystrophy. Its lead candidate – drisapersen – is in phase 3 clinical trials. Earlier this year, many thought the end was in sight for Prosensa when their phase 3 trial failed to meet its endpoint and the company’s partner - GSK – handed the drug back to Prosensa.

However, a reexamination of the data and subsequent meetings with regulatory agencies spurred the company to begin clinical trials again and have garnered the support of PPMD, CureDuchenne, and now BioMarin.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a progressive muscle disorder caused by the lack of functional dystrophin protein. Patients with Duchenne muscular dystrophy lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties.

There are an estimated 35,000 patients with Duchenne in the United States and Europe but the population has many subsets based on mutations of the dystrophin gene.

Both Sarepta’s eteplirsen or Prosensa’s drisaperson should be effective in the same 13% of the Duchenne population who would benefit from exon 51 skipping therapy (i.e., those with mutations near exon 51 of the dystrophin gene) while PTC Therapeutics’ ataluren should be effective in another 13% subset who have nonsense mutations in the dystrophin gene.

All three companies are starting, or have started, phase 3 confirmatory studies.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.