BioMarin to buy Prosensa for up to $840 million, gaining experimental DMD therapy drisapersen

BioMarin Pharmaceutical announced Monday a definitive agreement to buy Prosensa for up to $840 million, gaining rights to the experimental exon-skipping drug drisapersen for Duchenne muscular dystrophy (DMD). Under the deal, BioMarin will purchase all of Prosensa's outstanding shares for $17.75 per share, or approximately $680 million, and make two approximately $80 million contingent milestones related to approval of drisapersen in the US no later than May 15, 2016 and Europe no later than February 15, 2017, respectively.

"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," commented BioMarin CEO Jean-Jacques Bienaimé. The executive added "we will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible."

Prosensa initiated a rolling submission seeking FDA approval of drisapersen last month, with the company expecting to complete its filing in the first quarter of next year. The drugmaker also previously announced plans to seek European clearance of the exon-skipping therapy. BioMarin predicted that drisapersen, which it said is "a potential first-to-market" product, could be appropriate for as many as 10 000 patients, with follow-on drugs serving 35 000 people in its commercial regions.

Bienaimé suggested that the FDA could render a decision on drisapersen next year, adding that he was "somewhat comfortable" with analysts' projections that the drug could be priced at $250 000 to $300 000 per patient per year. BioMarin said that the purchase of Prosensa could be accretive to earnings by 2017 if drisapersen wins approval in the US and Europe.

The price of the deal, which is expected to close in the first quarter of 2015, represents a premium of 55 percent over Prosensa's closing price on November 21. BioMarin indicated that it will maintain Prosensa's operations at the company's headquarters in Leiden, Netherlands. BioMarin added that Prosensa's pipeline contains several drugs based on the company's RNA-modulating technology platform for the treatment of various genotypes of DMD and other genetic disorders. For related analysis, see ViewPoints: Can BioMarin pull a rabbit from this hat?

Other companies developing therapies for DMD include PTC Therapeutics and Sarepta Therapeutics. In August, PTC gained conditional marketing approval in Europe for Translarna (ataluren) as a treatment of nonsense mutation DMD in ambulatory patients aged five years and older. Meanwhile, Sarepta said last month that it plans to seek FDA approval of eteplirsen by mid-2015, delayed from a previous timeline of the end of this year, after the agency asked for further data to be included in the filing.

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