Prosensa is a biotechnology company based in Leiden, The Netherlands, which discovers and develops RNA-modulating therapeutics for the treatment of genetic disorders.
Jean-Jacques Bienaime, 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 are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition.”
Bienaime added, “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.”
BioMarin will maintain operations at Prosensa’s headquarters, based in Leiden, The Netherlands and integrate Prosensa personnel from that office.
The acquisition will provide BioMarin with worldwide rights to multiple orphan-drug candidates, including drisapersen, which is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Prosensa’s pipeline is comprised of several potential products that leverage their proprietary RNA-modulating technology platform for the treatment of various genotypes of Duchenne muscular dystrophy and other genetic disorders.