BioMarin Acquires Prosensa, Duchenne Muscular Dystrophy Products
By Cyndi Root
BioMarin Pharmaceutical has acquired Prosensa Holding N.V. and its rare disease candidate drisapersen for Duchenne muscular dystrophy (DMD). The companies announced the deal in a press release, stating that the definitive agreement is for stock shares worth approximately $680 million. The acquisition of the drug fits BioMarin’s portfolio of drugs for rare diseases including Vimizim for Morquio A syndrome and Naglazyme for mucopolysacchariodosis VI. 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 will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible.”
BioMarin and Prosensa Agreement
Under the terms of the agreement between BioMarin and Prosensa, BioMarin acquires all the shares of the company and maintains operations at Prosensa's headquarters in Leiden, The Netherlands. Prosensa’s personnel will be integrated into BioMarin. BioMarin also acquires Prosensa’s portfolio of products and its pipeline, including drisapersen, which the Food and Drug Administration (FDA) is currently reviewing for approval. In the pipeline are products derived from Prosensa’s RNA-modulating technology platform. BioMarin pays cash upfront for the shares and has pledged milestone payments if drisapersen is approved in the U.S. and the EU.
Drispersen
Drisapersen (PRO051) is an exon skipping drug that Prosensa is developing for patients with deletions of exon 50, exon 52, exons 45-50, exons 48-50, and exons 49-50. The agent is sequence-specific, which minimizes the risk for off-target effects. The drug is under a rolling review by the FDA. BioMarin and Prosensa hope to have it approved in the U.S. by mid-2016. The clinical trial program includes three placebo-controlled trials, DEMAND II/DMD114117, DEMAND V/DMD114876, and DEMAND III/DMD114044, and two long-term studies, DMD114673 and DEMAND IV/DMD114349. Studies have included 300 people so far worldwide and the retention rate averages 96 percent.
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatal genetic disease caused by a gene mutation. About 20,000 new cases are diagnosed every year in mostly boys, due to the Duchenne gene on the X-chromosome. There is no cure for the disease. Sarepta Therapeutics of Cambridge, MA is also working on a treatment, eteplirsen.