NOVATO, Calif., April 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that Firdapse™ (3,4-diaminopyridine) is now commercially available in the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). Launching immediately in Germany and the UK, the company expects to subsequently launch Firdapse in all major European markets by the end of 2010. Firdapse received marketing approval in the E.U. for the treatment of LEMS in December 2009 and is the first approved treatment for this indication, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe.
"The launch of Firdapse brings the first specifically approved treatment option for LEMS to patients in the E.U. and marks our fourth commercial product on the market. We look forward to meeting with the FDA in the second quarter of 2010 to determine the necessary regulatory path for Firdapse in the U.S., and we also continue to evaluate the best development strategy for this product in other indications in the U.S. and Europe," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We remain committed to the advancement of our product pipeline and anticipate several notable upcoming milestones including results from the Phase I/II GALNS trial this current quarter and results from the Phase II PEG-PAL trial and Phase I trial of BMN-195 for Duchenne muscular dystrophy, both in the third quarter of 2010."
Lambert Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disease with the primary symptoms of muscle weakness. Muscle weakness in LEMS is caused by autoantibodies to voltage gated calcium channels leading to a reduction in the amount of acetylcholine released from nerve terminals. The prevalence of LEMS is estimated at four to ten per million, or approximately 2,000 to 5,000 patients in the EU and 1,200 to 3,100 patients in the U.S. Approximately 50 percent of LEMS patients diagnosed have small cell lung cancer.