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FDA Grants Orphan Drug Designation for 3,4-DAP for LEMS (Lambert EatonMyasthenic Syndrome) LEMS

Mon, 11/30/2009 - 4:18am
BioMarin PharmaceuticalInc. announced today that the FDA has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP),amifampridine phosphate, for the rare autoimmune disease Lambert EatonMyasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drugdesignation in the E.U. Also, in October 2009, the Committee for MedicinalProducts for Human Use of the European Medicines Evaluations Agency adopted apositive opinion recommending approval of amifampridine phosphate for LEMS.If approved by the European Commission, amifampridine phosphate will be thefirst approved treatment for LEMS, thereby conferring orphan drug protectionand providing ten years of market exclusivity in Europe. "With its experience in the development and commercialization of orphandrugs, BioMarin is well-positioned to seek U.S. registration of amifampridinephosphate for LEMS, a serious and debilitating autoimmune disease oftenassociated with small cell lung cancer," said Jean-Jacques Bienaime, ChiefExecutive Officer of BioMarin. "We look forward to meeting with the FDA inearly 2010 to determine the necessary regulatory path for amifampridinephosphate in the U.S. We are also preparing to launch the product in Europein the first quarter of 2010, and will also evaluate the best developmentstrategy for amifampridine phosphate in other indications in the U.S. andEurope."
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