FDA Grants Orphan Drug Designation For 3,4-DAP

BioMarin has announced that the FDA has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

3,4-DAP has previously received orphan drug designation in the EU. Also, in October 2009, the Committee for Medicinal Products for Human Use of the European Medicines Evaluations Agency adopted a positive opinion recommending approval of amifampridine phosphate for LEMS.

If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe.

Jean-Jacques Bienaime, chief executive officer of BioMarin, said: “With its experience in the development and commercialisation of orphan drugs, BioMarin is well-positioned to seek U.S. registration of amifampridine phosphate for LEMS, a serious and debilitating autoimmune disease often associated with small cell lung cancer.

“We look forward to meeting with the FDA in early 2010 to determine the necessary regulatory path for amifampridine phosphate in the US. We are also preparing to launch the product in Europe in the first quarter of 2010, and will also evaluate the best development strategy for amifampridine phosphate in other indications in the US and Europe.”