Recently, BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. Food and Drug Administration (FDA) granted orphan drug status to its candidate 3,4-diaminopyridine amifampridine phosphate (3,4-DAP), for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS).

As a reminder, BioMarin acquired the privately held Huxley Pharmaceuticals Inc. last month which possessed rights to a proprietary form of 3, 4-diaminopyridine amifampridine phosphate for LEMS treatment.

In October 2009, the Committee for Medicinal Products for Human Use of the European Medicines Evaluations Agency gave a positive opinion and recommended the approval of amifampridine phosphate for LEMS. The European Commission (EC) is expected to deliver its decision regarding the approvability of the candidate in late 2009 or early 2010.BioMarin intends to launch the drug in Europe in the first quarter of 2010 and is looking to develop an appropriate strategy for the drug in the U.S. Once approved, the candidate will enjoy orphan drug status and 10 years of market exclusivity in Europe.

The drug has the potential to be used for other indications as well including multiple sclerosis (MS). However, the MS market is already dominated by big players such as Biogen Idec (BIIB), Pfizer (PFE) and Teva Pharmaceutical Industries (TEVA).

LEMS, a rare autoimmune disease, affects 1,200 to 3,100 people in the US and 2,000 to 5,000 in Europe. Muscle weakness, mainly in the legs and trunk, is the main symptom. Approximately half of the diagnosed LEMS patients are affected by small cell lung cancer. LEMS results in other problems as well such as reduced reflexes, drooping of the eyelids, facial weakness and problems in swallowing.

Currently, we are Neutral on BioMarin.

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