BioMarin Drug Cleared in Europe - Analyst Blog

BioMarin Pharma Inc. (BMRN) announced recently that its drug candidate 3, 4-diaminopyridine amifampridine phosphate (3,4-DAP), indicated for treating Lambert Eaton Myasthenic Syndrome (LEMS), has received marketing approval from the European Commission. 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. 

Amifampridine phosphate is the first drug to hit the market for treating the rare autoimmune disease. The drug enjoys orphan drug status and 10 years of market exclusivity in Europe. Amifampridine phosphate has also been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). BioMarin intends to launch amifampridine in the European Union in mid-March. Furthermore, the company is looking to develop an appropriate strategy for the drug in the U.S. after consulting the FDA. BioMarin also intends to evaluate amifampridine phosphate in a number of countries outside the U.S. and 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). 

BioMarin acquired the privately held Huxley Pharmaceuticals Inc. in October 2009, which possessed rights to a proprietary form of 3, 4-diaminopyridine amifampridine phosphate for LEMS. Huxley licensed the rights to the drug from EUSA Pharma. EUSA had in turn acquired the right from the original developer, Assistance Publique Hopitaux de Paris.
 
LEMS affects 1,200 to 3,100 people in the US and 2,000 to 5,000 in the European Union. 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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