BioMarin Pharma Inc. (BMRN) announced recently that its product Firdapse (Amifampridine phosphate) for treating Lambert Eaton Myasthenic Syndrome (LEMS) is commercially available in the European Union.

While the company has launched the product in Germany and the UK subsequent launches in other major European markets are expected by the end of the year. The LEMS drug received marketing approval from the European Commission in Dec 2009. Firdapse is the first drug to hit the European market for treating the rare autoimmune disease.

Firdapse enjoys orphan drug status and 10 years of market exclusivity in Europe. The drug has also been granted an orphan drug designation by the US Food and Drug Administration (FDA).

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 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.

BioMarin obtained the rights to Firdapse through the acquisition of Huxley Pharmaceuticals in Oct 2009. The company has three other products in the market. They are Aldurazyme, co-marketed with Genzyme Corporation (GENZ) for the treatment of MPS-I (mucopolysaccharidosis), Naglazyme for MPS-VI and Kuvan for phenylketonuria. The addition of Firdapse should augment its revenues in the coming quarters.

Currently, we are Neutral on BioMarin.

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