Recently, BioMarin Pharmaceutical Inc. (BMRN) initiated a late-stage study (n~30) for Firdapse (amifampridine phosphate) to treat patients suffering from Lambert-Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disorder.
The study, to be conducted across 25 sites across the globe, will evaluate the safety and efficacy of the drug. The double-blind, placebo-controlled randomized discontinuation phase III study will be followed by an open-label extension period.
Management stated that the study was being conducted to meet the requirements necessary for obtaining marketing approval of the drug from the US Food and Drug Administration (FDA). We remind investors that Firdapse was launched in the European Union (EU) by BioMarin in April 2010.
BioMarin obtained the rights to Firdapse through the acquisition of Huxley Pharmaceuticals in 2009. Muscle weakness, mainly in the legs and trunk, is the main symptom of the rare disease. 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.
We note that apart from Firdapse, BioMarin has three other products in the market. They are Aldurazyme, co-marketed with Sanofi-Aventis (SNY) for the treatment of MPS-I (mucopolysaccharidosis), Naglazyme for MPS-VI and Kuvan for phenylketonuria.
Apart from the marketed products, BioMarin boasts of a robust pipeline with multiple events lined up in the near future. We are encouraged by the company’s efforts to develop its pipeline.
The initiation of the late-stage study for the LEMS drug marks the fourth clinical trial conducted by BioMarin so far in 2011. We believe that the successful development and commercialization of the robust pipeline would help drive long-term growth at BioMarin.
Our Recommendation
Currently, we have a Neutral long-term stance on BioMarin. The company carries a Zacks #3 Rank (Hold rating) in the short-run.
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