September 1, 2010 (FinancialWire) (Go to http://www.financialwire.net/?s=ftrdnwswnd for all of today’s featured news.) — BioMarin Pharmaceutical, Inc. (NASDAQ: BMRN) said that it has received orphan drug designation from the U.S. Food and Drug Administration for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase in development for the treatment of Pompe disease. According to the company, an investigational new drug application for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is scheduled to start in the first quarter of 2011.

Pompe disease, a lysosomal storage disorder, is a progressive degenerative disease of the heart muscle, diaphragm and skeletal muscle. It is caused by a deficiency in the lysosomal enzyme acid alpha glucosidase which leads to the accumulation of glycogen in myocyte lysosomes and results in cell death. The incidence is one in 40,000 births.

California-based BioMarin develops and commercializes biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates.

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