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BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the Treatment of Pompe Disease

Mon, 08/30/2010 - 7:33am
Bio-Medicine.Org

NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.  An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.

"Receiving orphan drug designation from the FDA for BMN-701 is a significant milestone for our Pompe program.  As part of their assessment for designation, the FDA determined that BMN-701 is sufficiently different from alglusidase alfa (Myozyme/Lumizyme) to allow for a unique orphan designation.  For this reason, clinical superiority over alglusidase alfa will not be necessary to secure orphan exclusivity for BMN-701," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "This emphasizes our mission of developing innovative, products for orphan diseases with an unmet medical need.  We believe BMN-701 has the potential to possibly deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches using the recently acquired GILT technology."  

About Pompe DiseasePompe 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.  There are two main forms of Pompe disease: adult onset with an incidence of one in 57,000 births and infantile onset with an incidence of one in

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