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BioMarin Receives Orphan Drug Designation from FDA for Pompe Disease

Posted on: August 30, 2010

Excerpt from August 30, 2010 press release . . .

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.”

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