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MHRA Issues Notice of Acceptance for Phase 3 Trial of GALNS for the Treatment of MPS IVA
Date:12/14/2010

NOVATO, Calif., Dec. 14, 2010 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and issued a notice of acceptance for the Phase 3 trial of N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA).  The company expects to initiate the Phase 3 pivotal trial early in the first quarter of 2011.

"This important milestone reinforces our confidence in the Phase 3 trial design for GALNS as we move another step forward in the development of a treatment option for MPS IVA," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "Results from the successful Phase 1/2 trial, along with our ongoing dialogue with health authorities in Europe and the U.S., have helped inform the design of a robust Phase 3 trial to maximize the therapy's chance for success."

The proposed Phase 3 trial would be a randomized, double-blind study with three study arms: placebo, two mg/kg every week and two mg/kg every other week in approximately 160 patients worldwide.  The primary endpoint would be 6-minute walk distance with an entry criteria of baseline walk distance no greater than 325 meters.  The company has submitted a special protocol assessment (SPA) to the FDA, with a response expected by late January.

About MPS IVThe rate of incidence of MPS IVA is as yet unconfirmed and varies among different populations but estimates vary between 1 in 200,000 live births and 1 in 250,000 live births. There are several studies that have documented the incidence as high as 1 in 76,000 live births in Northern Ireland. The estimated prevalence is between 1,000 and 1,500 patients in the U.S., EU and Japan and between 1,500 to 2,000 patients in the rest of the
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SOURCE BioMarin Pharmaceutical Inc.
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