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BioMarin Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA
Date:2/1/2011

NOVATO, Calif., Feb. 1, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.  

"In under two years, we have progressed the GALNS program from Clinical Trial Application to initiation of the Phase 3 trial.  We have received FDA feedback and have finalized the design of the Phase 3 pivotal trial," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "The study will be conducted at approximately 40 centers worldwide including Brazil, Japan, Taiwan, most Western European countries, Canada and the U.S.  The trial is expected to enroll approximately 160 subjects and will be the largest enzyme replacement therapy trial conducted. There are no therapeutic options for MPS IVA patients who have a high unmet medical need. Initiation of this well-designed pivotal study is an important milestone for both the company and the MPS IVA community."

The Phase 3 trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of GALNS in patients with MPS IVA.  The study will explore doses of two mg/kg/week and two mg/kg/every other week for a treatment period of 24 weeks.  The primary endpoint is the six-minute walk test, and the secondary endpoints are the three-minute stair climb test and urine keratan sulfate concentration.  

About MPS IVMucopolysaccharidosis IVA (MPS IVA, also known as Morquio A Syndrome) is a disease characterized by deficient activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic skeletal dysplasia, short stature,
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SOURCE BioMarin Pharmaceutical Inc.
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