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BioMarin Announces Program for ERT for Treatment of MPS IVA - Morquio A Syndrome
Date:6/5/2008

NOVATO, Calif., June 5 /PRNewswire-FirstCall/ - BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced its program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome. BioMarin plans to initiate a Phase 1/2 clinical trial in the first quarter of 2009.

"With two MPS drugs on the market, we plan to leverage our clinical, manufacturing and regulatory expertise to efficiently develop a treatment for Morquio patients," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "Our planned program includes a clinical assessment study to measure the depth and breadth of disease as well as a separate GALNS Phase 1/2 study in Morquio Type A patients. Preliminary studies are promising and indicate that our drug candidate binds naturally to bone matrix and can adequately reach the growth cartilage after IV infusion. The skeletal system disease is a primary concern in the treatment of this disease."

The company has successfully developed and manufactures two FDA-approved enzyme replacement therapies for the treatment of MPS I and MPS VI. Naglazyme(R) (galsulfase) MPS VI is wholly developed and commercialized by BioMarin. Aldurazyme(R) (laronidase) for MPS I is manufactured by BioMarin and marketed by Genzyme Corporation.

Additional details of the MPS IVA program, along with an overview of BioMarin's product portfolio, advancements in the research and development pipeline and other ongoing programs will be provided today at BioMarin's R&D Day program in New York City. Interested parties may access a live audio webcast of the presentation via the investor section of the BioMarin website, http://www.BMRN.com. A replay of the presentation will be archived on the site for at least one week following the presentation. For general inquiries please email Morquio@bmrn.com

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SOURCE BioMarin Pharmaceutical Inc.
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