MHRA Issues Notice of Acceptance for Phase 3 Trial of GALNS for the Treatment of MPS IVA BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has...

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.

The 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 world for a total of 2,500 to 3,000 patients. Over 1,000 MPS IVA patients worldwide have been identified through The International Morquio Organization (IMO) survey and the BioMarin MorCAP registry program.

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany ; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU. For additional information, please visit www.BMRN.com . Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

BioMarin®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

SOURCE BioMarin Pharmaceutical Inc.