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Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The Lancet
Date:8/19/2007

NOVATO, Calif., Aug. 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that final results from the Kuvan(TM) (sapropterin dihydrochloride) pivotal Phase 3 clinical trial were published in the August 11, 2007, issue of The Lancet. The study suggests that treatment with Kuvan results in significant reductions in blood Phe levels in some phenylketonuria (PKU) patients. Kuvan, an investigational oral small molecule for the treatment of PKU, is being developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.

"This marks the first published Kuvan clinical study, and we expect additional publications in the coming months to build on the collection of data related to Kuvan," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "We recently received priority review from the FDA and hope to receive FDA approval by late November. In the interim, we are pleased to be able to provide Kuvan to PKU patients prior to commercial availability through our expanded access program."

The Phase 3 clinical study enrolled 89 patients with elevated blood Phe levels aged eight years and above at 29 sites in the United States, Europe and Canada. All patients enrolled had demonstrated a reduction in blood Phe levels following treatment with Kuvan in a Phase 2 screening study.

The patients were randomly assigned to receive placebo or 10 mg/kg of Kuvan daily for six weeks. Patients were evaluated every two weeks for changes in blood Phe levels and adverse events. The primary objective was to assess the efficacy of Kuvan compared with placebo for reduction of blood phenylalanine in patients with PKU. The secondary objective was to assess the safety of Kuvan compared with placebo. A total of 87 patients completed six weeks of treatment.

Highlights from the Phase 3 double-blind study are summarized below:

* Patients treated with Kuvan for six weeks had a mean decrease
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SOURCE BioMarin Pharmaceutical Inc.

Copyright©2007 PR Newswire.

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