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Kuvan, Naglazyme and Aldurazyme Data to Be Presented at the 57th Annual Meeting of the American Society of Human Genetics

NOVATO, Calif., Oct. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) announced today that data from clinical studies of Kuvan(TM) (sapropterin dihydrochloride), Naglazyme(R) (galsulfase), and Aldurazyme(R) (laronidase) will be presented at the 57th Annual Meeting of the American Society of Human Genetics in San Diego, California, October 23-27, 2007.

Data to be presented is summarized below:

Platform Presentation - Session 59 - Therapy for Genetic Disorders

Sapropterin dihydrochloride (sapropterin) increases Phenylalanine (Phe) tolerance in children with phenylketonuria (PKU) maintained on a Phe-restricted diet

Presented by Dr. Barbara Burton of Children's Memorial Hospital, Chicago Saturday, October 27 at 8:30am

-- Double-blind, placebo-controlled Phase 3 study in 90 subjects four to

twelve years of age with a diagnosis of PKU with hyperphenylalaninemia

and controlled on a Phe-restricted diet for more than six months.

-- Patients received sapropterin 20mg/kg/day for eight days. Responders

(greater than or equal to 30% reduction in blood Phe on Day 8) were

randomized 3:1 to sapropterin or placebo for ten weeks.

-- Primary endpoint was daily Phe supplement tolerated during 10 weeks

while maintaining adequate blood Phe control.

-- Mean daily Phe intake (dietary + supplement) increased from 16.8 to

43.8mg/kg/day with sapropterin (p<0.001) and from 16.3 to 23.5mg/kg/day

with placebo (p=0.079).

-- Sapropterin had an acceptable safety profile. The most common adverse

events were headache, abdominal pain, fatigue, and diarrhea.

Phase 3 extension 96-week study for Naglazyme (galsulfase) Enzyme Replacement Therapy (ERT) in MPS VI (Maroteaux-Lamy Syndrome) patients

Presented by Dr. Paul Harmatz of Children's Hospital, Oakland Saturday, October 27 at 8:15am

-- Pivotal, Phase 3 open-label extension study reported safety and

efficacy data of the 24-week pivotal Phase 3 trial, followed by a

72-week extension study for a total of 96 weeks.

-- Endpoints included 12-minute-walk-test (12MWT), 3-minute-stair-climb

(3MSC), level of urinary glycosaminoglycans (GAGS) and pulmonary


-- Patients receiving Naglazyme improved by a mean of 183 m +/- 26 m from

baseline to week 96 in the 12MWT (p<0.001).

-- The placebo group, which was switched to active drug at week 24,

improved by a mean of 117 m +/- 25 m from week 24 to week 96 (p<0.001).

-- Similar improvements in the rate of stairs climbed (3MSC) were also

observed (p<0.001).

-- A sustained reduction in urinary GAGs was observed in both treatment


-- Several pulmonary function measures showed improvement at Week 96,

including forced vital capacity (FVC), which improved 0.11 L +/- 0.05 L

(p=0.039) in the Naglazyme group from baseline and improved 0.07 L +/-

0.02 L (p<0.001) in the placebo/Naglazyme group from Week 24.

-- Safety data for the extension study was similar to that measured in the

first 24 weeks of treatment. The overall safety profile remained

favorable. Adverse events reported by the greatest number of patients

during the extension study included headache, cough, pyrexia, diarrhea,

arthralgia and extremity pain. None of the adverse events that

occurred during infusion were severe.

The following relevant posters will also be featured:

-- 2229 -- Safety of sapropterin dihydrochloride (sapropterin) in children

with phenylketonuria (PKU) on a phenylalanine (Phe)-restricted diet


-- 2230 -- Safety and efficacy of sapropterin dihydrochloride

(sapropterin) treatment over 22 weeks in patients with phenylketonuria

(PKU) (Randolph)

-- 2231 -- Dose-related effect of sapropterin dihydrochloride

(sapropterin) on blood phenylalanine (Phe) in patients with

phenylketonuria (PKU) (Wasserstein)

-- 1495 -- Clinical Characteristics of MPS I patients in the MPS I

Registry (Bodamer)

-- 2241 -- Urinary GAG behavior and clinical correlation in three patients

with MPS I-Scheie during irregular ERT (Munoz-Rojas)

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. Both products are being developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Naglazyme(R) is a registered trademark of BioMarin Pharmaceutical Inc.

Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC.


Investors Media

Eugenia Shen Susan Berg

BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.

(415) 506-6570 (415) 506-6594

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2007 PR Newswire.
All rights reserved

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