BIOMARIN'S RHASB GETS EUROPEAN ORPHAN DRUG DESIGNATION.

BioMarin Pharmaceutical Inc. (Nasdaq and SWX New Market: BMRN), Novato, Calif., has announced that the Committee for Orphan Medical Products of the European Agency for the Evaluation of Medicinal Products (COMP/EMEA) has rendered a positive opinion on the company's application for orphan drug designation in the European Community for rhASB (recombinant human arylsulfatase B), as enzyme replacement therapy for patients with Mucopolysaccharidosis (MPS) VI. With this designation, BioMarin will be granted ten years of market exclusivity if rhASB is the first product for MPS-VI to gain approval in the European market. In the United States, the FDA previously granted BioMarin orphan drug as well as fast track designations for rhASB in the treatment of MPS-VI.

Fredric D. Price, BioMarin's chairman and CEO, said, "This is another major milestone in the development of rhASB, a drug targeted at a disease for which there is currently no treatment available. Following successful completion of preclinical studies that indicated rhASB was both safe and effective in a naturally-occurring feline model of MPS-VI, we initiated a Phase I enzyme replacement trial in the United States in October 2000 with enzyme manufactured by BioMarin at our GMP facility licensed by the State of California.

"Our MPS-VI program builds on BioMarin's expertise in the development of enzyme replacement therapies, and positions us now with two orphan drug products in human clinical trials. We recently began a Phase III trial with Aldurazyme (TM), an enzyme replacement product for patients with MPS-I, another of the MPS family of genetic diseases. This trial is underway in collaboration with our joint venture partner, Genzyme General (Nasdaq: GENZ). Aldurazyme also received a positive opinion on orphan designation from COMP/EMEA."

The Product and the Disease

The MPS-VI clinical trial is testing the safety and efficacy of rhASB at two dosage levels using a variety of clinical and surrogate measures during a six-month patient evaluation period.

MPS-VI (also known as Maroteaux-Lamy syndrome) is a progressive, chronically debilitating genetic disease that proceeds from birth and results in early death. The patients are deficient in a single specific enzyme (ASB in the case of MPS-VI) that is produced in all cells and is required for the progressive, stepwise breakdown of certain complex carbohydrates found in all cells. This deficiency causes the progressive build-up of carbohydrate residues in the lysosomes, eventually resulting in cellular, tissue and organ dysfunction. MPS-VI patients are typically diagnosed at 6 to 24 months of age when they initially show various symptoms of the disease. Symptoms include inhibited growth, enlarged liver and spleen, skeletal and joint deformities, and upper airway obstruction. Life-threatening cardiopulmonary problems often develop in the teenage years and many patients do not survive past 20 to 30 years of age, particularly in the more severe cases of the disease. Patients with the milder cases of the disease may live longer but with significant medical problems. MPS-VI afflicts approximately 1,100 patients in the developed world.

About BioMarin Pharmaceutical Inc.

BioMarin specializes in the development and commercialization of therapeutic enzyme products. Since inception in 1997, BioMarin has applied its proprietary enzyme technology to develop products for lysosomal storage diseases and for the treatment of serious burns. Glyko, Inc., a BioMarin subsidiary, provides analytical and diagnostic products and services in the area of carbohydrate biology.

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