Genzyme General And BioMarin To Develop Treatment For Lysosomal Storage Disorder

Genzyme General and BioMarin Pharmaceutical, Inc. have formed a joint venture to develop and commercialize BioMarin's lead product candidate, alpha-L-iduronidase, a recombinant enzyme to treat the lysosomal storage disorder, mucopolysaccharidosis I (MPS I).

Under the terms of the agreement, Genzyme will make a $12.1 million milestone payment to BioMarin upon receipt of approval from the U.S. Food and Drug Administration of a Biologics License Application for alpha-L-iduronidase to treat MPS I. In addition, Genzyme has made an $8 million equity investment in BioMarin and will purchase $10 million of BioMarin stock, at the price offered to the public.

In preclinical studies, scientists demonstrated that enzyme replacement therapy with recombinant alpha-L-iduronidase may be effective in treating many of the symptoms of MPS I. The trial is being conducted at the Harbor-UCLA Research and Education Institute in Torrance, CA under the direction of Dr. Emil Kakkis, president of BioMarin Genetics and assistant professor, Division of Medical Genetics, Department of Pediatrics at the UCLA Medical Center.