BioMarin Pharmaceutical Inc. announced positive long-term results from phase 1 and phase 2 clinical studies of Aryplase, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis VI (MPS VI). Long-term data from both studies indicate that Aryplase is generally well tolerated and that patients continue to benefit from Aryplase treatment.

John Hopwood, Ph.D., of Brigham and Women's Hospital, Adelaide, Australia, and Paul Harmatz, M.D. of Children's Hospital Research Institute, Oakland, California, are presenting data from the phase 1 and phase 2 studies respectively, at the 53rd annual meeting of the American Society of Human Genetics in Los Angeles, California, from November 4-8, 2003.

"We are pleased to see the clinical improvements these patients experienced, especially those observed beyond the initial six months of treatment and well into the second year of weekly Aryplase infusions," stated Stuart Swiedler, M.D., Ph.D., vice president, clinical affairs of BioMarin. "Already, well in advance of a potential NDA filing, we have enrolled approximately 170 MPS VI patients in clinical and disease survey studies, who someday may benefit from Aryplase treatment."

BioMarin Pharmaceutical Inc. specializes in the development and commercialization of enzyme therapies for serious, life-threatening diseases and conditions.