New Therapy for Rare Children's Disorder

A clinical trial has demonstrated that patients seriously ill with mucopolysaccharidosis I (MPS-I) showed improvement when treated with an enzyme replacement therapy, recombinant human alpha-1-iduronidase (Aldurazyme). The findings, by Dr. Elizabeth Neufeld and colleagues at the National Institutes of Health and the University of California, Los Angeles, School of Medicine (CA, USA), were reported in the January 18 issue of The New England Journal of Medicine.

MPS-1 is a life-threatening genetic disorder that affects young people and for which there is currently no known adequate treatment. The 10 trial subjects ranged in age from 5-22. Analysis of the data following 52 weeks of therapy demonstrated that the drug was well tolerated. The improvements noted included decreased liver or spleen size, reduced excretion of complex carbohydrates in urine, improved range of motion in the shoulder, a reduction in sleep apnea, and near-double increases in height-growth velocity. A phase III clinical trial began in December 2000.

Aldurazyme was developed by BioMarin (Novato, CA, USA) and Genzyme General (Cambridge, MA, USA) and is being jointly commercialized by the two companies. They have received Orphan Drug designation for Aldurazyme from the U.S. Food and Drug Administration (FDA).

"Enzyme replacement therapy with recombinant alpha-1-iduronidase was first shown to reverse carbohydrate accumulation in canines with a naturally occurring form of the same disease. The results published today will highlight the potential of enzyme replacement therapy for MPS-1 patients,” said Emil Kakkis, M.D., principal investigator and vice president of Biomarin.



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