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Aldurazyme Phase III MPS-1 data

November 2, 2001 8:00 AM UTC

BioMarin (BMRN) and partner Genzyme (GENZ) said preliminary analysis from a double-blind, placebo-controlled Phase III trial showed patients given Aldurazyme had a statistically significant increase in the primary endpoint of pulmonary capacity (p=0.028), but had a non-significant trend in the other primary endpoint of endurance, as measured by a six-minute walk test (p=0.066). Aldurazyme is a recombinant alpha-L-iduronidase enzyme to treat mucopolysaccharidosis-I (MPS-I), a genetic disorder leading to dysfunction of cellular tissue and organ systems. Following a meeting with the FDA in 1999, the partners said they would conduct the confirmatory Phase III study. ...