Updated Phase III Aldurazyme data

BioMarin (BMRN; SWX:BMRN) and partner Genzyme (GENZ) updated data on the primary endpoints - pulmonary function as measured by forced vital capacity (FVC) , and six-minute walk test - from an ongoing open-label extension of a placebo-controlled Phase III study of Aldurazyme laronidase enzyme replacement therapy in 45 mucopolysaccharidosis I (MPS I) patients. In 22 patients who received Aldurazyme for 62 weeks during the trial and extension study, forced vital capacity (FVC) scores improved 5.4% points compared to baseline. The same patients had a mean 40-meter improvement in their six-minute walk tests.

In the 23 patients who received placebo during the placebo-controlled portion of the trial, FVC scores improved 2.6% points during the extension compared to baseline (pre-initiation of Aldurazyme treatment). The same patients had a mean 32.4 meter improvement in their six-minute walk tests. The most common adverse events were headache, rhinitis and pharyngitis. ...