Biotech Tracker News - Aldurazyme Should be Approved
November 25, 2002 BioMarin (NASDAQ: BMRN) and Genzyme’s (NASDAQ: GENZ) Aldurazyme (laronidase) has generated additional data demonstrating its utility in the clinical management of mucopolysaccharidosis I (MPS I). Our modeling estimates a >90% probability that Aldurazyme will be approved by the FDA. Aldurazyme will be reviewed by the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee on January 15, 2003.  Data from an ongoing, open-label, phase III extension study showed utility for the endpoints of pulmonary function as measured by percent predicted Forced Vital Capacity (FVC) and endurance as measured by distance covered in a six-minute walk test. The data were generated 36 weeks after the initiation of the open-label extension phase and 62 weeks after initiation of the inciting placebo-controlled trial. The data have been submitted to the FDA for review as part of Aldurazyme’s BLA dossier originally submitted on July 26, 2002. The data will also be submitted to European regulators.  62-week data for 22 Aldurazyme recipients demonstrated:  improved mean FVC change – +5.4 percentage points  improved six-minute walk test – mean +40.0 meter change  maintenance of FVC and six-minute walk test improvements observed after 50 weeks.  36-week data for the 23 placebo recipients switched to Aldurazyme for open-label investigation demonstrated:  improved mean FVC change – +2.6 percentage points; the bulk of was seen between weeks 25 and 36 of open-label investigation  improved six-minute walk test – mean +32.4 meter change.  Aldurazyme’s adverse effect profile appeared reassuring and predictable. One previously reported extension study death is still considered unrelated to Aldurazyme administration.
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