FDA approves Aldurazyme

The FDA granted marketing approval for Aldurazyme laronidase to treat mucopolysaccharidosis I (MPS I) from BioMarin (BMRN; SWX:BMRN) and Genzyme (GENZ). The enzyme replacement therapy was approved to treat patients with Hurler and Hurler-Scheie forms of the disease, and for Scheie patients with moderate to severe symptoms. GENZ expects to launch the product in the coming weeks.

The companies' post-marketing commitments include completing an ongoing trial that is measuring the safety and pharmacokinetics of Aldurazyme in patients under five years old. The partners also will run an open-label dose optimization study in 32 patients, and will continue their Phase III extension study for four years from when the trial was started. GENZ and BMRN also plan to collect long-term Aldurazyme data via a registry. In January, FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously that the companies demonstrated meaningful treatment effects of Aldurazyme, and recommended that any safety concerns be addressed after approval (see BioCentury, Jan. 20, 2003). ...