BioMarin Pharmaceutical Inc and Genzyme General announced that the Committee for Proprietary Medicinal Products (CPMP) of the European Union has issued a positive opinion on the companies' marketing application for Aldurazyme (laronidase), an investigational enzyme replacement therapy for treatment of mucopolysaccharidosis I (MPS I), a rare, progressive and debilitating genetic disorder.

The positive CPMP opinion is the final step before formal approval to market Aldurazyme in the 15 countries of the European Union. The companies have been advised that the committee's opinion will be forwarded to the European Commission, which is expected to make a final decision on marketing authorization within three to four months. The Commission generally follows the advice of the CPMP, but it is not obliged to do so.

The CPMP is a scientific body made up of representatives from the 15 member states of the European Union, Iceland and Norway. The Committee reviews medicinal product applications on their scientific and clinical merit, and provides advice on their approval to the European Commission, including recommendations about product labeling. For Aldurazyme, the Committee proposed a label to indicate that Aldurazyme is approved as long-term enzyme replacement therapy in patients with a confirmed diagnosis of MPS I, to treat the non-neurological manifestations of the disease.

The CPMP reviewed the companies' clinical data for Aldurazyme, which were submitted in March 2002 and included safety and efficacy data from a Phase 1/2, 10-patient, open-label trial and extension data, from a Phase 3, 45- patient, randomized, double-blind, placebo controlled trial, as well as from the open-label extension portion of the Phase 3 trial.