BioMarin Pharmaceutical Inc and Genzyme General announced that the U.S. Food and Drug Administration (FDA) has accepted for review their Biologics License Application (BLA) filing for Aldurazyme, and has granted the application priority review status.
Priority review status specifies that the FDA will respond to the filing within six months from the date of the completed BLA filing, which was July 29, 2002. Priority review is an FDA procedure designed to accelerate the review of products that address an unmet medical need.
Aldurazyme is an investigational enzyme replacement therapy for patients with mucopolysaccharidosis I (MPS I), a progressive, debilitating and fatal genetic disease for which no specific drug treatments currently exist. BioMarin and Genzyme included clinical data from the six-month, placebo-controlled Phase 3 trial of Aldurazyme, six months of data from the ongoing open-label Phase 3 extension study, and three years of data from the Phase 1 trial and extension study.
On March 1, 2002, BioMarin and Genzyme also submitted a Marketing Authorization Application (MAA) to the European Agency for the Evaluation of Medicinal Products (EMEA) for approval to market Aldurazyme in the European Union. The Agency accepted the MAA and is currently reviewing the application. BioMarin and Genzyme expect a response from the EMEA in the first half of 2003.
MPS I is a progressive, debilitating and fatal genetic disease caused by a deficiency of the enzyme alpha-L-iduronidase. This deficiency leads to the accumulation of complex carbohydrates in the lysosomes of cells, leading to the progressive dysfunction of cellular, tissue and organ systems. Resulting symptoms, which span a spectrum of severity, can include impaired cardiac and pulmonary function, delayed physical development, skeletal and joint deformities, reduced endurance, and in some cases, delayed mental function. A majority of patients die before adulthood from complications of the disease.
BioMarin and Genzyme formed a joint venture in 1998 to develop and commercialize Aldurazyme worldwide. Under the terms of the joint venture, if approved for commercial sale, BioMarin will manufacture Aldurazyme and Genzyme will have responsibility for the commercialization of the product. The companies have obtained Orphan Drug designation and Fast Track status for Aldurazyme for the treatment of MPS I from the FDA and orphan medicinal product designation from the EMEA.