Genzyme Pompe Program Update—November 10, 2008

The US Food and Drug Administration (FDA) held a meeting of its Endocrinologic and Metabolic Drugs Advisory Committee on October 21, 2008 to discuss Genzyme’s Biologics License Application (BLA) for alglucosidase alfa produced at the 2000 L bioreactor scale to treat late-onset Pompe disease in the United States. The Committee voted 16-1 that the Late Onset Treatment Study (LOTS) established the effectiveness of alglucosidase alfa produced at the 2000 L scale for the treatment of patients with late-onset disease.  The majority of the committee members recommended an ‘Accelerated Approval’, which would allow the 2000L to be approved but with the requirement to conduct a verification study of clinical benefit of therapy with alglucosidase alfa.  The committee also recommended that treatment with the 2000 L product should be made available to all patients with late-onset disease, without restrictions based on age.