Pompe Program Update

Category: Archive
July 16, 2005

Steady progress is being made in all areas of the Genzyme Pompe program and the goal of obtaining worldwide regulatory approval for the investigational rhGAA enzyme remains one of Genzyme’s highest priorities.

The screening study for late-onset Pompe patients has begun at sites in the US and the Late-Onset Treatment Study (LOTS) is still on-schedule for initiation in the third quarter of 2005 (August-September). In addition, Genzyme has “re-opened” the Late-Onset Expanded Access Program. This program will be open to a limited number of patients world-wide, and the process of identifying and enrolling patients has begun. At this time there are over 130 total patients receiving the investigational enzyme through clinical studies and expanded access programs. .

The submission for regulatory approval of investigational enzyme (rhGAA) in Europe is currently under review by the authorities there (European Agency for the Evaluation of Medicinal Products or EMEA). Genzyme will be working closely with the authorities in Europe during this time and it is anticipated that there will be a response to the submission in early 2006.

The regulatory submission to the FDA in the US (Biologics License Application or BLA) is still on track for mid-2005 and Genzyme will issue a press release when this event occurs.

Back to news articles