US Pompe Program Update – December 3, 2009
Genzyme would like to provide an update to the US Pompe community about the
status of the Alglucosidase Alfa Temporary Access Program (ATAP) and the
application for Lumizyme™ (alglucosidase alfa) approval in the United States.
In a press release dated December 3, 2009, it was announced that Genzyme will
reopen enrollment in ATAP to provide access to treatment for severely affected adults
with Pompe disease prior to commercial approval of Lumizyme. Enrollment in the
program will be available at currently active ATAP study sites to patients who meet the
eligibility criteria. Please speak to your physician about ATAP or contact Genzyme
Medical Information at 800-745-4447, option 2 for more information.
Genzyme and the Food and Drug Administration (FDA) also have agreed on a path
toward approval of Lumizyme. In November, Genzyme received a Complete Response
letter for its Biologic License Application (BLA) for Lumizyme. Genzyme had been
seeking approval of Lumizyme produced at a 2000 L bioreactor scale, manufactured at
its Allston plant. In its letter, the FDA stated that satisfactory resolution of deficiencies
related to the Allston plant would be required before the Lumizyme application can be
approved. Since receiving the Complete Response letter, Genzyme has had several
discussions with the FDA on the most expeditious path toward approval. Based on
these discussions, Genzyme will now seek approval of the product produced at a
larger 4000 L bioreactor scale, in a resubmission to the agency. The 4000 L-scale
product is produced at Genzyme’s manufacturing plant in Geel, Belgium. Genzyme
anticipates that the resubmission will be made this month.
Genzyme continues to produce Myozyme® (alglucosidase alfa) at the 160 L scale and
this product is reserved for patients in the US who are 17 years old and younger.
We will continue to keep you informed of relevant updates as information becomes
available. We appreciate your ongoing support as we continue to work to provide
access to treatment for the full Pompe community.