Genzyme Corporation (Nasdaq: GENZ) announced today that it will reopen enrollment in the Alglucosidase Alfa Temporary Access Program (ATAP), a program which provides access to treatment for severely affected adults with Pompe disease prior to commercial approval of Lumizyme™ (alglucosidase alfa). Genzyme and the FDA have also agreed on a path toward approval of Lumizyme.
In November, Genzyme received a Complete Response letter for its Biologic License Application (BLA) for Lumizyme. The company had been seeking approval of Lumizyme produced at a 2000 liter (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, the company 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 Lumizyme is produced at Genzyme’s manufacturing plant in Geel, Belgium. Genzyme anticipates that the resubmission will be made this month.
“The reopening of the ATAP program will enable more U.S. patients with Pompe disease access to treatment prior to commercial approval of Lumizyme,” said Genzyme Chairman and Chief Executive Officer Henri A. Termeer. “The U.S. patient community has been very supportive, and we will continue to work closely with the FDA to finalize our efforts and to bring this important therapy to the Pompe community.” The company has provided therapy free of charge to approximately 170 patients since 2007, and will work with the 81 active study sites in the U.S. to enroll additional patients into this program. The ATAP program will remain open until commercial approval of Lumizyme.
Lumizyme (alglucosidase alfa produced at the 4000 L bioreactor scale) is marketed outside of the United States as Myozyme®. Myozyme is commercially approved for the treatment of Pompe disease in over 40 countries worldwide.