Genzyme's development program for Lumizyme, a therapy to treat Pompe disease, appears to be back on track. The FDA had rejected Genzyme's BLA for Lumizyme in November, saying that the biotech company needed to fix problems at its Allston plant before it could make the therapy in Massachusetts. Based on follow-up talks with the FDA, Genzyme will now seek approval of the product produced at a larger 4000 L bioreactor scale produced at Genzyme's manufacturing plant in Geel, Belgium. Genzyme says it will resubmit its BLA this month.
Genzyme also says that it is reopening its temporary access program to provide the therapy to patients ahead of an approval. "The U.S. patient community has been very supportive," says CEO Henri A. Termeer, "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 the 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 the program. The ATAP program will remain open until commercial approval of Lumizyme, says Genzyme.
Production at the Allston plant had been halted after regulators discovered it had been contaminated, triggering a staggering set of problems for Genzyme. But on Tuesday morning the biotech announced that it had resumed some shipments of Cerezyme from the facility.
- here's the Genzyme release