FDA Approves Genzyme’s Lumizyme

Genzyme received marketing approval from the FDA for Lumizyme (alglucosidase alfa), produced at the 4000 L bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the first treatment approved in the U.S. to treat patients with late-onset Pompe disease. Lumizyme is a lysosomal glycogen-specific enzyme for patients eight years and older with late (non-infantile) onset Pompe disease who do not have evidence of cardiac hypertrophy.

“This is an important day for the Pompe community, especially for those patients with late-onset Pompe disease in the U.S. who are awaiting treatment for this devastating disease,” said Genzyme chairman and chief executive officer, Henri A. Termeer. “We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date.”

A Risk Evaluation and Mitigation Strategy (REMS) called the Lumizyme ACE (alglucosidase alfa control and education) Program will be implemented for Lumizyme to ensure appropriate use for the intended patient populations. Genzyme will begin this process immediately to certify and enroll prescribers and healthcare facilities and to help prescribers to enroll all patients that they intend to treat with Lumizyme.