Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has granted U.S. marketing approval for Lumizyme™ (alglucosidase alfa), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the first treatment approved in the United States specifically to treat patients with late-onset Pompe disease.
“We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date.”
"This is an important day for the Pompe community, especially for those patients with late-onset Pompe disease in the United States 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."
Lumizyme (alglucosidase alfa) is a lysosomal glycogen-specific enzyme indicated for patients 8 years and older with late (non-infantile) onset Pompe disease (GAA deficiency) who do not have evidence of cardiac hypertrophy. The safety and efficacy of Lumizyme have not been evaluated in controlled clinical trials in infantile-onset patients, or in late (non-infantile) onset patients less than 8 years of age.
Genzyme began work on a therapy for Pompe disease ten years ago, and the company has invested nearly $1 billion to support the development program. In 2006, Genzyme received approval for Myozyme® (alglucosidase alfa) in Europe and in other countries outside of the United States manufactured at a 2000 L bioreactor scale and indicated to treat all patients with Pompe disease. At this time, Genzyme also received FDA approval for Myozyme manufactured at a smaller 160 L bioreactor scale in the United States, which, because of its limited capacity, has been reserved for children and infants in the United States. In 2009, Genzyme received approval outside of the United States for manufacturing Myozyme in 4000 L bioreactors at its state-of-the-art manufacturing facility in Geel, Belgium, and began to transition patients globally to the product manufactured at this larger scale. To prepare for growing demand for alglucosidase alfa, Genzyme has installed a third 4000 L bioreactor in Geel with an anticipated approval in 2011.
Genzyme has worked closely with patients and physicians in the U.S. Pompe community during the preapproval period to assure that the most severely affected late-onset patients could access therapy in advance of Lumizyme approval. In May 2007, Genzyme began providing alglucosidase alfa free-of-charge to patients in the United States through a program known as the Alglucosidase Alfa Temporary Access Program (ATAP). Nearly 200 severely affected adults in the United States with Pompe disease are currently receiving treatment under the ATAP program. Genzyme will now work closely with the treating centers and prescribers to insure that patients in the ATAP program can continue to access therapy during the transition to commercial supply. Genzyme will also begin working with U.S. healthcare professionals to help adult patients who have been waiting to access treatment. In effort to preserve 160-L scale product for infantile-onset patients, Genzyme will begin to transition eligible patients who are receiving Myozyme onto Lumizyme.
Because Genzyme will market two approved alglucosidase alfa products in the United States, a Risk Evaluation and Mitigation Strategy called the Lumizyme ACE (alglucosidase alfa control and education) Program will be implemented for Lumizyme to ensure appropriate use for the intended patient populations. All prescribers of Lumizyme, and healthcare facilities where Lumizyme will be dispensed and administered, are required to be certified and enrolled in the Lumizyme ACE Program prior to treating patients with Lumizyme. Prescribers must also ensure patients enroll in the Lumizyme program prior to receiving therapy. 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.