Genzyme Press Release (April 3, 2006)
Product is First Treatment for Pompe Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that it has received marketing authorization for Myozyme® (alglucosidase alfa) in the European Union. Myozyme has been approved for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Pompe disease, a debilitating, progressive and often fatal disorder affecting fewer than 10,000 people worldwide. The product is the first treatment ever approved for Pompe disease and one of the first for an inherited muscle disorder.
“This is an extraordinary moment for Pompe patients and their families,” said Henri A. Termeer, chairman and chief executive officer of Genzyme Corp. “The effort to develop Myozyme has required the enormous commitment of many people throughout Genzyme and across the Pompe community, who have worked with a great sense of urgency and have overcome tremendous challenges. Our focus now is to ensure that Myozyme is available to all patients who need treatment.”
Myozyme has received orphan medicinal product designation in Europe. The orphan medicinal products regulation is designed to encourage the development of treatments for rare disorders such as Pompe disease, for which no therapies have existed previously. Genzyme will introduce Myozyme in Europe on a country-by-country basis, as pricing and reimbursement approvals are obtained.
Ria Broekgaarden, of the Dutch Pompe patient organization VSN (Vereniging Spierziekten Nederland) and secretary of the International Pompe Association, said, “For people with Pompe disease, this is a very important moment in history. The approval of this treatment represents great hope and progress for all Pompe patients, which in turn will give them a new perspective on their future.”
Pompe disease manifests as a broad spectrum of clinical symptoms. All patients typically experience progressive muscle weakness and breathing difficulty, but the rate of disease progression can vary widely depending on the age of onset and the extent of organ involvement. When symptoms appear within a few months of birth, babies frequently display a markedly enlarged heart and die within the first year of life. When symptoms appear during childhood, adolescence or adulthood, patients may experience steadily progressive debilitation and premature mortality due to respiratory failure. They often require mechanical ventilation to assist with breathing and wheelchairs to assist with mobility.
Genzyme began working to develop a treatment for Pompe disease in 1998. In 2003, the company initiated clinical studies of Myozyme, which produced highly encouraging results and formed the basis of the company’s regulatory submissions. In the pivotal clinical study, 83 percent of patients treated with Myozyme were both alive and free of invasive ventilator support at 18 months of age, compared with two percent of patients in the historical cohort. The trial, which met its primary endpoint, enrolled 18 patients with infantile-onset Pompe disease, who began receiving therapy at approximately six months of age. Approximately 39 percent of patients treated with Myozyme developed infusion associated reactions, which were mostly mild to moderate in nature. Two patients experienced serious infusion reactions. One experienced urticaria, the other experienced urticaria, rales, tachycardia, decreased oxygen saturation, bronchospasm, tachypnea, periorbital edema and hypertension.
Genzyme recently completed enrollment in its clinical trial involving patients with late-onset Pompe disease. Ninety patients are participating in this international, placebo-controlled study. Currently, more than 270 patients in 30 countries are receiving Myozyme through clinical trials, expanded access programs, or pre-approval regulatory mechanisms.
Genzyme manufactures Myozyme at two facilities in the United States. To ensure that it is able to meet the anticipated demand for the product in Europe and throughout the world, the company expects to also produce Myozyme in the future at its new protein manufacturing facility in Geel, Belgium, and its new fill/finish facility in Waterford, Ireland…