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First Therapy Ever Approved in Canada for Ultra-Rare Form of Muscular Dystrophy Health Canada approves Myozyme® for the treatment of Pompe Disease MISSISSAUGA, ON, CANADA -- December 8, 2006 -- Following the August 2006 approval by Health Canada, Genzyme Canada today announced the availability of Myozyme® (alglucosidase alfa), the first treatment approved for Pompe disease and the first for an inherited muscle disorder in the family of diseases known as muscular dystrophy. Pompe disease is a rare, progressive, debilitating and often fatal neuromuscular disorder. Patient numbers are estimated at fewer than 100 Canadians and 10,000 people worldwide.1 "For myself and other Canadians with Pompe disease, and for our families, Myozyme enables us to fight back against this terrible disease that literally saps your muscle strength and sucks away your very breath," said Guy Ashford-Smith, President of the Canadian Association of Pompe.1 Myozyme is approved in Canada for use in individuals with Pompe disease (GAA deficiency) and is given as an intravenous infusion once every two weeks. Myozyme has been shown to improve ventilator-free survival in individuals with infantile-onset Pompe disease as compared to untreated historical controls, whereas further studies are currently being conducted to evaluate the safety and efficacy of Myozyme in patients with other forms of Pompe disease. Because of the potential for severe infusion reactions, appropriate medical support measures should be readily available when Myozyme is administered. "As the first treatment for this rare and deadly form of muscular dystrophy, Myozyme is opening a door to a new era in treatment for patients," said Dr. Robin Casey, Director of Inherited Metabolic Disorders Program, Department of Medical Genetics and Pediatrics, Alberta Children's Hospital. "Prior to Myozyme, as physicians we were only able to offer our patients palliative care to treat the symptoms of Pompe, a disease that takes a terrible toll on patients and their families." Pompe disease, also known as Acid Maltase Deficiency or Glycogen Storage Disease Type II, is caused by a deficiency in the enzyme acid alpha-glucosidase (GAA), which is needed to break down glycogen, a stored form of sugar used for energy. The build-up of glycogen causes progressive muscle weakness throughout the body with patients presenting with musculoskeletal, respiratory, and cardiac manifestations of the disease. Individuals who are the most severely affected by Pompe disease will have symptoms by two to three months of age.2 Disease progression is rapid and these infants rarely live beyond one year of age. For others with Pompe disease, the disease onset may occur in childhood or adolescence, or later as an adult. The longer an individual with Pompe disease has symptoms, the earlier they will become wheelchair or ventilator dependent.3 Symptoms vary depending on the severity of the disease and the age of symptom onset. Infants exhibit a severe lack of muscle tone, often characterized as "floppy baby" syndrome, their heart is also affected and usually becomes enlarged – up to three times its normal size – and they usually die from cardiac or respiratory complications before one year of age. In older patients, symptoms are typically generalized muscle weakness, including skeletal, which may require the need for a wheelchair and other assistive devices, and respiratory, which may require mechanical ventilation. Basis of Approval(4) A pivotal randomized, multi-centre clinical study (AGLU01602) of Myozyme demonstrated the product's safety and efficacy. Initiated in 2003, the trial enrolled 18 patients with infantile-onset Pompe disease, who began receiving therapy at approximately six months of age. In the study, 83 per cent of individuals treated with Myozyme were both alive and free of invasive ventilator support at 18 months of age. By comparison, only 2 per cent of individuals in an untreated historical group were alive at 18 months.5 About Myozyme The development of Myozyme from recombinant DNA technology is a process that takes many months in specially built bio-reactors. Genetically modified cells are grown in large tanks over several months. Kept in constant motion, the cells multiply in this living production facility and begin to express the recombinant human protein. Liquid is drawn off daily and the enzyme is collected for a multi-stage purification process to isolate and purify the desired protein. Genzyme began developing a treatment for Pompe disease in 1998. Today, more than 450 individuals in 30 countries are being treated with Myozyme through clinical trials, expanded access programs, charitable access programs, pre-approval regulatory mechanisms, or commercially.6 About the Pompe Disease Registry To further global understanding and awareness of Pompe disease, Genzyme created the Pompe Disease Registry, which documents the natural course of the disease, significant clinical outcomes, and disease management practices. More than 150 physicians and healthcare associates from around the world actively participate in the Pompe Registry, which has enrolled more than 250 people with Pompe disease. Myozyme is the fourth enzyme replacement therapy developed by Genzyme for a rare genetic disease. Genzyme has developed PrFabrazyme® (agalsidase beta) for Fabry disease, PrCerezyme® (imiglucerase) for Type 1 and Type 3 Gaucher disease and, in collaboration with BioMarin Pharmaceutical Inc., PrAldurazyme® (laronidase) for MPSI. REFERENCES: 1. Acid Maltase Deficiency Association (AMDA). Accessed November 30, 2006. 2. www.pompecanada.com http://rarediseases.about.com/cs/pompedisease/a/071903.htm 3. Disease severity in children and adults with Pompe disease related to age and disease duration, M.L.C. Hagemans, Neurology 2005;64:2139–2141 4. Study # AGLU01602; Randomized, Open-label, Multicenter, Safety, Efficacy, Pharmacokinetic and pharmacodynamic, Dose Ranging Study of Myozyme in 18 Pompe patients with a mean age of 4.6 months. 5. Kishnani P, Corzo D, Nicolino M, et al. Recombinant human acid a-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2006 (in press). 6. Myozyme Producet Monograph. SOURCE: Genzyme Canada E-mail this page to a friend or colleague! To print, use this version