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Late-onset Pompe disease drug goes on trial


18 September,2005: Genzyme Corp announced that it has initiated a clinical trial evaluating the safety and efficacy of alglucosidase alfa (Myozyme) in patients with late-onset Pompe disease. The trial is designed to provide additional support for Myozyme's use. 

Genzyme has already submitted European and U.S. marketing applications for Myozyme. Regulatory action on these applications is expected in early 2006. Genzyme anticipates submitting marketing applications for Myozyme in Japan and several other countries by the end of this year, the company said in a press release.

If approved, Myozyme will be the first treatment developed for patients with Pompe disease, a debilitating and often fatal neuromuscular disorder affecting fewer than 10,000 people worldwide. Patients with Pompe disease display a range of symptoms that correspond with the age of disease onset and the rate of disease progression. Progressive weakness in the muscles used for mobility, breathing and digestion are the most common symptoms. In infants, heart muscle is typically severely affected. 

The late-onset trial is a randomized, double-blind, placebo-controlled study that will enroll at least 72 patients. It is being conducted at five sites in the United States and two in Europe. Participants will be assigned to receive intravenous infusions of either Myozyme at a dose of 20 mg/kg or a placebo every other week for 52 weeks. Approximately two-thirds of participants will receive Myozyme. The study has two primary efficacy endpoints: (1) to determine the effect of Myozyme on functional endurance as measured by the Six-Minute Walk Test; and (2) to determine the effect of Myozyme on respiratory muscle weakness as measured by Forced Vital Capacity. These endpoints are the same as those used in the pivotal clinical trial of Aldurazyme (laronidase), which was approved for the treatment of MPS I disease in 2003.

Genzyme recently completed an observational study involving approximately 60 patients with late-onset Pompe disease. The study was designed to evaluate endpoints for the treatment study. Genzyme anticipates that many of the patients from the observational study may meet the entry criteria for the treatment study. 

Pompe disease is one of more than 40 genetic diseases called lysosomal storage disorders, which are caused by a deficiency or malfunction of specific enzymes found in cell lysosomes. Pompe disease manifests as a broad spectrum of clinical symptoms with varying rates of disease progression, typically divided into two broad classifications:

Infantile-onset Pompe disease: Babies who present with symptoms in the first few months of life are typically considered to have this form of Pompe disease. Without treatment, an infant may become seriously ill and die from cardiac or respiratory complications within the first year of life. Common symptoms of infantile-onset Pompe disease include severe and rapidly progressive muscle weakness, enlarged heart, respiratory insufficiency and frequent respiratory infections, cardiac complications, delayed development and feeding problems. About one-third of people with Pompe disease have infantile-onset Pompe.

Late-onset Pompe disease: Late-onset patients may present with muscle or respiratory weakness anytime during childhood or adulthood, and disease progression is less rapid but steady. These patients will experience a shortened life-span due to progressive respiratory failure and often require mechanical ventilation to assist with breathing as well as mobility aids such as canes, walkers or wheelchairs. Common symptoms of late-onset Pompe disease include progressive muscle weakness in the legs and hips; gradual loss of ability to walk, run or jump; respiratory problems; morning headaches; scoliosis; daytime sleepiness; and difficulty gaining or maintaining weight. About two-thirds of people with Pompe disease have the late-onset form of the disease.

Founded in 1981, Genzyme has grown from a small start-up to a diversified enterprise with 2004 revenues of $2.2 billion and more than 7,600 employees in locations spanning the globe. With many established products in more than 80 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune diseases, and diagnostic testing.  



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