Genzyme Corp. recently announced that it has initiated a clinical trial evaluating the safety and efficacy of Myozyme® (alglucosidase alfa) in patients with late-onset Pompe disease. The trial is designed to provide additional support for Myozyme’s use.
Because a treatment for Pompe disease is urgently needed, 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.
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(R) (laronidase), which was approved for the treatment of MPS I disease in 2003.
“Conducting this study is consistent with our commitment to confirm the safety and efficacy of Myozyme for patients across the spectrum of Pompe disease,” said Richard A. Moscicki, senior vice president and chief medical officer for Genzyme. “Our approach has been to pursue approval for Myozyme as soon as possible, given the tremendous medical need. At the same time, we have worked to put in place a broad, supportive program that includes natural history studies, a disease registry, an expanded access program and additional clinical research.”
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.