Genzyme Reports Interim Results from Pivotal Study of Myozyme(R)
CAMBRIDGE, Mass., April 26 /PRNewswire-FirstCall/ -- Genzyme Corp. (Nasdaq: GENZ - News) announced today that it has completed a planned analysis of interim data from its pivotal clinical trial of Myozyme® (alglucosidase alfa), which is being studied for the treatment of Pompe disease. The interim analysis was included in the trial's protocol to allow for the potential expedited submission of a biologics license application. It found that the trial has already met one of its key secondary efficacy endpoints and that there is a high probability the study will meet its primary efficacy endpoint upon completion.
"The results are extremely encouraging and confirm our plan to submit a BLA in the middle of this year," said Richard A. Moscicki, senior vice president and chief medical officer for Genzyme Corp. "This analysis will allow us to seek U.S. approval for Myozyme as quickly as we had expected and to supplement our previously filed European marketing application with data from the pivotal study. We have proceeded with a great sense of urgency throughout the development of Myozyme, given the devastating nature of Pompe disease."
The pivotal trial, known as AGLU01602, includes 18 patients with infantile-onset Pompe disease. These patients were enrolled in the trial and began receiving Myozyme by 6 months of age. Because of the rapidly progressive and fatal nature of infantile-onset Pompe disease, outcomes for these patients are being compared with a matched historical cohort rather than a placebo cohort.
The study's primary endpoint is the proportion of patients treated with Myozyme who are alive and free of invasive ventilator support at 18 months of age, compared with the proportion of patients who were alive at 18 months of age in the historical cohort (2 percent). Results for the primary endpoint will be known this summer, when patients will have completed 52 weeks of treatment.
By 12 months of age, 89 percent of patients treated with Myozyme (16 of 18) were alive and free of invasive ventilator support compared with 17 percent of patients who were alive at 12 months of age in the historical cohort. This result meets a secondary efficacy endpoint and indicates the trial will very likely meet its primary endpoint.
The interim analysis also found the following:
* All patients treated with Myozyme showed a reversal in cardiomyopathy, a
condition in which the heart muscle becomes enlarged and heart function
is impaired. This reversal was measured by decreases in left
ventricular mass index from baseline.
* 72 percent of patients treated with Myozyme demonstrated gains in motor
development as measured by the Alberta Infant Motor Scale.
* All patients evaluated demonstrated gains in cognitive, language and
personal/social skills from baseline.
* 83 percent of patients developed antibodies to Myozyme and 44 percent
experienced infusion associated reactions.
Genzyme will submit data from study AGLU01602 to the European Medicines Agency, which is reviewing a marketing authorization application (MAA) for Myozyme filed in December 2004. The agency's Committee for Human Medicinal Products is expected to make a decision on the application later this year. The MAA contains data from other studies, including AGLU01702, which enrolled patients with infantile-onset Pompe disease who were older than those in AGLU01602 and whose disease was more advanced.
Genzyme is pursuing approval for Myozyme's use as a long-term enzyme replacement therapy for all patients with a confirmed diagnosis of Pompe disease, defined as acid alpha-glucosidase deficiency. There is currently no approved treatment for the disease. More than 100 patients are now receiving Myozyme in clinical studies, through Genzyme's expanded access program, or through pre-approval mechanisms sponsored by governments in several European countries. The Myozyme program is Genzyme's largest research and development initiative.
About Pompe disease
Pompe disease is an inherited, progressive muscle disease that affects fewer than 10,000 people worldwide. The disease is caused by a deficiency of an enzyme known as acid alpha-glucosidase. This deficiency leads to the excessive accumulation of glycogen in the body, particularly in the muscles. Pompe disease manifests as a broad spectrum of clinical symptoms with varying rates of disease progression. Infantile-onset patients present in the first months of life with an enlarged heart and skeletal and respiratory muscles weakness, and most die from cardiac or respiratory complications by one year of age. Late-onset patients may present with muscle or respiratory weakness anytime during childhood or adulthood, and disease progression is less rapid. Late-onset patients often require mechanical ventilation for breathing assistance and mobility aids such as canes, walkers or wheelchairs. Late- onset patients will experience a shortened lifespan due to progressive respiratory failure. Pompe disease belongs to a family of approximately 40 rare inherited diseases known as lysosomal storage disorders.... |
