CAMBRIDGE, Mass. — An experimental drug for treating a rare genetic disease appears effective, according to a late-stage clinical trial.
Sanofi subsidiary Genzyme said Tuesday that the drug eliglustat tartrate was effective in improving spleen size in previously untreated patients with Type 1 Gaucher disease in the phase-3 "Engage" trial. Gaucher disease is an enzyme deficiency disorder that affects fewer than 10,000 people worldwide and causes fat cells to collect around the spleen, liver and bone marrow. Patients with the disease may experience enlargement of the spleen and liver, anemia, excessive bleeding and bruising, bone disease and other symptoms.
"The efficacy and safety data from our Engage trial are consistent with what were observed in our phase-2 study, continuing to suggest that eliglustat tartrate is a potent, well-tolerated oral compound that may become a meaningful option for patients and physicians," Genzyme president and CEO David Meeker said. "The development of eliglustat tartrate has been underway for more than a decade and is the largest clinical program ever focused on Gaucher disease, demonstrating our ongoing commitment to innovation on behalf of this community."
The company has enrolled more than 370 patients for various studies of the drug. Top-line data from the second phase-3 trial, "Encore," are expected early next year.