Sanofi’s Genzyme recently announced that treatment with its Cerdelga (eliglustat) reduced spleen size 28 percent compared to placebo in patients with Gaucher disease.
The company announced that results from the ENGAGE registration study evaluating Cerdelga in treatment-naïve patients with Gaucher disease type 1 were published in The Journal of the American Medical Association. According to Genzyme, treatment with the drug for nine months resulted in significant improvements in spleen volume, hemoglobin level, liver volume and platelet count compared to placebo.
The Phase III registration trial included 40 eligible treatment-naïve patients with Gaucher disease type 1 who had splenomegaly in addition to thrombocytopenia and/or anemia at study entry. Patients were stratified by baseline spleen volume and randomized to receive Cerdelga (50 or 100 mg twice daily) or placebo for nine months, following assessment for improvements in disease manifestations. The late-stage trial met its primary endpoint of a statistically significant reduction from baseline in spleen size, by a mean of 28 percent in Cerdelga patients. Secondary endpoints were also met, with Cerdelga associated with an increase in hemoglobin levels, decrease in liver volume and increase in platelet levels.
“We are very encouraged by these results as they ultimately point to a safe and effective oral treatment option for patients living with Gaucher disease,” said lead author Pramod Mistry, MD, PhD, FRCP, Professor of Pediatrics & Internal Medicine at Yale University School of Medicine.
Gaucher disease is an inherited condition, which affects fewer than 10,000 people worldwide. People with the disease do not have enough of the enzyme glucocerebrosidase, which breaks down a certain type of fat molecule. As a result, lipid engorged cells amass in different parts of the body, primarily the spleen, liver and bone marrow. The accumulation of these cells can cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and several other signs and symptoms.
Last August, US health regulators approved Cerdelga, the only first-line oral therapy for certain adult Gaucher disease type 1 patients. The drug is a novel glucosylceramide analog, designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide.