Cerdelga (eliglustat) pills are as effective and safe as the injectable drug Cerezyme (imiglucerase) as a maintenance therapy for Gaucher disease after stabilizing patients on Cerezyme, according to the Phase 3 ENCORE clinical trial.
The results of the four-year study were reported in the article, “Eliglustat Maintains Long-term Clinical Stability in Patients with Gaucher Disease Type 1 Stabilized on Enzyme Therapy,” published in the journal Blood.
Both Cerdelga and Cerezyme are approved by the U.S. Food and Drug Administration (FDA) to treat Gaucher disease, and both are manufactured by Genzyme.
Gaucher disease type 1 is an inherited, systemic disease that commonly causes enlargement of the liver and spleen (hepatosplenomegaly), low iron in the blood (anemia), low platelet counts in the blood, and bone disease. It is caused by the accumulation of a naturally occurring substance called glucosylceramide in a type of immune cell called macrophages.
Cerezyme is an injectable drug consisting of a biological substance that breaks down glucosylceramide. It’s an enzyme replacement therapy, or ERT. Cerdelga is a chemical in a pill form that inhibits the manufacture of glucosylceramide by the body, called substrate reduction therapy. Cerezyme has been used to treat Gaucher disease type 1 since the 1990s, while Cerdelga was approved by the FDA in 2014.
The ENCORE trial (NCT00943111) included 157 patients with Gaucher disease type 1 who had been stabilized with Cerezyme treatment for one year and were then placed on maintenance therapy with Cerdelga for up to four years (46 patients took it for four years). Four patients withdrew from the study due to adverse events.
“Eliglustat treatment resulted in stable mean hemoglobin concentration, platelet count, and spleen and liver volumes for up to 4 years,” the article reported. “Mean bone mineral density Z-scores [a measure of bone density] also remained stable and were maintained in the healthy reference range for up to 4 years.”
Patients’ blood, liver and spleen parameters were stable in 85% or more cases for up to four years, and 92% or more patients achieved acceptable values (established therapeutic goals) in liver, spleen, and blood tests for the four years the trial lasted.
“We conclude that clinical stability, judged by composite and individual measures, was maintained in eliglustat-treated patients with Gaucher disease type 1 who remained in the ENCORE trial for up to 4 years. Eliglustat was generally well-tolerated and no new or long-term safety concerns were identified,” the authors wrote.