Health Canada has approved Cerdelga (eliglustat capsules), manufactured by Sanofi Genzyme, for treating Gaucher disease type 1 (GD1) — three years after the U.S. Food and Drug Administration gave its blessing to the drug.
The company says Cerdelga is currently the only first-line oral therapy for patients who are specific enzyme metabolizers, as detected by a simple test.
Gaucher disease is a rare inherited genetic disorder in which fatty molecules accumulate in certain parts of the body. GD results from a missing or deficient enzyme called glucocerebrosidase that normally breaks down waste material within cells. Consequent buildup of fatty waste material called glucosylceramide in the body’s organs and tissues can cause a wide range of symptoms including liver and spleen enlargement (hepatosplenomegaly), anemia; easy bruising caused by a decrease in blood platelets (thrombocytopenia), chronic fatigue, lung disease, and bone disease.
Cerdelga’s approval will “significantly improve” the quality of life of GD1 patients “due to the elimination of burdensome biweekly intravenous infusions,” Christine White, president of the National Gaucher Foundation of Canada , said in a press release.
Sanofi Genzyme said that current GD1 therapies — enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) — aim to reduce levels of glucosylceramide (GL-1), a fatty substance found in cells, and thereby slow the onset of symptoms. Cerdelga, a SRT, decreases GL-1 production by blocking absorption of materials cells use to make GL-1.
The company said Cerdelga was evaluated in a clinical trial involving almost 400 patients, as well as two Phase 3 clinical studies. Investigators found that in untreated patients with GD1, Cerdelga improved platelet and red blood cell levels, spleen and liver volumes, and bone outcomes. Most side effects caused by Cerdelga were mild to moderate in severity, it said.
“The approval of an oral therapy for Gaucher disease is a welcome addition to patients,” said Dr. Aneal Khan of Alberta Children’s Hospital in Calgary. “Having an oral therapy that is well-tolerated and works on the various aspects of Gaucher disease will make therapy easier. However, it is important that we still have ERT as a suitable option for patients.”
Sanofi Genzyme says it has been researching GD oral therapy for 15 years and claims its Cerdelga clinical development program is the largest ever conducted for GD. Peter Brenders, general manager of Sanofi Genzyme Canada, said the drug’s approval is a “significant milestone” for the company.