americanpharmaceuticalreviewMay 02, 2017
Tag: Cerdelga , Health Canada , Sanofi Genzyme
Sanofi Genzyme, a division of Sanofi-Aventis Canada Inc., announced Health Canada has approved Cerdelga (eliglustat capsules) for the treatment of adult patients with Gaucher Disease Type 1.
A rare genetic metabolic condition that causes fats to build up in certain parts of the body, Gaucher Disease Type 1 is progressive and may become life-threatening if symptoms are left untreated. Patients who are specific enzyme metabolisers, as detected by a simple test, will be eligible for Cerdelga treatment.
"Patient experience with Cerdelga indicates that this new treatment has a beneficial effect on a number of symptoms associated with Gaucher Disease Type 1 and significantly improves quality-of-life due to the elimination of burdensome bi-weekly intravenous infusions," Christine White, President of the National Gaucher Foundation of Canada said. "We are very excited that this new first-line oral therapy is now available in Canada for people living with Gaucher Disease Type 1."
Current therapies for Gaucher Disease Type 1, either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), aim to reduce the amount of glucosylceramide (GL-1), a fatty substance found in cells, and diminish the progression of symptoms which can damage the spleen, liver and bones. Cerdelga, a SRT, decreases production of GL-1 by blocking the absorption of materials that are used by cells to make GL-1, thereby minimizing the effects of the disease.
Cerdelga has been evaluated in a clinical program for Gaucher Disease involving almost 400 patients and two Phase 3 clinical studies. In untreated patients with Gaucher Disease Type 1, Cerdelga produced improvements in platelet and hemoglobin levels, spleen and liver volumes, and bone outcomes. In patients whose disease had been stabilized with ERT, Cerdelga maintained stability of these disease parameters.
Gaucher Disease is a rare genetic metabolic condition caused by deficient activity of the enzyme glucocerebrosidase. The function of this enzyme is to break down waste material within cells. The waste material that builds up is a fatty substance called glucosylceramide (GL-1), which can lead to a spectrum of potentially life-threatening symptoms. These symptoms can vary widely among individuals and may include progressive spleen and liver enlargement, thrombocytopenia, bone pain and fractures, and ultimately impaired spleen and bone function.
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