Long-term treatment with Cerdelga (eliglustat), for Gaucher disease type 1, is found to be consistently safe. Results of an eight years study proved it to be efficient in causing continuous improvement of various blood parameters, decrease volumes of spleen and liver, and reduce disease activity markers. The study was published in the American Journal of Hematology with the title of “Outcomes after 8 Years of Eliglustat Therapy for Gaucher Disease Type 1: Final Results from the Phase 2 Trial.”
Cerdelga decreases the quantity of glucosylceramide, a fatty substance that accumulates primarily in the cells of the bone marrow, spleen, and liver of patients suffering with Gaucher disease. A number of countries, including the U.S. and the European Union, have approved Cerdelga as a long-term treatment for the 90% of adults with Gaucher disease type 1 who do not have ultra-rapid CYP2D6 enzyme, a type of liver enzyme that metabolizes Cerdelga. Cerdelga has been studied in multiple clinical trials, involving previously untreated patients and those patients who had received enzyme replacement therapy in the past. Current clinical data represent the longest eliglustat therapy result to date.
Complete report can be read here