A U.S. Food and Drugs Administration has approved Cerdelga, a drug to treated adult patients suffering from a form of Gaucher disease.
The drug is a gelatin capsule that contains eliglustat and should be consumed orally. Cerdelga works by slowing down the production of fatting material in patients with Gaucher disease Type 1. It inhibits the metabolic process that forms these materials.
"Today's approval offers another important treatment option for patients with Type 1 Gaucher disease," said Amy G. Egan, M.D., M.P.H., deputy director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research, in a press statement. "In addition, Cerdelga received orphan drug designation from the FDA, reflecting the agency's focus and commitment to the development of treatments for rare diseases."
The effectiveness and safety of the drug was tested in two clinical trials involving 199 patients with the disease. In one trial, 40 patients received the drug at a starting dose of 42 mg two times a day. Some of them started receiving a dose of 84 mg two times a day after four weeks. Study participants continued the drug for nine months.
Compared to those treated with a placebo, those that were given the drug experienced a significant reduction in spleen volume from baseline to the end of the study. They also had better liver volume, blood platelet count, and red blood cell (hemoglobin) levels.
In the other trial, 159 participants with Type 1 Gaucher disease received either the enzyme replacement therapy drug imiglucerase or Cerdelga. Similar observations were made in this trial too.
The side effects of the drug include fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.
Gaucher disease is a lipid storage disease characterized by the deposition of glucocerebroside in cells of the macrophage-monocyte system. The disorder results from the deficiency of a specific lysosomal hydrolase and is characterized by a continuum of phenotypes. The severity widely varies; some patients present in childhood with virtually all the complications of Gaucher disease, whereas others remain asymptomatic into the eighth decade of life. According to national statistics, approximately 1 in 50,000 or 0.00 percent or 5,440 people in the United States suffer from this disease.
Cerdelga is manufactured by Cambridge, Massachusetts-based Genzyme
