The U.S. Food and Drug Administration today approved Elelyso (taliglucerase alfa) for long-term enzyme replacement therapy to treat a form of Gaucher disease
, a rare genetic disorder
Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and other organs. The major signs of Gaucher disease include liver or spleen damage, low red blood cell counts (anemia), low blood platelet counts, and bone problems.
Elelyso is an injection that replaces the missing enzyme in patients with a confirmed diagnosis of Type 1 (non-neuropathic) Gaucher disease and should be administered by a health care professional every other week. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.
"Today's approval provides for a new enzyme replacement therapy for the select number of patients with Type 1 Gaucher disease," said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDA's Center for Drug Evaluation and Research. "It also demonstrates FDA's commitment to developing treatments for rare diseases."
Due to the small number of affected patients, the efficacy of Elelyso was evaluated in a total of 56 patients with Type 1 Gaucher disease enrolled in two clinical trials. Many of these patients continued treatment on a longer-term extension study.
In one multi-center, double-blind, parallel-dose trial, the efficacy of Elelyso for use as an initial therapy was evaluated in 31 adult patients who had not previously received enzyme replacement therapy. Patients were randomly selected to receive Elelyso at a dose of either 30 units per kilogram or 60 units/kg.
At both doses, Elelyso was effective in reducing spleen volume, the study's primary endpoint, from baseline by an average of 29 percent in patients receiving the 30 units/kg dose and by an average of 40 percent in patients receiving the 60 units/kg dose after nine months of treatment. Improvements in liver volume, blood platelet counts, and red blood cell (hemoglobin) levels also were observed.