FDA Clears Eliglustat (Cerdelga) for Gaucher Disease


August 19, 2014

The US Food and Drug Administration (FDA) today approved eliglustat (Cerdelga, Genzyme) for treating adults with type 1 Gaucher disease, a genetic disorder affecting some 6,000 Americans, the agency announced.

A hard gelatin capsule, eliglustat is the only first-line oral therapy for the condition, according to Genzyme. Intravenous enzyme replacement is the standard treatment for Gaucher disease, caused by a deficiency of an enzyme called glucocerebrosidase. As a result of this deficiency, fatty materials collect in the spleen, liver, and bone marrow, leading to enlargement of the liver and spleen, anemia, low blood platelet counts, and bone problems.

Eliglustat inhibits the metabolic process that forms the fatty materials in patients with the enzyme deficiency.

"Today's approval offers another important treatment option for patients with Type 1 Gaucher disease," said Amy Egan, MD, MPH, deputy director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research.

The FDA determined eliglustat to be safe and effective based on 2 clinical trials involving 199 participants with type 1 Gaucher disease. In one trial, patients receiving the drug had a greater reduction in spleen volume, and improvement in liver volume, blood platelet count, and hemoglobin level compared with patients given a placebo. Both groups had not received enzyme replacement therapy beforehand.

The other trial compared eliglustat head-to-head with imiglucerase, an enzyme replacement therapy, in patients whose Gaucher disease had been previously stabilized by enzyme replacement. "Treatment with Cerdelga resulted in similar stabilization of hemoglobin level, platelet count, and spleen and liver volume as imiglucerase," the FDA said in a news release.

Fatigue, headache, nausea, diarrhea, and back pain were among the most common adverse events.

Genzyme stated in a news release today that a small number of adults with type 1 Gaucher disease who, as genetic lab tests indicate, metabolize eliglustat more quickly or at an undetermined rate will not be eligible for the drug.

More information on today's FDA decision is available on the agency's Web site.


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