FDA Approves Cerdelga for Type 1 Gaucher Disease

Gelatin capsule is designed to slow the body's production of abnormal fatty deposits

WEDNESDAY, Aug. 20, 2014 (HealthDay News) -- Cerdelga (eliglustat) has been approved by the U.S. Food and Drug Administration to treat type 1 Gaucher disease, a rare inherited disorder caused by the body's insufficient production of a key enzyme.

Lack of the enzyme glucocerebrosidase causes fatty deposits in the spleen, liver, and bone marrow. Symptoms include liver and spleen enlargement, anemia, low blood platelets, and bone problems, the FDA said in a news release. Gaucher affects about 6,000 people in the United States. Cerdelga is a gelatin capsule that's designed to slow the body's production of abnormal fatty deposits.

According to the FDA, Cerdelga was evaluated in two clinical studies involving 199 people with type 1 Gaucher disease. In the first study, compared to placebo, Cerdelga resulted in a greater reduction in spleen volume from baseline to the end of the study, and also resulted in greater improvement in liver volume, blood platelet count, and hemoglobin level.

In the second trial, the researchers found that treatment with Cerdelga resulted in similar stabilization of hemoglobin level, platelet count, and spleen and liver volume as imiglucerase. The most common side effects observed in both trials were fatigue; headache; nausea; diarrhea; and pain of the back, arms and legs, and upper abdomen.

Cerdelga is produced by Genzyme, based in Cambridge, Mass.

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