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FDA Approves Cerdelga (Eliglustat) for Gaucher Disease

Treatment targets type-1 disease in adults

The FDA has given the green light to Cerdelga (eliglustat, Genzyme) for the long-term treatment of adult patients with the type-1 form of Gaucher disease, a rare genetic disorder.

Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. This enzyme deficiency causes fatty materials to collect in the spleen, liver, and bone marrow. The major signs of Gaucher disease include liver and spleen enlargement, anemia, low platelet counts, and bone problems.

Cerdelga is a hard gelatin capsule containing eliglustat that is taken orally. In patients with type-1 Gaucher disease, the drug slows the production of the fatty materials by inhibiting the metabolic process that forms them.

Type-1 Gaucher disease is estimated to affect approximately 6,000 people in the U.S.

The safety and efficacy of eliglustat were evaluated in two clinical trials involving a total of 199 subjects with type-1 Gaucher disease. In one randomized, double-blind, placebo-controlled trial, the safety and efficacy of eliglustat were evaluated in 40 participants with type-1 Gaucher disease who had not previously received enzyme replacement therapy (ERT). The subjects received eliglustat at a starting dosage of 42 mg twice a day (BID), with most receiving a dosage of 84 mg BID after 4 weeks. The subjects continued the drug for 9 months.

Compared with placebo, treatment with eliglustat resulted in a greater reduction in spleen volume from baseline to the end of the study (by week 39) — the trial’s primary endpoint. Treatment with eliglustat also resulted in greater improvement in liver volume, blood platelet count, and hemoglobin level compared with placebo.

The other trial evaluated the safety and efficacy of eliglustat compared with that of ERT in 159 subjects with type-1 Gaucher disease previously treated with and stabilized on ERT. The subjects received either eliglustat or the ERT drug imiglucerase (Cerezyme, Genzyme). Treatment with eliglustat resulted in similar stabilization of the hemoglobin level, platelet count, and spleen and liver volumes compared with imiglucerase.

The most commonly observed side effects in the clinical trials of eliglustat were fatigue, headache, nausea, diarrhea, back pain, pain in the extremities, and upper abdominal pain.

Source: FDA; August 19, 2014.

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