Genzyme announced results from its extension study of the Phase 3 ENGAGE and ENCORE studies of Cerdelga (eliglustat) for the treatment of certain adults with Gaucher disease type 1.

ENGAGE is a randomized, double-blind, placebo-controlled, multicenter study. In the primary analysis period, improvements were seen across the following endpoints after nine months on Cerdelga: spleen size, platelet count, hemoglobin concentration, and liver volume. In the nine month extension phase, patients who switched from placebo to eliglustat showed improvements similar to the eliglustat-treated patients during the primary analysis while the eliglustat-treated patients continued to show improvements during the nine month extension period.

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ENCORE is a randomized, controlled, open-label non-inferiority study comparing eliglustat to Cerezyme (imiglucerase) in Gaucher disease type 1 patients stabilized on enzyme replacement therapy. The study, which met the primary analysis criteria for non-inferiority to Cerezyme, had a composite endpoint of each of the following parameters: spleen volume, hemoglobin concentration, platelet counts, and liver volume at 12 months. During the 12-month extension period, the patients who crossed over to eliglustat treatment from imiglucerase remained stable. Patients treated with eliglustat for 24 months also maintained stability of clinical parameters during the extension period.

Cerdelga is already indicated for the long-term treatment of adults with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers as detected by an FDA-cleared test.

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