Genzyme announced four-year follow-up data from its Phase 2 clinical trial of eliglustat tartrate for the treatment of Gaucher disease type 1. Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the four-year timepoint.

Genzyme previously reported that the 52-week Phase 2 trial of eliglustat tartrate had met its primary composite endpoint: a clinically meaningful response in at least two of three endpoints (improvements in spleen size, hemoglobin, and platelet levels) in individual patients. Patients have continued to receive eliglustat tartrate in the extension portion of the study for over four years. The data from patients on eliglustat tartrate after four years indicate continued or stabilized improvements across all endpoints: Spleen and liver volumes decreased from baseline by a mean of 63% and 28%, respectively; hemoglobin and platelet levels had increased from baseline by a mean of 2.3g/dL and 95%, respectively; and all patients (100%) had met at least three of the four hematologic and visceral therapeutic goals established for enzyme-replacement therapy. These data also indicate continued improvement in bone mineral density by DXA, with a mean T-score increase of 0.8 from baseline in the lumbar spine.

Eliglustat tartrate, a novel, oral glucosylceramide analog, is designed to partially inhibit the enzyme glucosylceramide synthase, which results in reduced production of glucosylceramide. Glucosylceramide is the substance that builds up in the cells and tissues of people with Gaucher disease. 

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