Genzyme announced data from its Phase 2 study of eliglustat tartrate for the treatment of Gaucher disease type 1. Genzyme previously reported that the trial had met its primary endpoint at one year, and that data demonstrated continued improvement through two years. The primary composite endpoint was a clinically meaningful response in at least two of three endpoints: improvements in spleen size, hemoglobin and platelet levels. Sustained or further improvements were observed across all endpoints, including bone disease, at the three-year time point.
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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