Genzyme Corp., a unit of Sanofi-Aventis (SNY, SNYNF.PK), Wednesday said that a four-year follow-up data from a phase 2 trial for its investigational oral drug 'eliglustat tartrate' for Gaucher disease, showed further improvements across all endpoints.
Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the four-year timepoint, Genzyme said in a statement.
Gaucher disease (pronounced Go-shay) is an inherited genetic disorder, in which patients lack the normal form of glucocerebrosidase, or GCD enzyme that breaks down specific fat molecules. The lack of this enzyme results in the accumulation of fat in liver, spleen and bone marrow. The disease affects about 1 in 100,000 persons in the general population.
Genzyme previously reported that the 52-week phase 2 trial of eliglustat tartrate had met its primary composite endpoint. 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.
The composite endpoint is clinically meaningful response in at least two of three endpoints i.e. improvements in spleen size, hemoglobin and platelet levels in individual patients.
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