logo
Share SHARE
FONT-SIZE Plus   Neg

Genzyme's Gaucher Drug Shows Positive Results In Follow Up Data Of Phase 2 Trial

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.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Editors Pick
The recent week marked the lull before the storm, with very few market moving economic numbers released during the week. However, the economic news flow picks up pace in the upcoming week, offering glimpses into every vehicle of economic growth, ranging from consumers to private sector activity to housing.... Life expectancy at birth in the U.S. is already lower than most other developed nations and is now projected to fall further behind by 2030, according to a new study. It also suggests that by 2030, national female life expectancy in South Korea could break the 90 year barrier. Department store chain J.C. Penney Co. Inc. on Friday reported a turnaround to profit in the fourth quarter as a slight decline in sales was more than offset by lower expenses. Adjusted earnings per share for the quarter beat analysts' expectations, while revenues slightly missed their estimates. Earlier today, J.C. Penny announced a plan to optimize its national retail operations.
comments powered by Disqus
Follow RTT