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

Quick Facts

Editors Pick
Alaska Air Group Inc., which completed its $2.6 billion acquisition of Virgin America last year, said that the combined company will adopt Alaska Air's name and logo. The Virgin America name will be retired, "likely sometime in 2019," the airline added. Apple Inc. has agreed to acquire Workflow, an iOS app for automating tasks, as well as its development team for an undisclosed sum. Workflow is a personal automation tool that enables the user to drag and drop combination of actions to create workflows for the iPhone, iPad and Apple Watch. After long negotiations to win over one of the largest and fastest growing smart phone markets, Apple has decided to assemble its 4-inch screen iPhone SE in India.
comments powered by Disqus
Follow RTT