Horizon scanning: Phase III data for eliglustat for Gaucher disease type 1

Source: BioSpace Area: News BioSpace has reported on new Phase III data for eliglustat tartrate, an investigational oral therapy for Gaucher disease type 1.   The randomised, double-blind, placebo-controlled ENGAGE study evaluated the safety and efficacy of eliglustat tartrate in 40 treatment-naïve patients with Gaucher disease type 1. The primary endpoint was spleen size - spleen volume decreased by a mean of 28% in patients treated with eliglustat tartrate and increased by a mean of 2% in those treated with placebo (p<0.0001). Improvements in a number of secondary endpoints (e.g. platelet levels) were also noted.   ENCORE was an open-label RCT designed to determine whether eliglustat tartrate is non-inferior to imiglucerase injection (Cerezyme®). In the trial, 160 patients with Gaucher disease type 1 who had begun enzyme replacement therapy at least three years prior to randomisation and who had reached therapeutic goals were randomised to receive either eliglustat tartrate or ...
Source: NeLM - News - Category: Drugs & Pharmacology Source Type: news