Phase III success for eliglustat tartrate (Sanofi/Genzyme) in Gaucher Disease

ENGAGE, the first Phase III trial of the investigational oral therapy, eliglustat tartrate,from Sanofi/Genzyme, in previously untreated patients with Gaucher disease type 1, met its primary endpoint. Patients treated with eliglustat tartrate had a statistically significant improvement in spleen size at nine months, compared with placebo. Spleen volumes in eliglustat tartrate treated patients decreased from baseline by a mean of 28 percent versus a mean increase of two percent in placebo patients, for an absolute difference of 30 percent (p<0.0001). in addition, all secondary endpoints were met, including improvements in hemoglobin levels and platelet levels, as well as liver volumes compared with placebo-treated individuals.the initial safety analysis from engage suggests that eliglustat tartrate was well tolerated. there were no serious adverse events reported in the primary analysis period.>