Phase III success for eliglustat tartrate (Sanofi/Genzyme) in Gaucher Disease - 03-Oct-2012

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.