Eliglustat tartrate: Additional Phase III data

Additional data from the double-blind, placebo-controlled, international Phase III ENGAGE trial in 40 patients showed that twice-daily eliglustat tartrate met the secondary endpoints of increasing platelet levels from baseline (absolute placebo-adjusted difference of 41%, p<0.0001), increasing hemoglobin levels from baseline (absolute placebo-adjusted difference of 1.2 g/dL, p<0.0006) and reducing liver volume from baseline (absolute placebo-adjusted difference of 7%, p<0.0072). On a tertiary endpoint, eliglustat tartrate significantly improved total bone marrow burden vs. placebo. The trial enrolled previously untreated patients with Type I Gaucher's disease to receive eliglustat tartrate or placebo for 9 months, after which placebo patients transitioned to receive eliglustat tartrate. Data were presented at the Lysosomal Disease Network World Symposium in Orlando. ...