Sanofi has posted promising late-stage data on its oral investigational Gaucher disease drug eliglustat.
The French drugmaker says that previously untreated patients with Gaucher disease type 1 on eliglustat had a statistically significant improvement in spleen size at nine months, compared with placebo. Specifically, spleen volumes decreased from baseline by a mean of 28% versus just 2% in placebo patients.
Sanofi's Genzyme unit noted that all secondary endpoints were met, including improvements in haemoglobin and platelet levels, as well as liver volumes compared with placebo-treated patients. Eliglustat was well tolerated and there were no serious adverse events reported.
Sanofi already dominates the Gaucher market with Cerezyme (imiglucerase), the standard of care which is administered through intravenous infusions. Genzyme chief executive David Meeker says the data are consistent with what were observed in Phase II, continuing to suggest that eliglustat "is a potent, well-tolerated oral compound that may become a meaningful option".
He added that the development of eliglustat "has been underway for more than a decade and is the largest clinical programme ever focused on Gaucher disease".