Sanofi has announced positive new data from two late-stage studies of its new Gaucher disease treatment eliglustat.
Results from the Phase III ENGAGE and ENCORE studies of eliglustat have been presented which show that both studies met their primary efficacy endpoints. Top-line data from ENCORE study presented by the French drugmaker's Genzyme unit showed that the new drug is non-inferior to Cerezyme (imiglucerase), the company's standard-of-care treatment for Gaucher which is administered through intravenous infusions.
The data from the trials, which revealed an improvement in spleen size in patients treated with eliglustat will form the basis of the registration package being put together by Sanofi. They are part of the largest clinical programme ever focused on Gaucher which involves some 400 patients treated in 30 countries.
Rogerio Vivaldi, head of rare diseases at Genzyme, says that the fresh data "reinforce our confidence that eliglustat may become an important oral option for patients with Gaucher disease". He added that the firm is "making excellent progress in our robust development plan for bringing eliglustat to the market".
The other treatments available for Gaucher, a rare inherited lysosomal storage disorder, are Shire's Vpriv (velaglucerase alfa) and Pfizer/Protalix Biotherapeutics' Elelyso (taliglucerase alfa).