Genzyme's eliglustat meets endpoints in Ph III studies for Gaucher disease

US biotech firm Genzyme, a subsidiary if French drug major Sanofi (Euronext: SAN) has released positive new data from two Phase III studies - ENGAGE and ENCORE - of eliglustat tartrate, its investigational oral therapy for Gaucher disease type 1. Both studies, presented on Friday at the 9th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Florida, met their primary efficacy endpoints and together will form the basis of Genzyme’s registration package for eliglustat tartrate.

The company is developing eliglustat tartrate, a capsule taken orally, to provide a convenient treatment alternative for patients with Gaucher disease type 1 and to provide a broader range of treatment options for patients and physicians. Genzyme’s clinical development program for eliglustat tartrate represents the largest clinical program ever focused on Gaucher disease type 1 with around 400 patients treated in 30 countries.

“The data presented at this year’s WORLD Symposium reinforce our confidence that eliglustat tartrate may become an important oral option for patients with Gaucher disease,” said Genzyme’s head of rare diseases, Rogerio Vivaldi, adding: “We are excited about this therapy’s potential and are making excellent progress in our robust development plan for bringing eliglustat tartrate to the market.”