Positive Ph III clinical trial extension results for Genzyme's Cerdelga

French pharma major Sanofi (Euronext: SAN) subsidiary Genzyme has reported positive extension study data from its Phase III ENGAGE and ENCORE studies of Cerdelga (eliglustat), a first-line oral therapy approved by the US Food and Drug Administration and the European Commission for the treatment of certain adults with Gaucher disease type 1.

The results from the studies were presented today at the 11th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Florida. Both extension studies demonstrated continued stability and/or improvements across established end points and published therapeutic treatment goals.

Genzyme developed Cerdelga, a capsule taken orally, to provide a treatment alternative for certain adult 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 Cerdelga represents the largest clinical program ever focused on Gaucher disease, with around 400 patients treated in 29 countries. The drug was approved in the USA last summer and in Europe last month.