Results of Phase III ENGAGE trial for Cerdelga (eliglustat) for Gaucher disease published in JAMA-Sanofi

Sanofi and its subsidiary Genzyme, announced the publication of results from the ENGAGE registration study evaluating Cerdelga (eliglustat) in treatment-naïve patients with Gaucher disease type 1 in the February 17, 2015 issue of The Journal of the American Medical Association. The ENGAGE study is a Phase III randomized, double-blinded, placebo-controlled, multinational registration trial of 40 eligible treatment-naïve patients with Gaucher disease type 1 who had splenomegaly in addition to thrombocytopenia and/or anemia at study entry. Patients were stratified by baseline spleen volume and randomized 1:1 to receive Cerdelga (50 or 100 mg twice daily) or placebo for nine months, following assessment for improvements in disease manifestation.The primary efficacy endpoint of the study demonstrated: A statistically significant reduction from baseline in spleen size by a mean of 28 percent compared in Cerdelga patients with a mean increase of two percent in placebo patients, for an absolute difference of 30 percent (P less than 0.0001) Secondary endpoints were also statistically significant: Hemoglobin levels increased from baseline by an absolute difference of 1.2 g/dL compared with placebo (P=0.006). Liver volume decreased from baseline by an absolute difference of 6.6 percent compared with placebo (P=0.0072). Platelet levels increased from baseline by an absolute difference of 41 percent compared with placebo (P less than 0.0001). There were no serious adverse events associated with either treatment group.

Comment: Cerdelga will be competing for market share with Vpriv (velaglucerase alfa) from Shire and Elelyso (taliglucerase alfa) from Pfizer/Protalix Biotherapeutics, though these are both injected. Actelion also has an oral drug on the market for type I Gaucher - Zavesca (miglustat) - but this is only used in people who cannot be treated with enzyme replacement therapy.