Positive Ph II results for Genzyme's oral Gaucher drug

French drug major Sanofi’s (Euronext: SAN) US subsidiary Genzyme yesterday revealed four-year follow-up data from patients enrolled in the Phase II clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate.

Sustained or further improvements were observed across all endpoints, including markers of bone disease, at the four-year timepoint. The results will be presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in San Diego, California.

Eliglustat tartrate, a capsule taken orally, is being developed to provide a convenient treatment alternative for adult patients with Gaucher disease type 1, and to offer a broader range of treatment options for patients and physicians to achieve individual therapeutic goals. Genzyme’s Cerezyme (imiglucerase for injection), the standard of care for patients with Gaucher disease type 1, is administered through intravenous infusions.