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news.Genzyme has announced three-year follow-up data of the phase 2 trial for its investigational oral therapy, eliglustat tartrate, indicated for the treatment of Gaucher disease type 1.
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Eliglustat tartrate is a novel glucosylceramide analog administeredorally, which partially inhibits the enzyme glucosylceramide synthase.
Currently, it is being developed to provide a convenient treatment alternative for adult patients with Gaucher disease type 1.
The three-year data suggest that eliglustat tartrate positively impacts (sustained and further improvements) indicators of bone disease through three years of follow up.
These indicators include bone mineral density in the lumbar spine, and dark marrow signal in the femur, which reflects the infiltration of lipid-laden Gaucher cells into bone marrow.
Genzyme president of Personalized Genetic Health John Butler said the company’s momentum continues through the phase 3 trials – the largest ever conducted for Gaucher – as it builds upon its foundation and commitment to extend therapeutic options available to patients and physicians.
Genzyme’s Gaucher disease portfolio includes Cerezyme (imiglucerase for IV injection).