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What Is Genzyme Reporting For Its Orphan Drug Eliglustat Tartrate For Gaucher Disease ? (October 2, 2012)

October 2, 2012

       Genzyme announces October 2, 2012, that the 1st Phase 3 trial (ENGAGE) for its new oral therapy, Eliglustat Tartrate, meets its primary endpoint in previously untreated Type 1 Gaucher Disease patients. “Patients treated with Eliglustat Tartrate had a statistically significant improvement in spleen size at 9 months, compared with placebo”.

        In a crowded Enzyme Replacement Therapy (ERT) market for Gaucher Disease, Eliglustat Tartrate may provide Genzyme with differentiation. The Gaucher Disease market is small in size, with < 10,000 patients worldwide.

       Here are the facts about Eliglustat Tartrate:

–  Is a new investigational oral therapy for Gaucher Disease

–  FDA Orphan Drug Designation (ODD) is given to Eliglustat on September 17, 2008 for the treatment of Gaucher Disease

–  Development of Eliglustat Tartrate has been going on for > 10 years

–  “Largest clinical program ever focused on Gaucher Disease

–  Is being developed to provide a “convenient treatment alternative” for patients with Type 1 Gaucher Disease – as Genzyme’s Cerezyme is administered through ongoing intravenous infusions

–  Complete ENGAGE study results are planned for presentation at the Lysosomal Disease Network WORLD Symposium, February 12 – 15, 2013 in Orlando, Florida

–  Data from the 2nd Phase 3 trial, ENCORE, is expected in early 2013.


FDA Orphan Drug Product Designation Database Record for Eliglustat October 2, 2012 Article titled, “Sanofi’s Genzyme hits main target in PhIII study of rare disease drug”

Genzyme October 2, 2012 News Release titled,”Genzyme Phase 3 Study of Oral Compound Eliglustat Tartrate for Gaucher Disease Meets Primary Endpoint” October 2, 2012 Article titled,”Sanofi’s Genzyme study on Gaucher meets endpoint” 

Copyright © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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