National Gaucher Foundation - Gaucher Disease
Slideshow Image 1
Slideshow Image 2
Slideshow Image 3


Genzyme Corporation
Eliglustat tartate (Genz-112638)

What is Eliglustat?

  • Eliglustat is a new investigational phase 3 compound from Genzyme Corporation that is being studied for type 1 Gaucher Disease.
  • Eliglustat works as a substrate reduction therapy by reducing glucocerebroside. formation.
  • This product is an oral agent (i.e. a pill) that is taken once or twice a day in contrast to an IV infusion for enzyme replacement therapy. Enzyme replacement therapy focuses on replenishing the enzyme that is deficient in Gaucher Disease and breaks down glucocerebroside that accumulates.
  • The clinical trials for eliglustat tartate are sponsored by Genzyme Corporation.

Is Eliglustat the same as the currently available oral compound? or Miglustat?

  • Miglustat (also known as Zavesca┬«) was developed and is marketed by Actelion Pharmaceuticals Ltd. and is an approved oral compound for type 1 GD in the United States.
  • Eliglustat is not miglustat (Zavesca┬«) and is being developed by Genzyme Corporation.

What are the Clinical Trials for Eliglustat?

  • Currently there are three clinical trials studying eliglustat in varying populations.
    ENGAGE: patients get treated with placebo or eliglustat
    ENCORE: patients get treated with Cerezyme® or eliglustat
    EDGE: patients get treated with one a day or twice a day dosing of eliglustat.
  • Please contact your treating physician or Genzyme medical information if you interested in participating in these studies.
  • Please call Genzyme Medical Information for any additional details (ie study sites, entry criteria, etc). Genzyme Medical Information 800-745-4447.

Additional details of the clinical trials can be found via clinical website. Please click here to connect directly to the eliglustat listings on the clinical website.

Eliglustat in the news

  • Genzyme Corporation Press Release 2/18/2011 Three-Year Data from Phase 2 Trial of Genzyme Gaucher Disease Oral Compound Suggest Sustained or Further Improvement Across All Endpoints
    Click here for Genzyme Press Release.
  • American Society of Hematology (ASH) Press Release on 5/3/2010 that highlights the publication of phase 2 eliglustat data. Click here for ASH Press Release.
  • The Phase 2 eliglustat study details results after 1 year of treatment has been published in Blood May 3, 2010
    • Citation: A Phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Lukina et al. Blood (2010).
    Click here for publication.

Any additional questions?
Please call Genzyme Medical Information