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How Does Genzyme’s Orphan Drug Eliglustat Tartrate Fit Into The Crowded Gaucher Disease ERT Market ?

December 24, 2012

For the past 6 months, the Orphan Druganaut Blog has followed the competition in the Gaucher Disease Enzyme Replacement Therapy (ERT) market.

Investors are asking how will Genzyme’s orphan drug Eliglustat Tartrate fit into the crowded Gaucher Disease ERT market. The number of Gaucher Disease patients worldwide is small, < 10,000. Eliglustat Tartrate may provide Genzyme with differentiation.

– The top 3 competitor drugs in the Gaucher Disease market are :

1)     Genzyme’s Cerezyme injection which is the standard of care (SOC)

2)     Shire plc’s Vpriv injection

3)     Pfizer & Protalix BioTherapeutics’ Elelyso injection.

– Eliglustat Tartrate is an oral capsule

– The approximate cost of the Gaucher Disease drugs :

1)     Cerezyme is $200,000/year

2)     Vpriv is $170,000/year

3)     Elelyso is $150,000/year

4)     Eliglustat Tartrate cost is not known at this point.

Eliglustat Tartrate

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

– 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.


Genzyme Pipeline

Orphan Druganaut October 2, 2012 Blog Post reviews the announcement by Genzyme of the positive Phase 3 trial results for orphan drug Eliglustat Tartrate for Type 1 Gaucher Disease.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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