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ERT Orphan Drug Competition In The Gaucher Disease Market And Upcoming 2013 Meeting

January 6, 2013

       There is fierce competition in the Gaucher Disease Enzyme Replacement Therapy (ERT) market. The Gaucher Disease market and rival ERT orphan drugs have been well documented by the Orphan Druganaut Blog for the past 6 months. Gaucher Disease and ERT drugs are to be a topic at the upcoming 9th Annual Lysosomal Disease Network World Symposium, February 13 – 15, 2013, in Orlando, Florida.

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

1) Genzyme’s Cerezyme (Imiglucerase) injection which is the standard of care (SOC) at $200,000/patient/year

2) Shire plc’s Vpriv (Velaglucerase Alfa) injection at $170,000/patient/year

3) Pfizer & Protalix BioTherapeutics’ Elelyso (Taliglucerase Alfa) injection at $150,000/patient/year.

Genzyme’s Eliglustat Tartrate is to be a future entrant into the already crowded Gaucher Disease ERT market. The number of Gaucher Disease patients worldwide is small, < 10,000. Eliglustat Tartrate may provide Genzyme with differentiation as it is an oral capsule.

Eliglustat Tartrate

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

Lysosomal Disease Network WORLD Symposium – Genzyme’s Eliglustat Tartrate Presentations :

Friday, February 15, 2013 (Session 5 – Clinical Research)

8:30 AM    M. Judith Peterschmitt        Biomarker Responses to Eliglustat, an Investigational Oral Substrate Reduction Therapy (SRT) for Gaucher Disease Type 1

11:15 AM     Pramod Mistry                     Phase III (ENGAGE), Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Investigate The Efficacy & Safety of Eliglustat in Patients with Gaucher Disease: Results After 9 Months of Treatment.

Lysosomal Disease Network WORLD Symposium – Pfizer & Protalix BioTherapeutics’ Elelyso Presentation :

Friday, February 15, 2013 (Session 5 – Clinical Research)

11:00 AM    Gregory Pastores        Plant Cell-Expressed Recombinant Glucocerebrosidase: Taliglucerase Alfa as Therapy for Gaucher Disease in Adults Patients Previously Treated with Imiglucerase: 24-Month Results.

Lysosomal Disease Network WORLD Symposium – Shire’s Vpriv Presentation :

Friday, February 15, 2013 (Session 5 – Clinical Research)

10:45 AM    Ari Zimran                   Bone Mineral Density Change in Type 1 Gaucher Disease Adults Given Velaglucerase Alfa for 2 Years.

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

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