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Gaucher Disease Market Business Developments

February 8, 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 7 months.

Top Drugs In Gaucher Disease Market

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.

Business Developments

Protalix BioTherapeutics, who partners with Pfizer for drug Elelyso (taliglucerase alfa) for Gaucher Disease, admits recently that the company has hired Citigroup to “explore strategic options” for possible product partnering, technology sharing, and other strategic alternatives. It is Elelyso’s “unique plant cell-based manufacturing system that is making the small Israeli biotech potentially worth $1 billion”. Elelyso is the first drug to win FDA approval for a drug made from a genetically engineered plant – it is made from carrot cells. Pfizer markets Elelyso in the US and Protalix BioTherapeutics has granted them exclusive, worldwide license to develop and commercialize the drug. Protalix BioTherapeutics keeps rights to Elelyso in Israel. This partnership should help get Elelyso approved in other markets.

Per a Bloomberg article this week, Pfizer has expressed interest in buying Protalix BioTherapeutics. This was reported by the Israeli financial newspaper Calcalist, without identifying the source of the information. Calalist reported that Novartis and Merck also may want to buy the company.

It will be interesting to see what Dr. David Aviezer, Protalix BioTherapeutics’ President and CEO, will present at the 15th Annual BIO CEO and Investor Conference on Tuesday, February 12, 2013 at 11:30 AM ET.

On February 7, 2013, Sanofi/Genzyme reports on its 2012 financial results for their ERT Gaucher Disease product Cerezyme and reports on the status of their new drug in development for Gaucher Disease, Eliglustat Tartrate :

  1. 2012 Cerezyme grew sales 6.0% to €633 million
  2. ENCORE, the second pivotal phase III trial of the new investigational twice-daily oral therapy, Eliglustat Tartrate for Gaucher Disease, met its primary efficacy endpoint of clinical stability compared to bi-weekly infusions of Cerezyme®.  More detailed topline results from the ENCORE trial will be released in association with the upcoming Annual WORLD Symposium (see below). No time frame was given on possible regulatory submissions.

Shire plc will report on the company’s full year 2012 earnings and developments on February 14, 2013.

As reported in a January 2013 Blog Post, Gaucher Disease and ERT drugs are to be a major topic at next week’s 9th Annual Lysosomal Disease Network World Symposium, February 13 – 15, 2013, in Orlando, Florida :

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.

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.

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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