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Impact Of Eliglustat Tartrate On The ERT Gaucher Disease Market Competition

February 16, 2013

Genzyme pioneered Enzyme Replacement Therapy (ERT) for Gaucher Disease with its orphan drug Cerezyme, which enjoyed a period of market monopoly.  Competition in the form of 2 new ERT drugs, Shire plc’s Vpriv and Pfizer and Protalix BioTherapeutics’ Elelyso, started to appear in 2010 as the new players in the marketplace. Genzyme’s strategy in the ERT Gaucher Disease marketplace has been so successful, that the company is seen as a business model for succeeding in bringing an orphan drug to market.  A CurrentPartnering.com article titled, “Shire Takes Its Specialty Seriously”, provides an interesting perspective on Shire plc’s successful specialty business model – the development and bringing to market of an orphan drug for a rare disease.

Three Top Competitor Drugs In ERT 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.

The Gaucher Disease ERT market is crowded with only a patient population worldwide of less than 10,000. If Genzyme’s new orphan drug Eliglustat Tartrate is to be a future entrant into the Gaucher Disease ERT market, it must differentiate itself from the other 3 drug competitors. Eliglustat Tartrate is an oral capsule, while the other 3 approved ERT drugs are injections. According to a Genzyme February 15, 2013 Press Release, “ …. Eliglustat Tartrate, a capsule taken orally, to provide a convenient treatment alternative for patients with Gaucher Disease …. and to provide a broader range of treatment options for patients and physicians ….”.

There has been a lot of publicity surrounding Genzyme’s presentation on Eliglustat Tartrate at last week’s 9th Annual Lysosomal Disease Network (LDN) World Symposium.  Genzyme gave 2 presentations this past Friday 15, 2013 :

  1. 8:30 AM M. Judith Peterschmitt Biomarker Responses to Eliglustat, an Investigational Oral Substrate Reduction Therapy (SRT) for Gaucher Disease Type 1
  2. 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.

According to a Genzyme February 15, 2013 Press Release, Eliglustat Tartrate met its main targets in 2 late-stage Phase III studies. The study results will be used to strengthen Genzyme’s request for regulatory approval when the company’s registration package is submitted. Eliglustat Tartrate could become the 1st oral treatment for Gaucher Disease which can shake up an already crowded ERT market.

The first Phase III study’s (ENGAGE) data is presented on February 15, 2013 at the 9th Annual LDN World Symposium, and compares the Eliglustat Tartrate pill with a placebo in a 40-patient trial. There is a decrease in spleen volume by a mean of 28 % compared with a mean increase of 2 % in placebo. The drug also met 3 other secondary endpoints :

  1. Increasing Hemoglobin levels
  2. Increasing Platelet        levels
  3. Decreasing liver volume.

The second Phase III study’s (ENCORE) results are released by Genzyme at the 9th Annual LDN World Symposium. This study compares Eliglustat Tartrate with Genzyme’s other drug, Cerezyme. The study with 160 patients, who are stable on Cerezyme, and are then either switched to Eliglustat Tartrate or remain on Cerezyme. Patients treated with the new pill for 1 year, had 84% of patients remaining stable in all 4 parameters  (spleen volume, hemoglobin level, liver volume, and platelet levels), compared to 94% on Cerezyme. No serious side effects are reported. Full data from the second study will be presented at a medical meeting in the second half of 2013.

A third late-stage Phase III (EDGE) study of Eliglustat Tartrate is testing currently whether the dosage for the pill can be reduced from twice daily to once daily.

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

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