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Lysosomal Storage Disorders: Approval In Canada For Gaucher Disease

June 16, 2014

This is the tenth Blog Post in a continuing  series of Blog Posts that examines Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post reviews the recent regulatory approval of Protalix BioTherapeutics’ (Israel) Enzyme Replacement Therapy (ERT), Elelyso (Taliglucerase Alfa), for Gaucher Disease (GD) by Health Canada at the end of May 2014. Elelyso is to be marketed in Canada by Pfizer.

Health Canada:

•   Grants regulatory approval for Elelyso for long-term ERT for GD Type 1 for both adult and pediatric patients

•   Elelyso may also be used for hematological manifestations in pediatric patients with a confirmed diagnosis of GD Type 3.

Elelyso Global Regulatory Activity

Regulatory Date Regulatory Agency & Action Taken
May 2012 FDA Approval
June 2012 EMA CHMP Opinion recommends against approval due to Shire plc’s exclusivity for Vpriv
September 2012 Israel’s Ministry of Health Approval
November 2012 EC adopts EMA CHMP’s recommendation
March 2013 Brazil’s National Health Surveillance Agency Approval
April 2013 Mexico’s Federal Commission for the Protection against Sanitary Risk (COFEPRIS) Approval
May 2014 Australia’s TGA Approval
May 2014 Health Canada’s Approval

.

Further regulatory filings are underway.

Gaucher Disease Market

With the GD market being dominated by injectable ERT treatments (Genzyme’s Cerezyme, Shire’s Vpriv, and Protalix BioTherapeutics’ Elelyso), there are new oral therapies on the horizon in development :

•   Genzyme’s Cerdelga (Eliglustat)

•   Protalix BioTherapeutics’ Glucocerebrosidase (GCD) or PRX-112.

Please Note: “First Pigment Analysis (5893349712)” by NASA ICE (First Pigment Analysis Uploaded by russavia) [CC-BY-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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