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Lysosomal Storage Disorders: Approval In Australia For Gaucher Disease

May 23, 2014

This is the ninth 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) in Australia.


GD is an inherited LSD caused by a defect in a particular enzyme which breaks down fatty substance, or lipid, called glucocerebroside. As a result of this defect, glucocerebroside accumulates in the body’s cells and organs, which prevents them from working properly. There are less than 10,000 people worldwide with GD. There are 3 subtypes of GD:

•   Type 1 is the most common form of GD with swelling, enlargement, and dysfunction of the spleen and liver; also lung and bone problems

•   Type 2 causes brain damage and death before 2 years of age

•   Type 3 is progressive and affects the brain, with patients living often into their early teens and adulthood.

Enzyme Replacement Therapy (ERT) is a lifelong treatment for GD, that involves injection of the appropriate enzyme responsible for the normal breakdown of the lipid in healthy people.

Protalix BIoTherapeutics

Protalix BioTherapeutics partners with Pfizer for the development and commercialization of the orphan drug Elelyso for the treatment of GD Type 1. Elelyso is the first drug to win FDA approval that is made from a genetically engineered plant – easily grown carrot cells. Protalix BioTherapeutics produces Elelyso using its unique plan cell-based ProCellEx Technology.


Australia’s Therapeutic Goods Administration (TGA) approves this week, Elelyso for injection as long-term ERT for the treatment of GD Type 1, in both adult and pediatric patients, with at least one of the following:

•   Splenomegaly

•   Hepatomegaly

•   Anemia

•   Thrombocytopenia.

Australia is the first country in which Elelyso is indicated for both adults and pediatric patients.

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

Further regulatory filings are underway.

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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