Lysosomal Storage Disorders: Approval In Australia For Gaucher Disease
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 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:
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.
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