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Why Is Protalix BioTherapeutics And Pfizer’s Orphan Drug Elelyso For Gaucher Disease In The News ?

November 1, 2012

       The European Commission (EC) on November 1, 2012, issues a Commission Decision refusing the Marketing Authorization for Pfizer and Protalix BioTherapeutics’ orphan drug Elelyso (Taliglucerase Alfa), an Enzyme Replacement Therapy (ERT), for the treatment of Gaucher Disease. The EC endorses the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) June 22, 2012 recommendation, not to issue a Marketing Authorization for Elelyso in the EU. This decision is not because of Elelyso’s safety or efficacy, but is based on the specific requirements of the EU Orphan Drug Regulation. 

       On June 22, 2012, CHMP issues its Opinion on Elelyso, and gives it a positive risk-benefit assessment, concluding that the benefits of the drug outweigh its risks. Despite this positive risk-benefit assessment, CHMP doesn’t recommend a Marketing Authorization, due to the fact that “Shire plc’s Vpriv has received prior Marketing Authorization with orphan drug designation for the same condition. Therefore, Shire’s treatment has orphan market exclusivity in the EU for a 10-year period starting in August 2010”. 


Pfizer & Protalix BioTherapeutics November 1, 2012 Press Release via  

Orphan Druganaut June 26, 2012 Blog Post titled,”Why was Pfizer’s drug Elelyso not recommended for gaucher disease in Europe ?”  

Orphan Druganaut August 13, 2012 Blog Post titled,”Pfizer, Protalix BioTherapeutics, and orphan drug Elelyso Sales”  

Orphan Druganaut September 27, 2012 Blog Post titled,”Good news for Protalix BioTherapeutics’ orphan drug Elelyso for Gaucher Disease”  

Orphan Druganaut October 2, 2012 Blog Post titled,”Internet Buzzing about Protalix BioTherapeutics’ orphan drug Elelyso for Gaucher Disease (October 1, 2012)”  

Orphan Druganaut October 3, 2012 Blog Post titled,”How does Genzyme’s orphan drug Eliglustat Tartrate fit into the Crowded Gaucher Disease ERT Market ?”  

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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