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Alexion Pharmaceuticals’ Orphan Drug Soliris (Eculizumab) And Ultra-Rare Diseases: SeekingAlpha.com Perspective

December 5, 2012

       SeekingAlpha.com’s article, “Alexion Pharmaceuticals: On The Road From Orphan Drugs To Blockbuster Profits” discusses and analyzes how Alexion Pharmaceuticals’ only marketed drug, orphan drug Soliris (Eculizumab), has generated blockbuster profits from multiple indications for ultra-rare diseases. Alexion Pharmaceuticals’ drug pipeline and future are also discussed in great detail. 

       Soliris is the world’s most expensive drug, costing approximately $409,500/year/patient. Soliris treats ultra-rare diseases such as Paroxysymal Nocturnal Hemogloginuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). 

       Here are the facts about Soliris in the US : 

–    Receives FDA Orphan Drug Designation (ODD) for paroxysmal nocturnal hemoglobinuria (PNH) on August 20, 2003 

–    Receives FDA Approval for PNH orphan indication & exclusivity start date is March 16, 2007 

–    Receives FDA ODD for atypical Hemolytic Uremic Syndrome (aHUS) on April 29, 2009 

–    Receives FDA Approval for aHUS orphan indication & exclusivity start date is September 23, 2011 

–    Receives FDA ODD for treatment of Shiga-Toxin on October 18, 2011   

–    Generates $295 million in sales in 2009; $541 million in sales in 2010

References 

Alexion Pharmaceuticals Drug Pipeline  

Blog Post titled, “Alexion Pharmaceuticals and Orphan Drug Soliris (Eculizumab)”  

Blog Post titled, “Does Alexion Pharmaceuticals’ Orphan Drug Soliris (Eculizumab) Have a New Rare Disease Indication ?”  

Blog Post titled, “Internet Buzzes Away On Alexion Pharmaceuticals and Orphan Drug Soliris”  

Blog Post titled, “Internet Buzzes (September 4, 2012): Alexion Pharmaceuticals and Orphan Drug Soliris”

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

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