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What Is The Most Expensive Orphan Drug In The World ?

December 21, 2012

The most expensive drug in the world in 2010, is orphan drug Soliris (Eculizumab) from Alexion Pharmaceuticals.

Soliris in the US

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

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

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

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

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

Costs > $409,000/annually/patient

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

– There are 4,000 – 6,000 PNH patients in the US.

Soliris is approved for patients with PNH in the US (2007), European Union (2007), Japan (2010), and other territories. It is the first and only treatment indicated for PNH . Soliris is also approved as the first and only treatment for patients with aHUS, another ultra-rare disease, in the US (September 2011) and in the European Union (November 2011).

References February 6, 2012 Article titled, “The 11 Most Expensive Medicines in America”

Orphan Druganaut August 15, 2012 Blog Post titled, “Is there a relationship between the most expensive US Medicines & orphan drugs?”

PNH Foundation

Thomson Reuters 2012 Report titled, “The Economic Power of Orphan Drugs”.

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

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