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Does Alexion Pharmaceuticals’ Orphan Drug Soliris (Eculizumab) Have A New Rare Disease Indication ?

October 10, 2012

          Alexion Pharmaceuticals’ orphan drug Soliris (most expensive drug in the world) may have a new rare disease indication, according to an October 9, 2012 article.

          Alexion Pharmaceuticals announces October 9, 2012, that Phase 2 Study data is presented at the American Neurological Association (ANA) Annual meeting in Boston, Massachusetts. The Investigator-Initiated Phase 2 Study of Soliris (Eculizumab) in patients with severe relapsing Neuromyelitis Optica (NMO) met its primary efficacy endpoint with high degrees of clinical and statistical significance.

         Also, per an Alexion Pharmaceuticals’ October 10, 2010 News Release, data will be presented on Soliris as a treatment for patients with aHUS & STEC-HUS at the American Society of Nephrology (ASN) Annual meeting, to be held October 30 – November 4, 2012 in San Diego, California..

        Per the article, “so far, Soliris has generated more than one billion dollars in sales for Alexion in its nearly five years on the market, and driven its stock recently to new 52-week highs. That success also has ushered in discussions around Wall Street and the global bioscience community about Alexion as a potential takeover target for one of the world’s big pharmas.”

        Facts about Neuromyelitis Optica (NMO) :

  1. An ultra-rare neurological disease
  2. Leads to severe weakness, paralysis, blindness, and other symptoms that lead to a premature death
  3. 50% of relapsing NMO patients sustain permanent severe disability within 5 years of disease onset
  4. 30% of patients die within 5 years of disease onset.

       Here are the facts about orphan drug Soliris in the US :

  1. Receives FDA Orphan Drug Designation (ODD) for paroxysmal nocturnal hemoglobinuria (PNH) on August 20, 2003
  2. Receives FDA Approval for PNH orphan indication & exclusivity start date is March 16, 2007
  3. Receives FDA ODD for atypical Hemolytic Uremic Syndrome (aHUS) on April 29, 2009
  4. Receives FDA Approval for aHUS orphan indication & exclusivity start date is September 23, 2011
  5. Receives FDA ODD for treatment of Shiga-Toxin on October 18, 2011  
  6. Costs > $409,000/annually/PNH patient in 2010
  7. Generates $295 million in sales in 2009; $541 million in sales in 2010
  8. There are 4,000 – 6,000 PNH patients in the US.

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

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