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Alexion Pharmaceuticals, Inc. (ALXN), BioMarin Pharmaceutical Inc. (BMRN): 5 of the World’s Most Ultra-Rare Diseases

Published on August 5, 2013 at 9:29 am by The Motley Fool in News
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  • Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR): Aegerion’s early claim to fame is its pill to treat homozygous familial hypercholesterolemia, or HoFH, known as Juxtapid. Priced at $295,000 per treatment, it’s significantly pricier than Kynamro, a competing drug developed by Isis Pharmaceuticals and Sanofi, but it also offers some advantages. Ease of use, for one, since it’s a pill versus Kynamro — which is given intravenously — and also from a safety perspective since Kynamro was associated with abnormal neoplasm growth in clinical trials. Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) is certainly having no issues marketing its HoFH drug to physicians as it basically doubled its sales projections for the remainder of 2013.
  • NPS Pharmaceuticals, Inc. (NASDAQ:NPSP): Also priced at $295,000 per year is NPS’ recently approved short bowel syndrome drug, Gattex. As my Foolish colleague Keith Speights points out, NPS Pharmaceuticals, Inc. (NASDAQ:NPSP)’ estimates its target audience at between 3,000 and 5,000 adults annually, which could add up to more than $1 billion in sales when all is said and done. Like Aegerion, NPS does have some competition in this space, but sales potential is rapidly growing. According to its first-quarter report, 42 patients were on its SBS drug with NPS commenting in June that 200 to 300 patients should be taking Gattex by year’s end.
  • Viropharma Inc (NASDAQ:VPHM): Often the forgotten stock of the biotech group, Viropharma Inc (NASDAQ:VPHM)’s ultra-rare treatment extraordinaire is Cinryze, which it acquired when it purchased Lev Pharmaceuticals in 2008. Cinryze helps to treat hereditary angioedema, a disease that affects about 6,000 people in the U.S., and is among the most expensive drugs in the world. Like Soliris or Juxtapid, its high price hasn’t slowed sales with Cinryze sales jumping 44% in the most recent quarter.

    • A true rarity can be a good thing
    The interest we’re witnessing from ultra-rare drug developers is certainly a positive sign. The success of Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and a few other players in the field has given more than enough encouragement to other biotechnology companies and even larger pharmaceutical players like Sanofi to enter the fray. There are literally thousands of orphan diseases yet to be untapped, so the opportunity for orphan and ultra-orphan drug developers is there for what I see as decades to come.

    The article 5 of the World’s Most Ultra-Rare Diseases originally appeared on Fool.com and is written by Sean Williams.

    Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong. The Motley Fool recommends BioMarin Pharmaceutical.

    Copyright © 1995 – 2013 The Motley Fool, LLC. All rights reserved. The Motley Fool has a disclosure policy.

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