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Hereditary Angioedema Orphan Drug Market And 2013 Business Developments

February 10, 2013

There are currently five drugs that share the international Hereditary Angioedema (HAE) therapeutic market landscape. The US HAE market is crowded because there are only 6,000 patients with 4 products available.

Four Orphan Drugs

  1. Dyax’s Kalbitor (Ecallantide) (US only) for acute HAE administered through subcutaneous injection
  2. CSL Behring‘s Berinert (C1-esterase-inhibitor, human, pasteurized ) (US & EU) for acute HAE & administered through IV infusion
  3. Shire’s Firazyr (Icatibant) (US & EU) for acute HAE & administered through subcutaneous injection
  4. ViroPharma’s Cinryze (C1 esterase inhibitor (human)) (US & EU) for HAE prophylaxis & acute HAE (EU only) & administered through IV infusion.

One Non-Orphan Drug

  1. Swedish Orphan Biovitrum’s (SOBI) Ruconest (recombinant human C1 inhibitor) (EU only) for HAE prophylaxis.

Until 2008, Berinert is the only therapy approved in Europe, while in the US there are no approved therapies. Since 2008, 5 HAE therapies are approved during 2008 – 2011, consisting of Cinryze, Firazyr, Berinert and Kalbitor in the US, and Cinryze, Firazyr and Ruconest in Europe. By  2019, according to Global Data, the following approvals are expected : Kalbitor (Europe), Rhucin (US), Firazyr (Japan), Kalbitor (Japan), and Cinryze (Japan). Global Data values the “global HAE therapeutics market at $113.8 million in 2011 with a forecast growth at a CAGR of 16.5 % to reach $385 million in 2019.”

According to a article,  ViroPharma’s Cinryze has approximately 60% of the HAE market share (measured by sales numbers).

2013 Business Developments

Currently, Dyax’s Kalbitor is approved of and marketed only in the United States. Since the beginning of 2013, Dyax has formed 2 strategic partnerships to obtain regulatory approval and commercialization of Kalbitor to increase the drug’s market scope. The first partnership is announced in January 2013, with Dyax teaming up with Novellus Biopharma (Columbia) for the development and commercialization of Kalibitor in certain regions in Latin America : Argentina, Brazil, Chile, Colombia, Mexico, and Venezuela. The second partnership is announced on Thursday, February 7, 2013, with Dyax and CVie Therapeutics developing and commercializing Kalbitor in China, Hong Kong, and Macau.

ViroPharma announces in January 2013 the online publication in The Journal of Pediatrics, of data demonstrating that the use of Cinryze in pediatric patients provides relief from symptoms of HAE attacks and reduction in the rate of attacks.  HAE Canada announces on February 6, 2013, that they have received word from Canadian Blood Services (CBS) that effective April 1, 2013, Cinryze will be available as the second supplier. CBS will officially cover the cost and distribution of Cinryze to the Canadian market.

CSL Behring announces in January 2013 the launch of a financial assistance program designed to cover patients’ out-of-pocket expenses for Berinert.

An article published online February 4, 2013 in the peer-reviewed, open access journal PLOS ONE,  reviews the results from the Icatibant Outcome Survey, which is funded and supported by Shire Human Genetic Therapies (HGT). The objective of this outcome survey is to examine the impact of the timing of Icatibant/Firazyr administration on the duration and resolution of HAE type I and II attacks. The conclusion is that the adminstering of Icatibant, particularly within the first hour of attack onset, significantly reduces attack duration and time to attack resolution. The following is the citation for this FREE journal article :

Maurer M, Aberer W, Bouillet L, Caballero T, Fabien V, et al.  (2013) Hereditary Angioedema Attacks Resolve Faster and Are Shorter after Early Icatibant Treatment. PLoS ONE 8(2): e53773. doi:10.1371/journal.pone.0053773.

In the February issue of the journal Expert Opinion on Orphan Drugs, a fascinating and thorough article is published that summarizes and reviews the medicinal products available for the treatment of HAE. The article outlines and reviews the many choices for HAE disease management. Here is the citation :

Farkas, Henriette.  Orphan drugs for the treatment of hereditary angioedema. Expert Opinion on Orphan Drugs. 20131:2, 141-156.

2013 US HAEA National Patient Summit – September 27 – 28, 2013, Orlando, Florida

HAE Day May 16, 2013

HAEi (International Patient Organization for C1-Inhibitor Deficiencies; a network of National HAE Patient Associations)

US Hereditary Angioedema Association

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

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