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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Hereditary angioedema


Other Names for this Disease
  • Angioedema, hereditary
  • Deficiency of C1 esterase inhibitor
  • HAE
  • HANE
  • Hereditary angioedema type 1
More Names
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Treatment


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How might hereditary angioedema be treated?

Medical treatment of hereditary angioedema (HAE) consists of preventing attacks and managing acute attacks once they occur. During attacks, patients may require respiratory support. They also may require large amounts of intravenous fluids to maintain hemodynamic stability.[1]

Until recently, no effective agent for acute attacks existed in the United States. Now, however, several agents have been approved, and others are in the midst of the U.S. Food and Drug Administration (FDA) approval process.[1]

In October 2008, the US FDA approved the use of C1-INH (Cinryze) for prophylaxis to prevent attacks. In October 2009, the FDA approved C1-INH (Berinert) for the treatment of acute abdominal and facial angioedema attacks in adolescents and adults with HAE. In December 2009, ecallantide (Kalbitor), a kallikrein inhibitor, was approved for the treatment of acute attacks.[1][2]  In August 2011, the FDA approved Firazyr (icatibant) Injection for the treatment of acute attacks in people ages 18 years and older.  Firazyr can be self-administered through an injection in the abdominal area so patients can treat themselves when they realize they are having an  HAE attack.[3]

An article from the eMedicine Journal provides more detailed information on these medications and other methods of treating HAE at the following link. You may need to register to view the article, but registration is free.
http://emedicine.medscape.com/article/135604-treatment 

The US Hereditary Angioedema Association also provides additional information about treatment of HAE.  
http://www.haea.org/treating-hae/treatments/

Orphanet, a database dedicated to information on rare diseases and orphan drugs, provides guidelines regarding emergency management of hereditary angioedema at the following link.
http://www.orpha.net/consor/cgi-bin/Disease_Emergency.php?lng=EN&stapage=FICHE_URGENCE_A1

Last updated: 11/11/2011

References
  1. Frank MM. Hereditary Angioedema: Treatment & Medication. eMedicine. 2010; http://emedicine.medscape.com/article/135604-treatment. Accessed 1/24/2011.
  2. HAE Treatments. US Hereditary Angioedema Association. http://www.haea.org/treating-hae/treatments/. Accessed 1/24/2011.
  3. FDA News Release. U.S. Food and Drug Administration. August 25, 2011; http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm269616.htm. Accessed 11/11/2011.


Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Hereditary angioedema. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name C1 esterase inhibitor (human)
Trade Name
(Manufacturer Name)
Cinryze®
(ViroPharma Incorporated)
Indication
The FDA has approved this product to be used in this manner.
Routine prophylaxis against angioedema attacks in patients with Hereditary Angioedema (HAE)
More Information about this product Drug Information Portal

Generic Name Icatibant
Trade Name
(Manufacturer Name)
Firazyr
(Shire Orphan Therapies)
Indication
The FDA has approved this product to be used in this manner.
Treatment of acute attacks of hereditary angioedema in adults 18 years of age and older
More Information about this product Drug Information Portal
Medline Plus Health Information

Generic Name Ecallantide
Trade Name
(Manufacturer Name)
Kalbitor
(Dyax Corp)
Indication
The FDA has approved this product to be used in this manner.
Treatment of acute attacks of hereditary angioedema in patients 16 years of age and older
More Information about this product Drug Information Portal