Haegarda, the first subcutaneous preventative treatment for patients with hereditary angioedema (HAE), is now available in American pharmacies. The drug, developed by pharmaceutical giant CSL Behring, was approved for routine prophylaxis to prevent HAE attacks in adults and adolescents by the US Food and Drug Administration (FDA) on June 22nd 2017.
HAE is a rare genetic condition caused by deficient or dysfunctional C1 Esterase Inhibitor (C1-INH), a protein found in the blood which helps the body control inflammation. Patients with HAE experience an accumulation of fluid in the body tissues, which can lead to significant swelling, known as angioedema. Symptoms of the condition include diarrhea, nausea and extreme pain. It can be potentially life threatening, particularly if attacks affect the face or throat, which can lead to closure of the airways.
The new drug from CSL Behring is the only subcutaneous prophylactic treatment approved in the US for routine prevention of HAE attacks. The plasma-derived concentrate of C1-INH works by restoring protein levels to above 40 percent of the normal levels - the amount associated with reduced risk of HAE attacks.
The availability of Haegarda in US pharmacies was welcomed by the US Hereditary Angioedema Association, the president of which, Anthony J. Castaldo, stated: "Patient care in the United States takes an important step forward with the approval of this subcutaneous treatment for preventing HAE attacks."
Haegarda prevented 95 percent of HAE attacks and nearly eliminated the use of on-demand rescue medication in 99 percent of patients involved in the clinical study. A number of other medications are currently available to treat HAE, including other C1-inhibitors, such as Cinryze (C1 esterase inhibitor); plasma kallikrein inhibitors, for example Kalbitor (ecallantide); and bradykinin receptor antagonists such as Firazyr (icatibant).
In related HAE news, in March this year, Attune Pharmaceuticals announced positive data from its recent pre-clinical studies for ATN-249, an oral plasma kallikrein inhibitor to counter C1-INH deficiencies in patients with HAE. The biotechnology firm hopes to begin Phase 1 clinical development of ATN-249 during the summer.
For more information on Haegarda, visit http://www.haegarda.com/
