Haegarda from CSL Behring is just one of two drugs approved to prevent attacks and is currently the only subcutaneous treatment.
Patients with the rare genetic disease hereditary angioedema (HAE) suffer from attacks of swelling (edema) in different parts of their bodies, including their hands, feet, faces and airways. The disease is caused by a lack of or incorrectly functioning C1-INH protein, which is involved in the control of inflammation. It is potentially life-threatening and occurs in 1 in 10,000 to 1 in 50,000 people.
CSL Behring’s recently FDA approved (June 22, 2017) C1 Esterase Inhibitor Subcutaneous [Human]) drug Haegarda for routine prophylaxis use in adolescent and adult patients to prevent hereditary angioedema (HAE) attacks is now available in the US and is the sixth treatment for the disease, but only the second preventive therapy. The other drug for prevention of attacks, Shire’s Cinryze, is also a C1 esterase inhibitor [human], but is not administered subcutaneously.
Haegarda, which replaces the deficient or dysfunctional natural C1-INH, was approved by FDA based on the results of a clinical trial involving 90 patients who received either a placebo or 40 or 60 IU/kg subcutaneous doses of Haegarda twice weekly for 16 weeks. The patients that received the drug suffered from fewer attacks than those that received the placebo. Specifically, at the approved dose of 60 IU/kg, HAEGARDA was shown to reduce the number of HAE attacks by a median of 95 percent relative to placebo, according to the company.
"Many patients experience the heavy burden of HAE attacks on their daily lives. Now, the HAE community has a new treatment option clinically proven to significantly reduce the frequency of HAE attacks," said Donald Levy, M.D., Professor of Medicine and Assistant Allergy Training Director, University of California Irvine.
“We’re proud to have helped advance care for the HAE community by offering the first subcutaneous prophylactic treatment option for patients,” said Bill Campbell, Senior Vice President and General Manager, North America, CSL Behring. “With the availability of HAEGARDA, patients affected by HAE and physicians at the frontlines of managing this debilitating condition have access to a new therapy with the potential to transform patients’ lives.”
