Rare Disease Report

Haegarda Reduces HAE Attacks by 98% in Most Severe Cases

OCTOBER 30, 2017
James Radke
CSL Behring’s Haegarda (C1 Esterase Inhibitor Subcutaneous [Human]) continues to exhibit effectiveness in treating hereditary angioedema (HAE).

At the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Boston, MA, Timothy Craig, D.O., Professor of Medicine and Pediatrics at Pennsylvania State University Medical School in Hershey, Pennsylvania, presented data showing the prophylactic use of Haegarda reduced the number of attacks by 98% in patients with the most severe form of HAE.

HAE is a genetic disorder in which patients experience rapid and uncontrolled swelling, most noticeably their extremities, face, and abdomen. It is caused by deficient or dysfunctional C1-inhibitor, a blood protein involved in the inflammation process. HAE attacks can be life threatening if swelling occurs near patient’s airways.

Haegarda was approved by the U.S. Food and Drug Administration (FDA) in June of this year as a prophylactic agent to prevent HAE attacks. The approval of Haegarda was based on a phase 3 COMPACT clinical trial in which 90 patients with symptomatic HAE received subcutaneous doses of Haegarda (40 IU/kg or 60 IU/kg) or placebo twice a week for 16 weeks in a cross-over designed trial. The study observed that patients in both treatment groups had significantly fewer attacks compared to placebo (mean reductions of HAE attacks of 55% and 84% for 40 IU/kg or 60 IU/kg, respectively). Based on that study, the dose of 60 IU/kg (twice weekly) was approved for routine prophylaxis to prevent HAE attacks.

In the data presented at ACCAI, Craig evaluated a subgroup of 21 patients with very frequent HAE attacks (defined as at least 4 HAE attacks per month) among the 43 patients who received 60 IU/kg in the COMPACT study.

The data showed that during a 16-week treatment period, HAE attacks dropped 98% when patients received Haegarda compared to placebo. Further, the data hypothesized that persons with severe HAE who normally would average 70 HAE attacks per year would on experience 6 attacks in a year if taking Haegarda as a prophylactic agent.

In a news release, Craig said, "Approximately 30% of HAE patients experience weekly attacks, and the majority of those attacks can be debilitating to daily living," adding,  "These data demonstrate the ability of Haegarda to effectively prevent HAE attacks in patients who have severe and frequent HAE attacks."

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