CSL Behring presents data indicating that, at the approved dose of 60 IU/kg, HAEGARDA®reduced the median number of HAE attacks per month by 98 percent in subjects who had frequent attacks, from a 16-week placebo period to a 16-week treatment period. Additionally, the breakthrough attack rate – extrapolated to one year – was reduced from approximately 70 attacks per year for subjects on placebo to approximately six attacks per year for the same subjects when on HAEGARDA. These findings were presented in an oral abstract session at the 2017 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting.
“Approximately 30 percent of HAE patients experience weekly attacks, and the majority of those attacks can be debilitating to daily living,” said Timothy Craig, D.O., Professor of Medicine and Pediatrics, Pennsylvania State University Medical School, Hershey, Pennsylvania. “These data demonstrate the ability of HAEGARDA to effectively prevent HAE attacks in patients who have severe and frequent HAE attacks.”
The presented subgroup analysis was based on data from the Phase III COMPACT trial. The subgroup analysis evaluated 21 severely impacted patients of the 43 study subjects who received 60 IU/kg of subcutaneous C1-esterase inhibitor (C1-INH) and a corresponding placebo over a 16-week treatment period each. High attack frequency was defined as experiencing at least one HAE attack per week (on average, greater than or equal to four attacks per month) while on placebo.
“These results reinforce the efficacy we’ve seen across a broader patient population, and add to our body of knowledge by showing that HAEGARDA is also highly effective in reducing the number of attacks in patients severely affected by HAE,” said Debra Bensen-Kennedy, MD, Vice President for Medical Affairs, North America, CSL Behring. “HAE is an extremely debilitating disease, and this continued research and the development of HAEGARDA reflects our commitment to improving patients’ lives.”
HAEGARDA is a self-administered, plasma-derived concentrate of C1-INH injected twice weekly subcutaneously. HAEGARDA targets the root cause of HAE by replacing deficient or dysfunctional C1-INH protein, restoring C1-INH levels above 40 percent, which is proposed to reduce the risk of HAE attacks. Subcutaneous administration of C1-INH builds and maintains steady-state functional C1-INH levels within three to four doses of HAEGARDA.
The U.S. Food and Drug Administration (FDA) approved HAEGARDA on 22 June 2017 for routine prophylaxis to prevent HAE attacks in adolescent and adult patients.
(Source: CSL Behring)