The efficacy and safety of HAEGARDA for routine prophylaxis to prevent HAE attacks were demonstrated in a multicenter, randomized, double-blind, placebo-controlled, crossover study (Study 1), and in a multicenter, randomized, open-label, active treatment-controlled study (Study 2).
Study 1:
The study assessed 90 adult and adolescent subjects with symptomatic HAE type I or II. The median (range) age of subjects was 40 (12 to 72) years; 60 subjects were female and 30 subjects were male. Subjects were randomized to receive either 60 IU/kg or 40 IU/kg HAEGARDA in one 16-week treatment period and placebo in the other 16-week treatment period. Patients self-administered HAEGARDA or placebo subcutaneously 2 times per week. Efficacy was evaluated for the last 14 weeks of each treatment period.
Eligible patients were also able to participate in Study 2 for up to 140 weeks. Approximately half of the subjects enrolled in Study 2 also participated in Study 1 (59/120, 49.2%).
Twice per week S.C. doses of 60 IU/kg or 40 IU/kg HAEGARDA resulted in a significant difference in the time-normalized number of HAE attacks (the rate of attacks) relative to placebo (Table 4). The time normalized number of HAE attacks in subjects dosed with 60 IU/kg was 0.52 attacks per month compared to 4.03 attacks per month while receiving placebo (p