A new global phase 2 study has demonstrated the potential of garadacimab, a fully human monoclonal antibody targeting factor XII, to achieve complete disease control in some patients with hereditary angioedema (HAE). The study, published in The Lancet Haematology, found that once-monthly dosing for over 2 years was well tolerated, efficacious, and improved quality of life.
“We report the long-term efficacy and safety data for garadacimab for the prevention of attacks in patients with hereditary angioedema with C1-INH deficiency over the open-label extension period (treatment period 2; TP2) of the phase 2 study,” the authors wrote. “We additionally describe patient-reported, investigator-reported, and health-related quality-of-life (HRQoL) outcomes for the full phase 2 study, encompassing both treatment periods.”
The randomized, double-blind, placebo-controlled study assessed the efficacy and safety of garadacimab in terms of patient-reported, investigator-reported, and quality-of-life outcomes over a 12-week treatment period and a 44-week or longer open-label extension period.
A total of 38 patients with HAE completed the treatment period and were assigned a range of doses of garadacimab for the open-label extension period. Two patients ultimately discontinued treatment, one for pregnancy and another at the patient’s request.
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The primary endpoint was the number of HAE attacks in patients receiving garadacimab 200 mg or 600 mg. Secondary endpoints included the number and percentage of patients responding to treatment, the proportion who were attack-free, those with mild, moderate, or severe attacks, use of rescue medication, adverse events, and patient-reported quality of life outcomes.
The results revealed that monthly garadacimab as prophylaxis was well tolerated, highly effective in preventing HAE attacks, and associated with improved quality of life. The authors noted that a phase 3 open-label extension study examining the efficacy, safety, and quality of life impact of garadacimab is already underway.
