CSL has announced positive top-line results from a Phase 3 study of its investigational candidate garadacimab in patients with hereditary angioedema.
The company said the study met its primary and secondary efficacy objectives and also demonstrated favourable safety and tolerability. It said it will shortly begin filing with global regulatory authorities.
The VANGUARD study evaluated the efficacy and safety of monthly subcutaneous garadacimab administration in the prevention of HAE attacks compared to the placebo for six months.
HAE is a rare, genetic and potentially life-threatening condition that causes painful, debilitating and unpredictable episodes of swelling of the abdomen, larynx, face and extremities, among other areas of the body. Garadacimab is a novel Factor XIIa-inhibitory monoclonal antibody
“These results underscore our belief that garadacimab has the potential to become a transformative first-in-class therapy for people living with HAE, a patient group that CSL has been serving for many years,” said Dr Bill Mezzanotte, the executive vice president, head of R&D and chief medical officer for CSL.
“CSL’s promise to patients guides us to meet their unmet need by pursuing the type of disruptive innovation we believe garadacimab represents. We look forward to sharing the full results of our phase 3 study in the coming months.”