Garadacimab (CSL312), an experimental hereditary angioedema (HAE) treatment, showed favorable safety and tolerability in a phase 3 clinical trial, which also met its primary and secondary efficacy outcome measures.
CSL, the developer of garadacimab, hopes to start filing with global health authorities for full approval of the treatment at the end of the fiscal year.
“These results underscore our belief that garadacimab has the potential to become a transformative first-in-class therapy for people living with HAE,” Bill Mezzanotte, MD, executive vice president, head of research and development, and chief medical officer at CSL, said in a press release.
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The multicenter, double-blind, randomized, placebo-controlled, parallel-arm clinical trial investigated the safety and efficacy of subcutaneous garadacimab in the prophylactic treatment of HAE.
Read more about HAE prophylaxis
The trial included 64 participants with C1-inhibitor-HAE, aged 12 years and above, who received either garadacimab or a placebo once a month for up to 6 months.
The primary outcome measure was the time-normalized number of HAE attacks during the treatment period. Secondary outcome measures included the change in the rate of HAE attacks, the time-normalized number of attacks requiring on-demand treatment, and the number of patients with adverse events, serious adverse events, antidrug antibodies, and clinically significant abnormalities on laboratory assessments.
According to the press release, the company will present the full results from the study at a scientific congress and publish them in a peer-reviewed journal.
Garadacimab is a novel, first-in-class monoclonal antibody inhibiting factor XIIa, which, when activated, initiates a cascade of events leading to the formation of edema. The experimental treatment has already been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of HAE.
References
CSL announces positive top-line phase 3 results for garadacimab as preventive treatment in patients with hereditary angioedema (HAE). News release. CSL Limited; August 17, 2022.
CSL312 (garadacimab) in the prevention of hereditary angioedema attacks. ClinicalTrials.gov. December 7, 2020. Updated August 3, 2022. Accessed August 19, 2022.
