CSL is on track to file for regulatory approvals of garadacimab

Australian biotechnology company CSL Limited (ASX: CSL) has announced positive top-line Phase III results for garadacimab (CSL312), the company’s investigational first-in-class monoclonal antibody inhibiting Factor XIIa being developed as a long-term preventive treatment for patients with hereditary angioedema (HAE).

The study met its primary and secondary efficacy objectives and also demonstrated favorable safety and tolerability. CSL said it aims to begin filing with global health authorities at the end of the current fiscal year for full approval.

The multicenter, double-blind, randomized, placebo-controlled, parallel-arm study (also known as VANGUARD) evaluated the efficacy and safety of monthly subcutaneous garadacimab administration in the prevention of HAE attacks compared to the placebo for six months. Full results from the study will be presented at an upcoming scientific congress and published in a peer-reviewed journal.

“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, 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 III study in the coming months.”

Orphan-drug designation for garadacimab as an investigational therapy for hereditary angioedema has been granted by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the company noted.

CSL is also investigating garadacimab for other indications, beyond HAE, where FXIIa inhibition may play an important role in improving clinical outcomes, including pulmonary fibrosis.