RARE Daily

CSL Reports Positive Top-Line Phase 3 Results for Its Preventive Treatment in Patients with HAE

August 18, 2022

CSL reported positive top-line phase 3 results for garadacimab, 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.

Photo: Bill Mezzanotte, executive vice president, head of R&D, and chief medical officer for CSL

The study met its primary and secondary efficacy objectives and also demonstrated favorable safety and tolerability. CSL aims to begin filing with global health authorities next 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 hereditary angioedema (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 Bill Mezzanotte, executive vice president, head of R&D, and chief medical officer for CSL.

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 (FXIIa mAb) currently in phase 3 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. Garadacimab uniquely inhibits the plasma protein, FXIIa. When FXIIa is activated, it initiates the cascade of events leading to edema formation. By targeting FXIIa, garadacimab inhibits the HAE cascade at its origin as compared with other HAE therapies that target downstream mediators. Garadacimab was discovered and optimized by scientists at CSL’s Bio21–based research site.

Orphan-drug designation for garadacimab as an investigational therapy for hereditary angioedema has been granted by both the U.S. Food and Drug Administration and the European Medicines Agency.

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.

Author: Rare Daily Staff

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