The company also announced the
Garadacimab is a novel, first-in-class, recombinant monoclonal antibody targeting activated FXII. FXIIa is a plasma protein that initiates the kallikrein-kinin cascade of HAE attacks. By targeting FXIIa, garadacimab inhibits this cascade at the top as compared to HAE therapies that target downstream mediators.
'CSL is a company with a deep heritage in developing innovative treatments for the rare disease community, and we are extremely proud that our first homegrown recombinant monoclonal antibody is progressing our commitment to support HAE patients in need,' said
Orphan-drug designation for garadacimab as a therapy for hereditary angioedema has been granted by both the FDA and the EMA.
The BLA and MAA are both supported by data from the pivotal, multicenter, randomized, double-blind, parallel-group VANGUARD trial, which evaluated the efficacy and safety of garadacimab as a prophylactic treatment for patients with HAE. The full results from VANGUARD were published in
About HAE and Garadacimab
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 (anti-FXIIa mAb) that has completed Phase 3 clinical development as a potential 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 FXII is activated, it initiates the cascade of events leading to edema formation. By targeting activated FXII (FXIIa), garadacimab inhibits this cascade at the top as compared to HAE therapies that target downstream mediators.
If approved, patients will have the benefit of an auto injector (pre-filled pen) for convenient administration.
CSL is also investigating garadacimab for other indications beyond HAE, including idiopathic pulmonary fibrosis, where FXIIa inhibition may play an important role in improving clinical outcomes.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL including our three businesses:
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