CSL's Garadacimab, a First-in-Class Factor XIIa Inhibitor, Receives FDA and EMA Filing Acceptance

Rhea-AI Summary

CSL (ASX:CSL; USOTC:CSLLY) announced that the FDA has accepted the company's Biologics License Application for garadacimab as a once-monthly prophylactic treatment for hereditary angioedema (HAE). The European Medicines Agency has also accepted the submission for CSL's Marketing Authorization Application for garadacimab. If approved, garadacimab would become the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa), with orphan-drug designation granted by both the FDA and EMA. The BLA and MAA are supported by data from the pivotal VANGUARD trial, with the ongoing open-label extension evaluating the long-term safety and efficacy of garadacimab in patients with HAE.

These regulatory milestones bring CSL one step closer to delivering on our promise to patients with a first-in-class recombinant monoclonal antibody for people living with HAE, a community CSL has been serving for over 40 years, in the U.S. and European Union.

KING OF PRUSSIA, Penn., Dec. 14, 2023 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for garadacimab (CSL312) as a once-monthly prophylactic treatment for hereditary angioedema (HAE). The company also announced the European Medicines Agency (EMA) has accepted the submission for CSL's Marketing Authorization Application (MAA) for garadacimab. If approved, garadacimab would become the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa).

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 Emmanuelle Lecomte Brisset, Pharm D, Senior Vice President and Global Head of Regulatory Affairs at CSL. "We believe that garadacimab has the potential to become a promising therapy in the prevention of HAE attacks and we look forward to working closely with global health regulators throughout the review process."

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 The Lancet (April 2023). The ongoing open-label extension of the VANGUARD study evaluates the long-term safety and efficacy of garadacimab (200 mg monthly) in patients with HAE.

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: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL.

For more information about CSL, visit www.CSL.com.

Media Contact
Valerie Bomberger
CSL
Office: +1 610-291-5388 
Mobile: +1 267-280-3829 
Email: valerie.bomberger@cslbehring.com 

In Australia: 
Kim O'Donohue
CSL
Email: Kim.ODonohue@csl.com.au
+61 449 884 603

Jimmy Baker
CSL
Email: Jimmy.Baker@csl.com.au
+61 450 909 211

View original content to download multimedia:https://www.prnewswire.com/news-releases/csls-garadacimab-a-first-in-class-factor-xiia-inhibitor-receives-fda-and-ema-filing-acceptance-302016048.html

SOURCE CSL

FAQ

What is the status of CSL's Biologics License Application for garadacimab?

The FDA has accepted the company's Biologics License Application for garadacimab as a once-monthly prophylactic treatment for hereditary angioedema (HAE).

What is the significance of the European Medicines Agency accepting the submission for CSL's Marketing Authorization Application for garadacimab?

The acceptance by the European Medicines Agency indicates progress in the potential approval of garadacimab for the treatment of HAE in the European Union.

What is the potential impact of garadacimab becoming the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa)?

Garadacimab would be the first treatment for HAE in the U.S. and EU to target activated Factor XII (FXIIa), offering a novel approach to addressing the condition.

What data supports the BLA and MAA for garadacimab?

The BLA and MAA for garadacimab are supported by data from the pivotal, multicenter, randomized, double-blind, parallel-group VANGUARD trial, as well as the ongoing open-label extension evaluating the long-term safety and efficacy of garadacimab in patients with HAE.