Health Canada Has Authorized CSL's ANDEMBRY® (garadacimab) as Once-Monthly Treatment for Hereditary Angioedema (HAE)

• ANDEMBRY is the first and only once-monthly treatment targeting factor XIIa to prevent recurrent attacks in people with HAE
• The authorization of ANDEMBRY expands CSL's HAE franchise and underscores the company's commitment to delivering innovative medicines to the HAE community

OTTAWA, Canada, Aug. 8, 2025 /CNW/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that Health Canada has granted a marketing authorization for ANDEMBRY^® (garadacimab) for routine prevention of attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. ANDEMBRY is a monoclonal antibody designed to target activated FXII (FXIIa), a plasma protein at the top of the HAE cascade that plays a key role in attacks of swelling in people with HAE. ANDEMBRY reinforces CSL's decades-long commitment to advancing innovation for the HAE community and is authorized with a prefilled pen for subcutaneous self-injection.

"As the first monoclonal antibody discovered and developed entirely by CSL, ANDEMBRY is a reflection of our commitment to delivering innovative treatment options for the patients who need them most," said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. "This approval marks a major step forward in HAE care, building on our more than 40-year legacy in HAE research and treatment optimization to offer people living with this life-threatening condition long-term control over their disease along with a convenient administration method."

HAE is a rare, chronic, and potentially life-threatening genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks of HAE are often painful and can affect multiple sites of the body, including the abdomen, larynx, face, and extremities. ANDEMBRY inhibits the top of the HAE cascade while other HAE therapies target downstream mediators.

"Health Canada's authorization of ANDEMBRY represents a significant advancement in the treatment of HAE," said Dr. Stephen Betschel, Chair of the Canadian Hereditary Angioedema Network. "This new therapy, which targets factor XIIa, offers a promising approach to preventing HAE attacks. By addressing the underlying mechanisms of HAE, ANDEMBRY provides an important option for patients to reduce the frequency of their attacks of angioedema. Having a new treatment option will further enhance our ability to help our patients manage their HAE effectively."

The Health Canada authorization is based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial evaluating the efficacy and safety of ANDEMBRY. The pivotal study (The Lancet, April 2023) demonstrated that treatment with ANDEMBRY:

• Reduced HAE attacks by a median of more than 99 percent and a reduced least squares mean of 89.2 percent, compared to placebo.
• Led to 62 percent of ANDEMBRY-treated patients remaining attack-free throughout the treatment period.
• The most common adverse reactions in the pivotal trial were injection site erythema, injection site bruising, injection site pruritus, injection site urticaria, headache, and abdominal pain.

A published interim analysis (Allergy, Oct 2024) of the ongoing open-label extension study (median ANDEMBRY exposure of 13.8 months) showed that ANDEMBRY has a favorable long-term safety profile and provides sustained reductions in HAE attacks.

"We are pleased to see Health Canada's authorization of ANDEMBRY for HAE and look forward to its availability in Canada," said Michelle Cooper, President, HAE Canada. "As the first treatment to target the top of the HAE cascade, this therapy represents a significant advancement in preventing HAE attacks. We believe it will provide meaningful benefits to the HAE community in Canada."

This regulatory approval for ANDEMBRY is another crucial step in building toward the global availability of ANDEMBRY, which was recently approved in Australia, the United Kingdom (UK), the European Union (EU), Japan, Switzerland, and the United States (US).

About HAE 
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death.

About ANDEMBRY
ANDEMBRY is a novel monoclonal antibody inhibiting factor XIIa (anti-FXIIa mAb) that has completed the Phase 3 pivotal study as a once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. ANDEMBRY is CSL's first homegrown recombinant monoclonal antibody to gain Health Canada approval. It was discovered and optimized by scientists at CSL's Bio21-based research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility. ANDEMBRY uniquely inhibits the plasma protein, FXIIa. FXII is the first protein activated in the HAE pathway, initiating the cascade of events leading to an HAE attack. By targeting activated FXII (FXIIa), ANDEMBRY inhibits this cascade at the top as compared to other HAE therapies that target downstream mediators.

About the VANGUARD Trial 
The multicenter, randomized, double-blind, parallel-group VANGUARD trial evaluated the efficacy and safety of ANDEMBRY, a first-in-class monoclonal antibody, as a prophylactic treatment for patients with hereditary angioedema. Patients aged 12 years and older with HAE type I or II underwent screening and a run-in study period to verify a baseline attack rate. Patients were randomized 3:2 to receive a loading dose of 400 mg followed by 200 mg of ANDEMBRY monthly (n=39) or volume matched placebo monthly (n=25) subcutaneously. After the six-month treatment period, patients were given the opportunity to continue into the open-label extension study, which is currently ongoing.

The ongoing open-label extension of the Phase 3 VANGUARD study is evaluating the long-term safety and efficacy of ANDEMBRY (200 mg monthly) for the prophylactic treatment of hereditary angioedema attacks.

Important Information for Canada
For more information and a complete risk/benefit profile, please refer to the Product Monograph available here.

About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat Hemophilia 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 more information about CSL, visit www.CSL.com.

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SOURCE CSL