Andembry (garadacimab) / CSL Behring - LARVOL DELTA

Public Access to ANDEMBRY (garadacimab) Expands Across Canada for the Prevention of Hereditary Angioedema (HAE) Attacks (Canada Newswire) - "The expanded public access represents an important milestone for eligible Canadians living with HAE and their families, providing access to an additional treatment option for this rare and potentially life-threatening swelling. Quebec became the first province in Canada to publicly reimburse ANDEMBRY. Effective July 30, 2026, ANDEMBRY is reimbursed by the Régie de l'assurance maladie du Québec (RAMQ) for eligible Quebec patients aged 12 years or older with Type I or Type II HAE who experience attacks that significantly interfere with their daily activities. In Alberta, public coverage for ANDEMBRY is available effective September 1, 2026, through the Alberta Drug Benefit List (ADBL) via Special Authorization."

Reimbursement • Hereditary Angioedema

Current and future therapies for bradykinin-mediated angioedema (PubMed, Dermatologie (Heidelb)) - "On-demand treatment options include plasma-derived and recombinant C1 inhibitor (C1INH) concentrates, the bradykinin B2 receptor antagonist icatibant, and, more recently, the first orally available plasma kallikrein inhibitor, sebetralstat...LTP therapies include subcutaneous and intravenous C1INH preparations, the oral kallikrein inhibitor berotralstat, the anti-kallikrein monoclonal antibody lanadelumab, the factor XIIa inhibitor garadacimab, and the antisense oligonucleotide donidalorsen. Currently under development are the oral bradykinin B2 receptor antagonist deucrictibant, which is intended for both on-demand treatment and long-term prophylaxis in different formulations, long-acting antibodies, such as navenibart, and CRISPR/Cas9-based gene-editing therapies, such as NTLA-2002 with potential functional curative properties. In particular, orally available and long-acting therapies are expected to improve adherence, self-management, and quality of life in..."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety Study in Subjects ? 12 Years of Age With Hereditary Angioedema Switching to Garadacimab (clinicaltrials.gov) - P4 | N=18 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Garadacimab and the future of hereditary angioedema prophylaxis: a step upstream in the bradykinin pathway. (PubMed, Ann Med Surg (Lond)) - "Adverse events were generally mild, including headache, nasopharyngitis, and injection-site reactions. The approval of garadacimab represents an important advancement in HAE prophylaxis, offering a novel upstream mechanism, convenient dosing, and potential improvement in quality of life of the patients."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gastrointestinal Disorder • Hereditary Angioedema • Infectious Disease • Pain

Zymogen and Protease Factor XII Functions Each Contribute to Venous Thrombosis and Vascular Stasis in a Murine Model of Sickle Cell Disease (ISTH 2026) - "FXIIa activity was inhibited by a monoclonal antibody (CSL312)...Ongoing studies will dissect the molecular and cellular mechanisms, as well as model-dependent characteristics underlining these observations, and determine how FXII is activated in SCD. Table or Figure Upload (1) Figure 1A-B Page 2 Table or Figure Upload (2) Figure 2 Page 3 DOI*10.1016/j.rpth.2026.103811"

Preclinical • Cardiovascular • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Thrombosis

Advancing beyond stand-alone NAb assays: perspectives and regulatory impact of applying integrated immunogenicity assessment in drug development. (PubMed, Bioanalysis) - "Two case studies involving low-risk monoclonal antibodies-garadacimab and clazakizumab-are presented, in which validated standalone NAb assays were available but not relied upon for primary clinical interpretation due to limited sensitivity and lack of added clinical value. In both cases, this approach enabled clear differentiation between detectable but clinically irrelevant immunogenicity and immunogenicity with clinical consequence and was accepted by regulatory authorities. These examples illustrate how integrated immunogenicity assessments can replace standalone NAb assays while remaining scientifically rigorous, clinically informative, and aligned with regulatory expectations."

Journal

Long-Term Integrated Safety and Efficacy of Garadacimab for Hereditary Angioedema Prophylaxis. (PubMed, Adv Ther) - P2, P3 | "These integrated data, representing the longest garadacimab exposure reported to date, confirm the favorable long-term safety profile of garadacimab (exposure ≤ 5.5 years), and durable efficacy with sustained protection against HAE attacks (exposure ≤ 3.2 years)."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab (clinicaltrials.gov) - P4 | N=18 | Active, not recruiting | Sponsor: CSL Behring | Recruiting ➔ Active, not recruiting | N=30 ➔ 18

Enrollment change • Enrollment closed • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab (clinicaltrials.gov) - P4 | N=30 | Recruiting | Sponsor: CSL Behring | Active, not recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Garadacimab for the long-term prophylaxis of hereditary angioedema. (PubMed, J Dtsch Dermatol Ges) - "Based on this and earlier clinical data, garadacimab has the potential to bring patients closer to achieving the guideline-recommended goals of disease control and normalization of life. Here, we provide an overview of the results from the garadacimab clinical development program."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Monoclonal antibodies in the management of hereditary angioedema. (PubMed, Expert Opin Biol Ther) - "This review focuses on the evaluation of three monoclonal antibodies developed for LTP of HAE, namely lanadelumab (Takhzyro®), garadacimab (Andembry®), and navenibart, in this order. The transition from older non-targeted LTP options to the use of monoclonal antibody-based therapies has fundamentally changed the treatment landscape by offering a safe, effective, and highly targeted solution. Although having different pharmacological characteristics, these LTP options share the same objective: to achieve complete disease control."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Status of Current Clinical Trials on Therapy for Hereditary Angioedema (IMMUNOLOGY 2026) - "The sole drug in phase 4 is CSL312 (Garadacimab), a fully human IgG4 monoclonal antibody targeting activated factor XIIa. Drugs in phase 3 include: NTLA-2002, a single-dose intravenous gene therapy targeting inactivation of the KLKB1 gene; Navenibart, an IgG1 monoclonal antibody inhibiting activated kallikrein; OCTA-C1-INH, a virus-inactivated, nanofiltrated, highly purified concentrate of C1-INH derived from pooled human plasma; ADX-324, an siRNA therapy to reduce hepatic production of prekallikrein (PKK); Donidalorsen, an antisense oligonucleotide targeted against hepatic PKK mRNA; Sebetralstat and berotralstat, both plasma kallikrein inhibitors that reduce production of bradykinin; and deucrictibant, a competitive bradykinin B2 receptor antagonist. Advances in gene therapy, biologics, RNA interference therapeutics, and improved replacement strategies hold promise for transforming both rescue and prophylactic management for HAE. Ongoing evaluation of safety,..."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Hereditary Angioedema • AVEN

Factor XII in Thrombosis and Thromboinflammation: From Molecular Biology to Clinical Translation. (PubMed, Int J Mol Sci) - "Most notably, garadacimab, a monoclonal anti-FXIIa antibody, has completed phase 3 trials and received regulatory approval for hereditary angioedema (HAE) prophylaxis, where it markedly reduces attack frequency with a favorable safety profile. This review summarizes current knowledge on FXII biology and evaluates its translational potential as a novel target for anticoagulant and anti-inflammatory therapies."

Journal • Review • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Hereditary Angioedema • Immune Modulation • Immunology • Inflammation • Ischemic stroke • Thrombosis • Vascular Neurology

Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab (clinicaltrials.gov) - P4 | N=30 | Active, not recruiting | Sponsor: CSL Behring | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Treatment Outcomes with Lanadelumab Every 2 Weeks and Garadacimab are Very Similar in Patients with Hereditary Angioedema. (PubMed, Drugs R D) - No abstract available

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Response to "Treatment Outcomes with Lanadelumab Every Two Weeks and Garadacimab Are Very Similar in Patients with Hereditary Angioedema". (PubMed, Drugs R D) - No abstract available

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Human Factors Engineering Evaluation of Garadacimab Administration Via Pre-Filled Autoinjector/Pen For Long-Term Prophylaxis in Hereditary Angioedema (AAAAI 2026) - "Conclusions Simulated administration using the pre-filled autoinjector/pen was successfully completed by the majority of intended users without prior training. Administration period was short (mean injection time 3.1 seconds, mean hold time 12.2 seconds), and participants found the device easy to use."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Safety and Efficacy in Patients With Hereditary Angioedema After Switching to Garadacimab: Interim Analysis of a Phase 4, Open-Label Study (AAAAI 2026) - P4 | "Results Six patients (data cutoff July 31, 2025; 3 female) switched to garadacimab from lanadelumab SC (n=3) or plasma-derived C1-esterase inhibitor SC (n=3)...No HAE attacks were reported when receiving garadacimab LTP. Conclusions Garadacimab demonstrated a favorable safety profile and efficacy against HAE attacks from first administration after switching from approved LTP medications."

Clinical • P4 data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Immunology • Infectious Disease • Respiratory Diseases

CSL312_4002: Safety Study in Subjects ? 12 Years of Age With Hereditary Angioedema Switching to Garadacimab (clinicaltrialsregister.eu) - P4 | N=5 | Recruiting | Sponsor: CSL Behring LLC

New P4 trial • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema (clinicaltrials.gov) - P3 | N=22 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks (clinicaltrials.gov) - P3 | N=171 | Completed | Sponsor: CSL Behring | Active, not recruiting ➔ Completed

Trial completion • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Donidalorsen (Dawnzera) for prevention of hereditary angioedema attacks. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Garadacimab-gxii-A Novel Prophylactic Treatment for Hereditary Angioedema: A Drug Review. (PubMed, Ann Pharmacother) - "Garadacimab reduces the frequency of HAE attacks with a safety profile comparable with existing prophylactic treatments. Its mechanism of targeting activated factor XII offers an alternative approach to HAE management. Ongoing monitoring and post-marketing data may further define its role in clinical practice."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

A novel role for SIX1 and EYA2 and a novel EYA2 inhibitor in myeloid leukemogenesis (ASH 2025) - "SIX1, together with itscofactor Eyes Absent 2 (EYA2), a protein tyrosine phosphatase, is involved in cell proliferation andembryogenesis and transcriptionally activates developmental genes... We first determined that increased SIX1 expression is associated with worse event free survival (EFS) andoverall survival, while EYA2 correlates solely with EFS using the Therapeutically Applicable Research toGenerate Effective Treatments (TARGET) database, which exploits a multiomic approach to evaluateprofiles of multiple cancers. We next queried the Broad Institute Cancer Dependency Map and identifiedincreased SIX1 expression in SHI-1 and OCI-M2 AML cell lines; neither displayed increased EYA2expression. SIX1 expression in these cells was validated by immunoblot, and shRNA knockdown of SIX1reduced proliferation in both cell lines."

Acute Myelogenous Leukemia • Esophageal Cancer • Hematological Malignancies • Leukemia • Ovarian Cancer • KMT2A • MLLT10 • SIX1

Comparative efficacy and safety of FDA-approved prophylactic treatments for hereditary angioedema, including garadacimab, haegarda, lanadelumab, and berotralstat: A network meta-analysis (ASH 2025) - "This network meta-analysis offers a detailed comparison of the efficacy and safety profiles ofFDA-approved prophylactic treatments for HAE. Garadacimab emerged as the most effective treatment,followed by Haegarda, Lanadelumab, and Berotralstat. These findings emphasize the importance ofevaluating both efficacy and safety when selecting the optimal treatment for HAE patients, withsignificant improvements in quality of life observed in the treatment group."

Retrospective data • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hereditary Angioedema • Novel Coronavirus Disease