Haegarda (C1 esterase inhibitor subcutaneous [human]) / CSL Behring - LARVOL DELTA

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

Real-world Impact of Long-term Prophylaxis on the Clinical Burden of Patients with Hereditary Angioedema (ACAAI 2025) - "Results A total of 499 patients were included (57 Haegarda [11%], 257 Orladeyo [52%], 185 Takhzyro [37%]) and followed for 11.3 months on average. In these 449 patients, annual HRU remained substantial before and after LTP initiation across settings (inpatient days: 2.4 and 1.7 days [0.3 and 0.2 admissions]; ED: 1.5 and 1.2 admissions; outpatient: 21.0 and 19.3 visits, respectively; Figure ). Conclusion This real-world study suggests that currently available LTP can partly reduce the clinical burden of patients with HAE but need remains for novel therapies that may further alleviate this burden."

Clinical • Real-world • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Metabolic Disorders • Obesity • Pulmonary Disease • Respiratory Diseases

Garadacimab (Andembry) for hereditary angioedema prophylaxis. (PubMed, Med Lett Drugs Ther) - No abstract available

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

Network Meta-Analysis of Pharmacological Therapies for Long-Term Prophylactic Treatment of Patients with Hereditary Angioedema. (PubMed, Drugs R D) - "Overall, garadacimab ranked as the most probable effective treatment among all comparators assessed, with lanadelumab Q2W or subcutaneous C1INH ranking second, across most outcomes."

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

Comorbidities in Canadian patients with hereditary angioedema: a quantitative survey study. (PubMed, Allergy Asthma Clin Immunol) - "This cohort of HAE patients, most of whom were on prophylaxis, reported an increased prevalence of certain comorbidities compared to the general Canadian population. Healthcare professionals should be aware of the potentially increased risk of autoimmune conditions, allergies, and asthma in patients with HAE."

Journal • Allergy • Asthma • Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Gastroenterology • Gastrointestinal Disorder • Hereditary Angioedema • Immunology • Inflammation • Inflammatory Arthritis • Inflammatory Bowel Disease • Lupus • Psoriasis • Psoriatic Arthritis • Pulmonary Disease • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • Seronegative Spondyloarthropathies • Urticaria

Real-World Episodes of Long-Term Prophylaxis for Hereditary Angioedema: a Descriptive Study Using Data from the Consortium of Independent Immunology Clinics (CIIC) (AAAAI-WAO 2025) - "Methods This retrospective study explored LTP outcomes with lanadelumab, berotralstat, and subcutaneous C1 esterase inhibitor (Haegarda) among US patients >2 years old with HAE Type I/II. Conclusions This real-world descriptive analysis of LTP use for HAE provides evidence of the extensive use, treatment persistency and effectiveness of lanadelumab. Robust real-world use together with demonstrated safety and effectiveness provide a basis to support patient-centered treatment decision-making."

Clinical • Real-world • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • STAT3

HEREDITARY ANGIOEDEMA TYPE II WITH NORMAL C4 LEVELS (ACAAI 2024) - "She was initially placed on lanadelumab and icatibant but only saw partial response. She was transitioned to C1 inhibitor replacement therapy with Haegarda and Berinert and saw significant improvement in symptoms...This case demonstrates how complete evaluation should be done when clinical suspicion remains high. Periorbital Edema in Patient with HAE Type II"

Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Hereditary Angioedema • Immunology • Inflammatory Arthritis • Pain • Psoriatic Arthritis • Rheumatology • Seronegative Spondyloarthropathies • Urticaria

An international survey assessing the effects of the duration of attack-free period on health-related quality of life for patients with hereditary angioedema. (PubMed, Orphanet J Rare Dis) - "This study shows that longer attack-free duration has an influential role for better HRQoL in patients receiving LTP. Prolonging the attack-free period is an important goal of therapy and recent advances in LTP have increased attack-free duration. However, opportunities exist for new treatments to further increase attack-free duration and improve HRQoL for all patients with HAE."

HEOR • Journal • Cardiovascular • Complement-mediated Rare Disorders • Gastrointestinal Disorder • Hereditary Angioedema

Garadacimab For Hereditary Angioedema Prophylaxis In Adolescents: Efficacy And Safety From The Phase 3 (VANGUARD) Study And Open-Label Extension (Second Interim Analysis) (AAAAI 2024) - P3 | "Garadacimab demonstrated a favorable safety profile and reduced monthly attack rate in adolescents, consistent with previously reported overall population data."

Clinical • P3 data • P3 data: top line • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Immunology

"and Berinert, Ruconest, Haegarda and now oral Orladeyo do the same." (@670rv)

Evolution of guidelines for the management of hereditary angioedema due to C1 inhibitor deficiency. (PubMed, J Investig Allergol Clin Immunol) - "The treatment goals became more ambitious, from a reduction in the frequency, severity, and duration of attacks to achieving total disease control and normalization of patients´ lives at present. Changes in terminology, such as the introduction of designations as 'first line", and the introduction of patient-reported outcome measures (PROM) to assess patients' perceptions of their self-experienced QoL and well-being, are also relevant and addressed in the document, together with still unmet needs in the management of C1-INH-HAE."

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

Efficacy, pharmacokinetics, and safety of subcutaneous C1-esterase inhibitor as prophylaxis in Japanese patients with hereditary angioedema: Results of a Phase 3 study. (PubMed, Allergol Int) - P3 | "C1-INH (SC) (60 IU/kg twice weekly) was efficacious and well tolerated as a prophylaxis against HAE attacks in Japanese patients with HAE types I or II, which was supported by the increased and maintained C1-INH functional activity. EudraCT Number 2019-003921-99; JapicCTI-205273."

Journal • P3 data • PK/PD data • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hereditary Angioedema

Robotic Gynecological Surgery In A Patient With Hereditary Angioedema. (ASA 2022) - "After expert consultation, prophylactic Icabitant (Firazyr) and C1 esterase inhibitor (Haegarda) were continued. Availability of rescue C1 esterase inhibitor (Berinert) was confirmed...Good multimodal pain management and PONV prophylaxis may aid in recognition of postoperative angioedema. Postoperatively, she was admitted for observation, had no acute attacks, and was discharged the following day."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Gynecology • Hereditary Angioedema • Pain

Prophylactic use of an anti-activated factor XII monoclonal antibody, garadacimab, for patients with C1-esterase inhibitor-deficient hereditary angioedema: a randomised, double-blind, placebo-controlled, phase 2 trial. (PubMed, Lancet) - P2 | "Garadacimab 200 mg and 600 mg every 4 weeks significantly reduced the number of monthly attacks versus placebo and was well tolerated during the study. Garadacimab is an efficacious, subcutaneous prophylaxis in patients with HAE-C1-INH and warrants phase 3 evaluation."

Journal • P2 data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Berotralstat (Orladeyo) for prevention of hereditary angioedema. (PubMed, Med Lett Drugs Ther) - No abstract available

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

Letting the patients speak: an in-depth, qualitative research-based investigation of factors relevant to health-related quality of life in real-world patients with hereditary angioedema using subcutaneous C1 inhibitor replacement therapy. (PubMed, Allergy Asthma Clin Immunol) - "From these interviews, a large number of common themes and concepts emerged: a greater sense of freedom and normalcy, increased productivity, and improved interpersonal relationships while using convenient and effective prophylaxis. These findings provide insights into real-world experiences and the many facets of HRQoL that are important to patients with HAE-C1INH."

Clinical • HEOR • Journal • Real-world evidence • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Depression • Hereditary Angioedema • Mood Disorders • Psychiatry

[VIRTUAL] Hereditary Angioedema Type 1, Pregnancy, and Acute SARS-CoV-2 Infection (CIS 2021) - "She had been maintained on subcutaneous C1-INH (Haegarda®) at 60 IU/Kg with disease control prior to presentation...Her mild clinical course might be attributed to C1-INH therapy that blunted the inflammatory response associated with Covid-19. While further studies are currently being conducted to investigate the effectiveness of C1-INH in decreasing severe inflammation associated with Covid-19, our case highlights the importance of maintaining adequate therapy in HAE patients, particularly during pregnancy."

Cardiovascular • Complement-mediated Rare Disorders • Critical care • Hereditary Angioedema • Immunology • Infectious Disease • Inflammation • Novel Coronavirus Disease • Pain • Respiratory Diseases

Lanadelumab (Takhzyro) for prevention of hereditary angioedema. (PubMed, Med Lett Drugs Ther) - No abstract available

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

Long-term health-related quality of life in patients treated with subcutaneous C1-inhibitor replacement therapy for the prevention of hereditary angioedema attacks: findings from the COMPACT open-label extension study. (PubMed, Orphanet J Rare Dis) - P3 | "Long-term C1-INH(SC) replacement therapy in patients with C1-INH-HAE leads to significant and sustained improvements in multiple measures of HRQoL. Trial registration A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema, NCT02316353. Registered December 12, 2014, https://clinicaltrials.gov/ct2/show/NCT02316353 ."

Clinical • HEOR • Journal • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Depression • Hereditary Angioedema • Mood Disorders • Psychiatry

[VIRTUAL] Despite Prophylactic Treatments, Break-through Attacks Continue among Patients with Hereditary Angioedema (AAAAI 2021) - "The most common medications were lanadelumab-flyo (Takhzyro®, 21%), C1 esterase inhibitor (C1-INH) administered subcutaneously (Haegarda®, 20%), C1-INH administered intravenously (Cinryze®, 18%), and oral androgens (15%). Physicians (78%) believe patients accurately report their attack frequency.  Conclusions Many patients with HAE on prophylactic medication, including newer subcutaneous therapies, still experience attacks and require on-demand medication treatment. Attack rates should be assessed regularly."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Immunology

Long-Term Efficacy of Subcutaneous C1 Inhibitor in Pediatric Patients with Hereditary Angioedema. (PubMed, Pediatr Allergy Immunol Pulmonol) - "Data from pediatric subjects treated with C1INH (SC) for up to 2.55 years and adult subjects revealed similar efficacy. C1INH (SC) is effective and well tolerated as long-term prophylaxis in children, adolescents, and adults with HAE."

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

Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks. (PubMed, J Allergy Clin Immunol Pract) - P3 | "In patients with frequent HAE attacks, long-term replacement therapy with C1-INH(SC) is safe and exhibits a substantial and sustained prophylactic effect, with the vast majority of patients becoming free from debilitating disease symptoms."

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

"BTW $CSL.AX stock has been on a tear (2018-19), thanks to Haegarda and demand for blood plasma & IVIG $QURE" (@JacobPlieth)

"#RAReports @US_FDA approved #SubcutaneousProducts are: Admelog(@sanofi ), Benlysta(Human Genome Sciences & @GSK ), DUPIXENT & Kevzara (@Regeneron & Sanofi), FASENRA(@AstraZeneca ), Fiasp(@novonordisk ), HAEGARDA(@CSLBehring ), HEMLIBRA(@Roche ) For report https://t.co/TE01HJixTG" (@RootsAnalysis)

Haegarda (C1 esterase inhibitor subcutaneous [human]) now available in the United States for patients with hereditary angioedema (PRNewswire) - "CSL Behring announced today that HAEGARDA...is now available in the United States. The U.S. Food and Drug Administration...approved HAEGARDA on June 22, 2017 for routine prophylaxis to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients."

Launch US • Antibody-mediated Rejection • Complement-mediated Rare Disorders