Takhzyro (lanadelumab-flyo) / Takeda - 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

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia (clinicaltrials.gov) - P=N/A | N=50 | Not yet recruiting | Sponsor: Takeda

HEOR • New trial • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Long-term Prophylaxis With Lanadelumab in Patients With Angioedema Due to C1 Inhibitor Deficiency: A Real-life Study in Spain. (PubMed, J Investig Allergol Clin Immunol) - No abstract available

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

Management of hereditary angioedema with normal C1Inh: a series of 163 French patients. (PubMed, Orphanet J Rare Dis) - "HAE patients with a normal C1inh have specific clinical features, including a later age at first attack than HAE patients with a normal C1inh, high sensitivity to estrogens of HAE-FXII and the location of the HAE-PLG on the tongue. The treatments used for HAE patients with C1Inh deficiency appear to be effective and safe. Low-dose progestin-only pills are good contraceptive options."

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

Implementation of genetic diagnosis and personalized management of hereditary angioedema in a Chinese regional center: a community case study of three families. (PubMed, Front Allergy) - "The program included standardized clinical assessment and real-time biochemical screening (C4, C1 inhibitor concentration/function), targeted SERPING1 sequencing and variant classification American College of Medical Genetics and Genomics (ACMG), family cascade screening and genetic counseling, stratified personalized treatment (on-demand icatibant and lanadelumab prophylaxis), electronic follow-up Angioedema Control Test (AECT)/Angioedema Quality of Life Questionnaire (AE-QoL), and quality management [Standard Operating Procedures (SOPs) and provincial External Quality Assessment (EQA) planning)]. Key facilitating factors included cascade screening and genetic counseling, standardized testing pathways and variant classification, flexible prophylactic strategies adapted to economic conditions, and electronic quality and outcome monitoring. This program has expanded the domestic SERPING1 variant spectrum and provides preliminary insights and references for the future..."

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

Hereditary Angioedema Prophylaxis Therapy: Berotralstat and Lanadelumab Safety Profile. (PubMed, Medicina (Kaunas)) - " The results confirmed the literature's data on the gastrointestinal adverse effects of berotralstat, as well as site reactions and infections associated with lanadelumab. Notably, musculoskeletal and connective tissue disorders, immune system disorders, vascular disorders, and metabolic issues occurred more frequently in patients using lanadelumab."

Journal • Retrospective data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Infectious Disease • Musculoskeletal Diseases • Rheumatology

A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries (clinicaltrials.gov) - P=N/A | N=40 | Not yet recruiting | Sponsor: Takeda

New trial • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Investigation of the Impact of Hereditary Angioedema (HAE) on Health-Related Quality of Life: A Systematic Literature Review (ISPOR-EU 2025) - "From 100 HRQoL references accepted, 31 were full-text journal articles reporting 27 unique studies that assessed treatment with berotralstat (n=4), C1-INH (n=3), donidalorsen (n=3), garadacimab (n=2), avoralstat, danazol, and lanadelumab (n=1 each); ten included a placebo comparator and twelve were non-interventional studies. Only one study was identified that captured HRQoL during and between patients' HAE attacks. There remains difficulty in accurately quantifying the impact of each attack on HRQoL, likely due to the transient and unpredictable nature of attacks. This highlights the need for further research to improve understanding of how to measure patient HRQoL during attacks."

Clinical • HEOR • Review • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hereditary Angioedema

Swelling from the Inside Out: Atypical AKI Triggered by Gastrointestinal Hereditary Angioedema Flare (KIDNEY WEEK 2025) - "He had missed his scheduled Takhzyro prophylaxis for over five weeks...Immunosuppressive agents (Cellcept, Acthar) were held...Bradykinin-mediated GI angioedema should be considered in the differential of unexplained AKI in patients with known or suspected HAE. Prompt treatment with Firazyr can reverse systemic symptoms and facilitate renal recovery, underscoring the importance of multidisciplinary collaboration in complex cases."

Acute Kidney Injury • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Endocrine Disorders • Focal Segmental Glomerulosclerosis • Gastrointestinal Disorder • Glomerulonephritis • Hereditary Angioedema • Immunology • Inflammation • Nephrology • Rare Diseases • Renal Disease

Requirements for Payer Recognition of Disease-Modifying Effect in New Drug Launches (ISPOR-EU 2025) - "The study examined recurring features cited by payers as value drivers, including clinical innovation, trial design, safety, and cost-effectiveness. All products achieving premium pricing addressed high unmet need, either by treating severe orphan diseases (e.g., Fintepla, Cablivi) or late-line settings (e.g., Omvoh)...Safety advantages and cost offsets (e.g., with Fintepla, Takhzyro, Tavneos) played a supportive role in reinforcing the perception of value. Achieving a disease-modifying profile and premium pricing is most viable when targeting high unmet need with compelling efficacy and clinical innovation... Achieving a disease-modifying profile and premium pricing is most viable when targeting high unmet need with compelling efficacy and clinical innovation. Novel mechanisms and clear differentiation enhance payer acceptance. While safety and economic benefits can reinforce value, they are insufficient without demonstrable clinical impact."

Cognitive Disorders

Current Use of Hereditary Angioedema (HAE) Treatments in Sweden: An Analysis of the National Prescribed Drug Register (ISPOR-EU 2025) - "Icatibant was the most dispensed concomitant treatment to LTP, followed by pdC1-INH. The observed prevalence aligns with prior estimates, though the incidence, when measured as number of new patients treated for HAE, is higher than what has been observed elsewhere. Despite restrictive reimbursement criteria, lanadelumab is the most prescribed LTP. The dispensing of on-demand/short-term treatment at least three times annually, indicates that a large share of patients potentially are not attack-free despite treatment with LTP."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Implementing a Value-Based Contract for Hereditary Angioedema Prophylaxis in Colombia: A Real-World Case Study... (ISPOR-EU 2025) - "Lanadelumab trials showed 44.4% of patients being attack-free after 6 months of treatment...VBAs provide sustainable financing approaches, emphasizing transparency, resource prioritization, and impactful interventions. Innovation must balance therapeutic efficacy and economic sustainability, prioritizing patients' access to treatments that improve quality of life.Stakeholder Perspective: This case reflects a joint payer-industry perspective demonstrating how structured real-world data, co-managed by HMOs and a pharmaceutical partner, can guide treatment strategies improving access in complex, high-cost conditions."

Case study • Clinical • Real-world • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Rare Diseases

Acquired Angioedema with C1 Esterase Inhibitor Deficiency Alerts to Possible Worsening Malignancy (ACAAI 2025) - "Prior to these episodes of angioedema, the patient’s lymphoma was presumed to be in remission on zanubrutinib...The patient acute angioedema responded to treatment with icatibant...Addition of Rituximab seemed to be helpful in managing underlying lymphoma. Future directions will involve possible weaning of the frequency of lanadelumab and/or Rituximab."

B Cell Lymphoma • Cardiovascular • Dermatology • Hematological Malignancies • Hereditary Angioedema • Immunology • Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Rare Diseases • Splenic Marginal Zone Lymphoma • Urticaria

Berotralstat Is Effective In Early Onset HAE With Normal C1 Esterase Inhibitor (ACAAI 2025) - "Previously failed treatments included C1 esterase inhibitor, lanadelumab-flyo, progestin, and omalizumab, which were either ineffective or caused significant side effects. She required icatibant injections for acute attacks due to the severity of symptoms...In one case series that demonstrated the effectiveness of berotralstat in this condition, most patients had adult-onset presentations (average age of onset for these patients was 32 years), whereas only 2 out of the 15 patients had childhood-onset presentations (ages unreported). Our case demonstrates the effectiveness of berotralstat in early onset HAE-nC1-INH."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Hereditary Angioedema with Normal C1 Esterase Inhibitor in a Male with PLG and SERPING1 Mutations (ACAAI 2025) - "Prednisone and montelukast offered only fleeting relief as attacks intensified. Icatibant swiftly halted flares, with symptoms clearing in days. Five months later, we began biweekly lanadelumab-flyo (300 mg q2wk)...Discussion This rare male case of HAE-nC1-INH with PLG and SERPING1 mutations underscores the urgency and necessity of repeated complement testing and targeted therapies to provide life-altering symptomatic management. Clinicians should maintain a high degree of suspicion and consider confirmation with genetic sequencing in cases of refractory angioedema in male patients."

Cardiovascular • Complement-mediated Rare Disorders • Fatigue • Hereditary Angioedema • Pulmonary Disease • ANGPT1

Of Swells and Cells: Two Birds One Inhibitor (ACAAI 2025) - "Due to severity and frequency of attacks, she was started on lanadelumab, responding well with only 4 mild breakthrough attacks requiring icatibant each year. Discussion Early reports of HAE-nC1 were described exclusively in female patients, supporting an association with estrogen. In the setting of our patient’s concomitant use of an aromatase inhibitor letrozole for estrogen receptor positive breast cancer, a clinical remission of HAE-nC1 seems plausible."

Breast Cancer • Estrogen Receptor Positive Breast Cancer • Hormone Receptor Breast Cancer • Solid Tumor • ER

A Case: Hepatic Abscesses and Cholecystitis Secondary to Hereditary Angioedema (ACAAI 2025) - "Case Description A 75-year-old male with HAE type I presented with throat swelling after missing two doses of lanadelumab...Treatment consisted of icatibant, cefepime, metronidazole and bowel decompression...He improved and was discharged on omadacycline with outpatient follow-up...Upon continued symptoms, repeat imaging showed hepatic abscess development likely from bacterial translocation from gut edema and resulting cholecystitis complications 2 . This case showcases the multitude of complications that may arise in HAE patients."

Clinical • Cardiovascular • Complement-mediated Rare Disorders • Gastroenterology • Gastrointestinal Disorder • Hepatology • Hereditary Angioedema • Infectious Disease

Switching To Donidalorsen For Hereditary Angioedema: 1-Year Results From The Phase 3 OASISplus Study (ACAAI 2025) - P3 | "Methods Patients with HAE on stable doses (≥12 weeks) of lanadelumab, subcutaneous (SC) or intravenous C1 inhibitor (C1INH), or berotralstat switched to 80-mg donidalorsen SC every 4 weeks without washout using a predefined algorithm. Conclusions Donidalorsen was well tolerated by patients switching from prior LTPs. Improvements in HAE attack rate, QoL, and disease control were sustained through 1 year."

P3 data • Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Hereditary Angioedema • Infectious Disease • Respiratory Diseases

Prolonged QT Syndrome Complicated By Cardiac Arrest: Did Berotralstat Play A Role? (ACAAI 2025) - "Berotralstat was discontinued in favor of lanadelumab-flyo. In this case, there were numerous complications of hypomagnesemia, hypokalemia, and use of haloperidol (metabolized by CYP2D6 and CYP3A4) that resulted in prolongation of QT interval, Torsade’s de pointes, and ultimately cardiac arrest. Prior to starting Berotralstat as HAE prophylaxis, patients should be evaluated for prolonged QT, advised to avoid QT prolonging medications, and advised to avoid medications metabolized by CYP2D6 or CYD3A4."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Ventricular Tachycardia • Women's Health • CYP3A4

The Many Faces Of Angioedema (ACAAI 2025) - "Icatibant 30mg dose was given with improvement in swelling. Hemodialysis activates the kallikrein-kinin system, increasing bradykinin. Our patient continued to have recurrent episodes related to hemodialysis, requiring prophylactic Lanadelumab."

Cardiovascular • Chronic Kidney Disease • Cystic Fibrosis • Dermatology • Genetic Disorders • Immunology • Pulmonary Disease • Renal Disease • Respiratory Diseases • Urticaria

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

Leveraging lanadelumab: A phase 4 evaluation of hereditary angioedema by attack rate subgroups. (PubMed, Ann Allergy Asthma Immunol) - No abstract available

Journal • P4 data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Misdiagnosed Hereditary Angioedema with Recurrent Abdominal Pain: A Novel SERPING1 Frameshift Variant. (PubMed, J Asthma Allergy) - "Since initiating lanadelumab (300mg biweekly) on April 26, 2023, the patient has completed 11 injections and remains in good condition...Complement C4 remains a critical screening biomarker for patients with HAE. In addition, we reported a novel frameshift variant in the coding region of the SERPING1 gene, and the specificity of the position offers a unique point of interest in the discussion of the disease's causative locus."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gastroenterology • Gastrointestinal Disorder • Hereditary Angioedema • Pain • Rare Diseases

Real-World Evidence on the Management of Hereditary Angioedema with Normal C1 Inhibitor. (PubMed, J Allergy Clin Immunol Pract) - "COC withdrawal is the most effective first-line intervention in HAE-FXII, with greater efficacy when combined with progestins. Tranexamic acid and lanadelumab may serve as complementary options in selected cases. Late relapse highlights the need for long-term follow-up, with intensified monitoring during periods of hormonal fluctuation."

HEOR • Journal • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • ANGPT1