Takhzyro (lanadelumab-flyo) / Takeda - LARVOL DELTA

Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema. (PubMed, World Allergy Organ J) - P=N/A | "Patients receiving lanadelumab were excluded, as it was not yet widely available during the eligibility period...Attenuated androgens (AA; stanozolol and danazol) in LTP were used by 24.7% of patients, while tranexamic acid (TA) and C1-esterase inhibitor (C1-INH) replacement products were used for LTP by 9.8% and 6.5%, respectively...It imposes a substantial burden on patients' HRQoL, more particularly, but not exclusively, for those with frequent attacks and for women. The results suggest a need for improved HAE management.ClinicalTrial.gov study identifier NCT04957641."

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

Management of Airway Compromise on Anesthetic Induction in a Patient with Hereditary Angioedema (ASA 2025) - "Despite preoperative stability and biweekly Lanadelumab therapy, bronchospasm developed during attempted endotracheal intubation requiring emergent intubation via existing open tracheal stoma. The bronchospasm was refractory to epinephrine, albuterol, and hydrocortisone...This case underscores the critical need for early recognition, preparation, and treatment of HAE-related airway compromise during anesthesia. Icatibant should be considered first-line therapy in such scenarios, especially when conventional treatments for bronchospasm fail."

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

One-year real-life outcomes of lanadelumab therapy in Romanian patients with hereditary angioedema due to C1-inhibitor deficiency. (PubMed, Front Allergy) - "LTP with lanadelumab provided effective disease control and significantly improved quality of life in patients with HAE-C1INH over the course of one year. Regular evaluations at relatively short intervals by the availability and ease of administration of validated questionnaires serve as a useful tool for clinicians in the comprehensive assessment of HAE patients and offer a valuable means of monitoring treatment effectiveness."

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

Insights from the first 820 patients from the Brazilian Multicenter Registry of Hereditary Angioedema: the key role of genetic testing and targeted therapies. (PubMed, J Allergy Clin Immunol Pract) - "Brazilian patients with HAE share common aspects with global patients, including predominance in women, and HAE-C1INH as the most common subtype. Available genetic testing allowed for identification of a notable proportion of HAE-FXII (19.4% of the patients). Despite recent advances, access to first-line therapies for long-term prophylaxis of HAE attacks remains limited."

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

Three-year real-world outcomes of lanadelumab prophylaxis in hereditary angioedema: Complete disease suppression and psychosocial benefits in two East Asian patients. (PubMed, Intractable Rare Dis Res) - "Findings confirm its sustained efficacy, safety, and psychosocial benefits, including enhanced quality of life and emotional recovery. These findings suggest that lanadelumab may play an important role in the long-term management of HAE in Asian clinical settings."

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

Expanding the Genetic and Clinical Spectrum of Hereditary Angioedema with Normal C1 Inhibitor: Novel Variants and Treatment Insights. (PubMed, J Clin Immunol) - "Lanadelumab effectively reduced attack frequency in most patients; however, the variability in treatment response, particularly in MYOF and HS3ST6 carriers, highlights the need for individualized therapeutic approaches. These findings provide new insights into the genetic and clinical complexity of HAE-nC1-INH and emphasize the importance of genetic testing in refining diagnosis and optimizing treatment strategies, contributing to a more precise understanding of hereditary angioedema."

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

Hinder: Hemodialysis.-Induced Hypotension Therapy for End Stage Kidney Disease (clinicaltrials.gov) - P2 | N=28 | Active, not recruiting | Sponsor: Vanderbilt University Medical Center | Recruiting ➔ Active, not recruiting | Trial completion date: Feb 2025 ➔ Aug 2025

Enrollment closed • Trial completion date • Chronic Kidney Disease • Hypotension • Nephrology • Renal Disease

A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China (clinicaltrials.gov) - P=N/A | N=115 | Completed | Sponsor: Takeda | Recruiting ➔ Completed

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

Matching-adjusted indirect comparison between garadacimab and lanadelumab for the long-term prophylactic treatment of patients with hereditary angioedema. (PubMed, J Comp Eff Res) - P2, P3 | " These MAICs showed improved efficacy and QoL with garadacimab compared with lanadelumab across multiple endpoints. These findings demonstrate that garadacimab may provide improved therapeutic benefit compared with lanadelumab in the long-term prophylactic treatment of patients with HAE."

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

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis. (PubMed, J Allergy Clin Immunol Pract) - P3 | "Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52."

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

A single-centre retrospective study on the clinical characteristics of patients with hereditary angioedema and the therapeutic effect of lanadelumab. (PubMed, Orphanet J Rare Dis) - P4 | "(1) The median age at onset, diagnostic delay, and precipitating factors in this cohort were consistent with previously reported data from domestic studies. However, the proportion of Type 2 patients was greater than that in prior domestic reports, and a trend towards earlier diagnosis in younger patients was observed; notably, this cohort identified a high proportion (27.3%) of patients with positive autoantibodies or confirmed autoimmune diseases for the first time in China. (2) After treatment with lanadelumab, patients experienced significant improvements in symptoms, quality of life, and anxiety/depression levels. Symptom control was achieved by D30 prior to the drug steady state-period and was maintained throughout the entire treatment period. No serious adverse reactions were observed during the treatment, indicating a high safety profile for the medication."

Journal • Retrospective data • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Depression • Hereditary Angioedema • Immunology • Mood Disorders • Psychiatry

Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: real-world evidence. (PubMed, Ann Allergy Asthma Immunol) - P, P=N/A | "In these real-world datasets, on-treatment lanadelumab attack rates were low regardless of baseline disease activity. Patients from all 4 subgroups experienced improvements in health-related quality of life and disease control. Overall, these findings support long-term prophylaxis with lanadelumab across disease activity levels."

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

Clinical presentation and management of 239 children and adolescents with hereditary angioedema. (PubMed, J Allergy Clin Immunol Pract) - No abstract available

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

Acquired Angioedema due to C1-Esterase Inhibitor Deficiency: A Diagnostic and Therapeutic Challenge. (PubMed, J Investig Allergol Clin Immunol) - No abstract available

Journal • Cardiovascular • Hereditary Angioedema

Long-term Real-world Outcomes in Patients with Hereditary Angioedema Receiving Lanadelumab for 3 or More Years. (PubMed, Int Arch Allergy Immunol) - "In this real-world HAE study, patients treated with lanadelumab for ≥3 years experienced improvements in attack frequency, disease severity, and QoL."

Journal • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hereditary Angioedema • Pain

Treatment Patterns and Characteristics of Patients with Hereditary Angioedema Treated with Lanadelumab: A US Retrospective Chart Review. (PubMed, Drugs Real World Outcomes) - "Lanadelumab dosing intervals can be individualized to maintain effective disease control. A dosing interval extension may be considered in well-controlled disease."

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

Hereditary angioedema in a paediatric patient presenting with abdominal pain and duodenal dilation. (PubMed, BMJ Case Rep) - "Acute treatment with Berinert was successful, and the patient was later placed on long-term prophylaxis with lanadelumab, which is a monoclonal antibody that inhibits Kallikrein. This case illustrates the importance of considering HAE in children with unexplained abdominal symptoms, particularly in the context of a family history of the condition. Early identification and treatment are essential to managing this life-threatening disorder."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Genetic Disorders • Hereditary Angioedema • Inflammation • Pain • Pediatrics

Comment on: "A national survey of four decades of hereditary angioedema prophylaxis" - The emerging role of Garadacimab. (PubMed, Clin Immunol) - No abstract available

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

A national survey of four decades of hereditary angioedema prophylaxis: Efficacy and safety of old and new drugs. (PubMed, Clin Immunol) - "Modern prophylaxis usage is increasing among Hungarian HAE-C1INH patients, following the global trends. Both modern and traditional LTP options proved safe and effective in our patient population when used with appropriate monitoring."

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

Sustained Effectiveness, Tolerability, and Safety of Long-Term Prophylaxis with Lanadelumab in Hereditary Angioedema: The Prospective, Phase 4, Noninterventional EMPOWER Real-World Study. (PubMed, Adv Ther) - P=N/A | "Real-world data from EMPOWER showed marked HAE attack rate reduction up to 36 months after initiating lanadelumab in patients newly treated with lanadelumab and maintenance of low attack rates in patients established on lanadelumab. No new safety signals were identified."

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

Lanadelumab for prevention of attacks of non-histaminergic normal C1 inhibitor angioedema: results from the randomized, double-blind CASPIAN Study and CASPIAN open-label extension. (PubMed, Front Immunol) - P3 | "Overall results suggest potential clinical benefit in symptom control. https://www.clinicaltrials.gov/, identifiers NCT04206605, NCT04444895."

Clinical • Journal • Cardiovascular

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

Assessment of Conditional Listing for Highly Uncertain and High-Priced Drugs in Taiwan (ISPOR 2025) - " A total of 12 drugs were considered for conditional listing between 2022 and 2024, which included Pemazyre, Tepmetko, Qarziba, Kymriah, Vitrakvi, Blincyto, Vydamax, Takhzyro, Polivy, Velexbru, Spevigo, and Koselugo. The conditional listing opens up new opportunities for high-priced drugs. However, the 2-year real-world evidence for Pemazyre remains insufficient for the government to make a final reimbursement decision. Consequently, the results of the HTA will pose challenges in the future in Taiwan."

Hereditary angioedema plasma proteomics following specific plasma kallikrein inhibition with lanadelumab. (PubMed, Front Immunol) - "Canonical pathway and local network analyses of HAE-C1INH plasma proteomics suggest dysregulation in KKS, coagulation, cell adhesion, and connective tissue degradation that approach that of healthy controls following treatment with lanadelumab. Proteomic analyses of plasma from patients with HAE-C1INH before and after treatment with lanadelumab compared with healthy controls confirmed known HAE-C1INH biomarkers and identified additional potential biomarkers of plasma kallikrein dysregulation for further investigation."

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

Diffusion dimensionality modeling of subcutaneous/intramuscular absorption of antibodies and long-acting injectables. (PubMed, J Pharmacokinet Pharmacodyn) - "The diffusion dimensionality models were challenged with previously published subcutaneous absorption data for 13 antibody drugs and insulin lispro, and the long-acting injectable antipsychotic drugs: subcutaneous Perseris™, intramuscular Invega Sustenna®, Risperdal Consta®, and olanzapine...Three antibodies (CNTO5825, ACE910 and ustekinumab) were best described by the 2-dimensional diffusion equation. The 2- and 3-dimensional diffusion equations were equivalent for ABT981, guselkumab, adalimumab, nemolizumab, omalizumab, and secukinumab. Golimumab, DX2930, AMG139, and mepolizumab were best described by the 3-dimensional diffusion equation. All the long-acting antipsychotic dosage forms except Risperdal Consta were modeled satisfactorily. Diffusion dimensionality models are a parsimonious and effective approach for modeling drug absorption profiles of subcutaneously and intramuscularly administered small molecule and protein drugs and their dosage forms."

Journal • CNS Disorders