Takhzyro (lanadelumab-flyo) / Takeda - LARVOL DELTA

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

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

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

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

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

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

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

Budget Impact Analysis of Lanadelumab Versus C1 Inhibitor for Hereditary Angioedema Long Term Prophylaxis: A Brazilian Private Sector Perspective (ISPOR 2025) - "Long-term prophylaxis with Lanadelumab offers a new therapeutic option, more effective compared to currently available C1 esterase inhibitors (C1-INH). The budget impact analysis suggests that the incorporation of Lanadelumab for the prophylaxis of HAE can generate substantial savings for the Brazilian supplementary health system"

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

Cost Effectiveness Analysis of Lanadelumab vs C1 Inhibitor for Long-Term Prophylaxis of Hereditary Angioedema: A Brazilian Private Sector Perspective (ISPOR 2025) - "Lanadelumab proved to be dominant, providing a more effective and cost-saving option for the prophylaxis of HAE attacks compared to C1-INH IV"

Cost effectiveness • HEOR • Cardiovascular • CNS Disorders • Complement-mediated Rare Disorders • Depression • Genetic Disorders • Hereditary Angioedema • Mood Disorders • Pain • Psychiatry

Case Report: Identification of a novel mutation, c.1067T > A, in the SERPING1 gene in a Chinese male with type 1 hereditary angioedema. (PubMed, Front Allergy) - "Genomic DNA extracted from peripheral blood samples underwent PCR amplification and Sanger sequencing, which identified a de novo heterozygous mutation in the SERPING1 gene at chr11:57379227, confirming a novel missense mutation NM_000062.c.1067T > A (p.V356E). Ultimately, the patient was diagnosed with HAE-C1INH-Type1 and successfully protected from recurrent attacks through subcutaneous administration of lanadelumab."

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

Safe Off-Label Use of Icatibant as Treatment for Acute Hereditary Angioedema Attacks in a Pediatric Patient (CIS 2025) - "Case Description: Despite previous success with Berinert for abortion of peripheral attacks, this patient's family was frustrated with long wait times and care in the ED...Eventually, the patient was approved through her insurance to use icatibant 15 mg SQ for attacks, and lanadelumab was also initiated for maintenance therapy given increasing attack frequency through manufacturer assistance and eventual insurance approval through assistance of a multidisciplinary team... This case illustrates the potential for safe off-label use of icatibant down to 2 years of age for acute attacks of HAE. Penn State Health has been prescribing and administering icatibant down to 2 years of age despite being off-label, and we hope this work encourages FDA approval and other institutions to make icatibant available as a weight-adjusted dose as an option for abortive treatment until oral rescue medications are available to reduce drug burden in pediatrics."

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

Hereditary angioedema-induced laryngeal edema: A diagnostic and therapeutic race against time. (PubMed, Am J Emerg Med) - "The patient improved with icatibant and was placed on lanadelumab prophylaxis. Genetic analysis to confirm the presence of SERPING1 mutations was not conducted in this case, which remains a limitation for further genotype-phenotype correlation studies. This case highlights the need for early recognition and targeted therapy in HALE to prevent life-threatening complications."

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

Adherence and persistence among patients with hereditary angioedema receiving long-term prophylaxis in the United States. (PubMed, Allergy Asthma Proc) - "Adherence and persistence rates for all three LTP treatments were uniformly high. Berotralstat adherence andpersistence were comparable with those observed after lanadelumab or SC-pdC1-INH initiation in the main analysis, among patients with two or more claims for their index LTP, and among cohorts assigned based on the first claim for qualifying LTP."

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

A CASE OF HEREDITARY ANGIOEDEMA WITH NORMAL C1 INHIBITOR (HEREDITARY ANGIOEDEMA TYPE III) WITH A PATHOGENIC VARIANT IN THE PLG GENE (PubMed, Arerugi) - "She was treated with tranexamic acid as a long-term prophylactic. After switching from lanadelumab to twice-weekly subcutaneous injections of C1-inhibitor (C1-INH), she had no attacks requiring additional treatment, although she had several minor attacks. To establish appropriate treatment for HAE type III, accumulation of cases, determination of the causative gene in each case, and elucidation of the pathology are warranted."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Genetic Disorders • Hereditary Angioedema • Pain

Comment on "Long-term prevention of hereditary angioedema attacks with lanadelumab in adolescents". (PubMed, Ann Allergy Asthma Immunol) - No abstract available

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