Takhzyro (lanadelumab-flyo) / Takeda - LARVOL DELTA

Real-world quality of life in patients with hereditary angioedema receiving lanadelumab or other long-term prophylaxis. (PubMed, Allergy Asthma Proc) - "Both physicians and patients reported higher QoL in those receiving lanadelumab. Continued real-world research in patients with HAE to further assess the effect of lanadelumab on QoL will help guide patient care."

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

Switching Long-Term Prophylaxis to Donidalorsen for Hereditary Angioedema: 1-Year OASISplus Results. (PubMed, Allergy) - P3 | "In patients who switched from another LTP, donidalorsen was well tolerated and improved long-term HAE attack rates, quality of life, and disease control."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Hereditary Angioedema • Inflammation • Pruritus

Lanadelumab's Enduring Effectiveness and Safety in the Management of Hereditary Angioedema for Patients from Puerto Rico: Data from EMPOWER. (PubMed, Int Arch Allergy Immunol) - P=N/A | "Findings from this analysis support the clinical benefits and safety of lanadelumab treatment for HAE prophylaxis in patients from Puerto Rico."

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

Real-world Adherence and Persistence with Long-term Prophylaxis among Patients with Hereditary Angioedema (AAAAI 2026) - "Rationale The treatment landscape for hereditary angioedema (HAE) is evolving rapidly, with several long-term prophylaxes (LTP) approved in the last decade, including Haegarda (subcutaneous injection, every 3-4 days), Takhzyro (subcutaneous injection, every 2-4 weeks) and Orladeyo (once-daily oral capsule). Conclusions These findings suggest suboptimal adherence and persistence to recently approved LTP. Alternative options with a less frequent dosing schedule may help increase adherence and persistence, and could lead to improved outcomes for patients with HAE."

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

Safety and Efficacy of Donidalorsen in Adolescent Patients With Hereditary Angioedema: 1-Year Results From OASISplus (AAAAI 2026) - P3 | "Methods Patients on stable doses (≥12 weeks) of berotralstat, intravenous (IV) or subcutaneous (SC) C1INH, or lanadelumab switched to donidalorsen 80 mg SC once every 4 weeks without washout per a prespecified algorithm. Findings from 7 additional adolescent patients enrolled in a separate open-label extension cohort of OASISplus were consistent with those reported here. Conclusions In adolescents, donidalorsen had an acceptable safety and tolerability profile and improved HAE attack rates and quality of life, consistent with overall results from OASISplus."

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

Additional Benefit of Switching to Donidalorsen for Patients with Hereditary Angioedema Having Breakthrough Attacks: Findings from the OASISplus Study (AAAAI 2026) - P3 | "Methods Post-hoc analysis of data from a 52-week study of patients who switched directly from a prior LTP therapy (lanadelumab, C1-INH, or berotralstat) to donidalorsen (OASISplus, NCT05392114, n=64). Conclusions Patients with HAE on LTP therapy who switched to donidalorsen experienced improvements in subjective disease control and QoL regardless of baseline attack rate. Furthermore, patients with a higher baseline breakthrough attack rate reported significantly greater improvements in subjective disease control and QoL."

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

Improved Access, Reduced Burden: Patient-Reported Outcomes in Canadian HAEnC1-INH Patients from 2017 to 2024 (AAAAI 2026) - "C1-INH use for acute treatment declined from 50% (2017) to 31% (2024) while icatibant increased from 12% to 43% respectively...Newer medications (e.g., lanadelumab) were first used (29%) in 2024...The proportion reporting very high impact on well-being (2017: 48%; 2024: 17%) and ability to work full-time (2017: 52%; 2024: 30%) declined but fear of laryngeal attacks remained high (2020: 68%; 2024: 78%). Conclusions Treatment, attack rates, impact on well-being and ability to work full time have improved since 2017 for HAEnC1-INH patients but fear of laryngeal attacks remains high."

Clinical • Patient reported outcomes • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Longitudinal Survey of Adult Patients with Hereditary Angioedema Receiving Long-Term Prophylaxis with Lanadelumab: 12 Month Findings (AAAAI 2026) - "Global treatment satisfaction scores were high (M12 TSQM-9: 88.0). Conclusions Patients receiving lanadelumab for ≥3 years who completed a 12-month survey (thus receiving lanadelumab for ≥4 years) experienced low attack rates, numerical improvements in QoL and HAE severity, and consistently high treatment satisfaction."

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

Hereditary angioedema due to C1 inhibitor deficiency: insights from the italian ITACA Registry (AAAAI 2026) - "395/1003 subjects (39,38%) were on long term prophylaxis, of whom 186/395 (47%) with lanadelumab...70 attacks (13,18%) were reported as severe. Conclusions HAE still represents a significant burden on the quality of life in this large cohort of italian patients."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Lanadelumab Posology For Hereditary Angioedema (HAE) Long-term Prophylaxis (LTP) In Pediatric Patients Aged 2 to <12 Years Generates Exposure Comparable To Adult Patients: A Population Pharmacokinetics and Pharmacodynamics (PopPKPD) Analysis (AAAAI 2026) - "cHMWK suppression simulated in children aged 2–89% of exposure parameters within reference range). Conclusions Current US lanadelumab posology is expected to generate pharmacokinetics and pharmacodynamics comparable between pediatric and adult patients."

Clinical • PK/PD data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Substantial Burden Of Disease In Pediatric Patients With Hereditary Angioedema (HAE): Results From A Multinational Caregiver Survey (AAAAI 2026) - "Rationale Lanadelumab is approved as an effective HAE long-term prophylaxis (LTP), reducing attack frequency and severity in patients aged ≥2 years...In the 4 weeks preceding the survey, HAE affected daily, physical, and social activities in ≥74% of children and mean±SD 5.0±7.5 school days were missed. Conclusions Caregivers highlighted most children with HAE experience a substantial humanistic burden associated with the disease, underscoring the need for effective LTP as most were untreated with LTP."

Clinical • HEOR • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Mood Disorders • Pediatrics

Real-World Lanadelumab Effectiveness in Older Adults with HAE: Pooled Analysis From INTEGRATED and ENABLE (AAAAI 2026) - P, P=N/A | "Conclusions In this analysis of real-world data, older adults with HAE experienced regular attacks requiring long-term prophylaxis prior to lanadelumab initiation. Lanadelumab produced marked reductions in monthly HAE attack rates, supporting its use in this population."

Real-world • Real-world evidence • Retrospective data • 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

A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea (clinicaltrials.gov) - P=N/A | N=35 | Not yet recruiting | Sponsor: Takeda

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

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

Enrollment open • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Management of pregnancy, delivery and breast-feeding in hereditary angioedema: an analysis of 15 pregnancies with conventional treatment approaches and a case of Lanadelumab use. (PubMed, Orphanet J Rare Dis) - No abstract available

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

Clinical features of hereditary angioedema involving the gastrointestinal tract: A retrospective analysis. (PubMed, World Allergy Organ J) - "Lanadelumab demonstrated superior efficacy over danazol in reducing both the frequency and severity of edema attacks, while icatibant significantly shortened the duration of edema episodes. This study updates the clinical and imaging features of gastrointestinal edema in Chinese patients with HAE, identifies biomarkers of gastrointestinal edema attacks, and highlights the efficacy of lanadelumab and icatibant, aiding timely diagnosis and treatment."

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

Lanadelumab for Monoclonal Gammopathy of Angioedema-Associated Significance in Acquired C1-Esterase Inhibitor Deficiency: A Case Series. (PubMed, J Investig Allergol Clin Immunol) - No abstract available

Journal • Cardiovascular • Hereditary Angioedema • Monoclonal Gammopathy

Novel SERPING1 Genetic Variant in Two Family Members with Hereditary Angioedema. (PubMed, Acta Med Port) - "We report two cases of hereditary angioedema (HAE) in a Portuguese family: a 27-year-old male, under lanadelumab, and his 57-year-old father, kept on on-demand treatment...We report the identification of a novel pathogenic SERPING1 variant in a family with HAE type 1, assisted by the Hereditary Angioedema Database Annotation variant prioritization tool. Our results may contribute to the identification of additional families with the same variant and can further enhance the knowledge about this condition."

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

A Survey of Lanadelumab in Participants With Hereditary Angioedema (clinicaltrials.gov) - P=N/A | N=155 | Completed | Sponsor: Takeda | Recruiting ➔ Completed | N=100 ➔ 155

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

Pain management in patients with hereditary angioedema: A case report. (PubMed, Medicine (Baltimore)) - "This case underscores the importance of early recognition of HAE presenting solely with abdominal pain and demonstrates that multidisciplinary, targeted pain management can lead to rapid and sustained symptom relief. Awareness of such atypical presentations is critical for optimizing outcomes in HAE patients."

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

Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study. (PubMed, Allergy Asthma Clin Immunol) - P=N/A | "Lanadelumab lowered the HAE attack rate among newly treated Canadian patients and maintained a low attack rate among those previously established on lanadelumab, demonstrating robust effectiveness across both patient groups. Effectiveness, tolerability, and safety of lanadelumab were consistent with the overall population from the EMPOWER Study, supporting the use of lanadelumab as a first-line long-term prophylactic treatment for patients with HAE in Canada."

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

Type II hereditary angioedema with an apparently de novo SERPING1 mutation in China: A case report and family screening. (PubMed, Medicine (Baltimore)) - "This case emphasizes the need to consider HAE in unexplained recurrent angioedema and demonstrates the clinical utility of genetic testing and family screening. Lanadelumab was effective and well-tolerated for prophylaxis, consistent with current guideline recommendations."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Gastrointestinal Disorder • 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 | Trial completion date: Aug 2025 ➔ Jan 2026

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

Efficacy and acceptability of lanadelumab for long-term prophylaxis in hereditary angioedema: A Chinese multicenter real-world analysis. (PubMed, World Allergy Organ J) - "Successful symptom-guided dosing interval extension was achievable in most patients. Despite clinical benefits, its high cost imposes financial burdens on our patients, but this should be weighed against the cost savings achieved through improved disease control."

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