Takhzyro (lanadelumab-flyo) / Takeda - LARVOL DELTA

Assessment of Conditional Listing for Highly Uncertain and High-Priced Drugs in Taiwan (ISPOR 2025) - "The review and assessment process were conducted independently by two researchers. 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."

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

A Single-center Retrospective Study on the Clinical Characteristics of Patients with Hereditary Angioedema and the Therapeutic Effect of Lanadelumab (ChiCTR) - P4 | N=20 | Recruiting | Sponsor: Renji Hospital affiliated to Shanghai Jiaotong University School of Medicine; Renji Hospital affiliated to Shanghai Jiaotong University School of Medi

New P4 trial • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Effective long-term prophylaxis with lanadelumab in adolescents with hereditary angioedema: EMPOWER/ENABLE. (PubMed, Pediatr Allergy Immunol) - P, P=N/A | "These data support lanadelumab's effectiveness/safety in adolescents with HAE, consistent with results from Phase 3 lanadelumab studies in mixed adult/adolescent populations."

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

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

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

A Survey of Lanadelumab in Participants with Hereditary Angioedema (clinicaltrials.gov) - P=N/A | N=100 | Recruiting | Sponsor: Takeda | Trial completion date: Aug 2026 ➔ Jan 2026 | Trial primary completion date: Aug 2026 ➔ Jan 2026

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

The European Medicines Agency (EMA) Has Approved an Additional Subcutaneous Administration Option for TAKHZYRO (lanadelumab) for Patients Aged 12 Years and Above with Recurrent Attacks of Hereditary Angioedema (HAE) (PRNewswire) - "Takeda...announced today that the EMA has approved an additional 2 mL pre-filled pen option for TAKHZYRO (lanadelumab) for subcutaneous administration in adolescents (aged 12 years and above) and adult patients with Hereditary Angioedema (HAE)."

EMA approval • Hereditary Angioedema

Comparison of real-world healthcare resource utilization and costs among patients with hereditary angioedema on lanadelumab or berotralstat long-term prophylaxis. (PubMed, J Comp Eff Res) - " Patients with HAE initiating lanadelumab versus berotralstat may require less on-demand and supportive HAE treatments and incur lower treatment-related and total healthcare costs. The ability to reduce lanadelumab dosing frequency after an attack-free period may be key in treatment selection, given the combination of cost savings and lower healthcare resource utilization."

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

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

Lanadelumab in Long-term Prophylaxis of Acquired Angioedema (clinicaltrials.gov) - P4 | N=5 | Recruiting | Sponsor: Bernstein Clinical Research Center

New P4 trial • Cardiovascular

Real-World Analysis of Treatment Patterns Among Patients with Hereditary Angioedema in the United States (AAAAI-WAO 2025) - "For those receiving index antifibrinolytics, attenuated androgens, C1INH IV, C1INH SC, berotralstat, progestins or lanadelumab, 19.2%, 13.4%, 12.7%, 5.7%, 4.3%, 4.3% and 3.7% switched therapy, respectively. In this study, most patients administered index LTP maintained their prescribed LTP; those who switched did so early with one third having multiple LTP switches. These data describe the ongoing optimization of HAE management through LTP, with potential for further optimization in future."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Enduring Effectiveness and Safety in Patients Newly Treated With Lanadelumab: An EMPOWER Subgroup Analysis (AAAAI-WAO 2025) - P=N/A | "Conclusions New lanadelumab patients from the real-world EMPOWER Study experienced marked attack rate reductions after initiating lanadelumab. Lanadelumab effectiveness and safety were aligned with previous studies, reinforcing lanadelumab as a first-line option for long-term prophylaxis."

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

Hereditary Angioedema Disease Control After Switching To Donidalorsen From Prior Long-Term Prophylaxis: Results From The OASISplus Open-Label Extension Study (AAAAI-WAO 2025) - P3 | "In patients reporting poorly-controlled disease at baseline, mean monthly HAE attack rates decreased by 73% (lanadelumab), 60% (C1INH), and 76% (berotralstat). Conclusions Switching from lanadelumab, C1INH, or berotralstat to donidalorsen improved patient-reported disease control and reduced HAE attack rates."

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

Sustained Effectiveness With Long-Term Lanadelumab Treatment: An EMPOWER Subgroup Analysis (AAAAI-WAO 2025) - P=N/A | "Most HAE attacks were mild or moderate in severity (77.7%) and were treated with HAE medications (81.3%), most frequently with icatibant. Conclusions Established lanadelumab patients from the real-world EMPOWER Study reported low HAE attack rates during the study, suggesting sustained lanadelumab effectiveness; lanadelumab safety was consistent with previous studies. These results add to the data supporting lanadelumab as a first-line long-term prophylaxis option."

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

Optimizing Lanadelumab Dosing Regimens for Enhanced Quality of Life in Hereditary Angioedema: A Systematic Review (AAAAI-WAO 2025) - "However, it is important to consider that some patients may prefer a less frequent dosing schedule. Therefore, personalizing the dosing schedule, while still keeping the disease under control, could continue to maintain the treatment's effectiveness while boosting patient satisfaction."

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

Real-World Effectiveness and Safety of Lanadelumab in Adults and Adolescents with Hereditary Angioedema in Argentina (AAAAI-WAO 2025) - "All reported TEAEs were mild to moderate in severity. Conclusions This real-world study demonstrates the effectiveness and safety of lanadelumab in adults and adolescents with HAE in Argentina and supports its use as first-line option for prophylaxis."

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

Patient-Reported Injection-Site Pain And Treatment Satisfaction After Switching From Long-Term Prophylaxis To Donidalorsen For The Treatment Of Hereditary Angioedema: Results From The OASISplus Study (AAAAI-WAO 2025) - P3 | "Global treatment satisfaction (TSQM-II) scores increased (i.e., improved satisfaction) from baseline to Week 16: berotralstat (50.9) to donidalorsen (92.5), C1INH (76.1) to donidalorsen (88.4), and lanadelumab (84.3) to donidalorsen (88.6); effectiveness and convenience domains also improved from baseline to Week 16. Conclusions Patient-reported ISP and treatment satisfaction improved after switching from prior LTPs to donidalorsen."

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

Canadian Physicians Experiences with Management of Hereditary Angioedema in Pregnancy (AAAAI-WAO 2025) - "For treatment of attacks in pregnancy, 42% use intravenous plasma derived C1 inhibitor (pdC1-INH), 12% use icatibant, and 9% use subcutaneous pdC1-INH. For short-term prophylaxis (STP), 42% use intravenous pdC1-INH, 14% use subcutaneous pdC1-INH, 7% use tranexamic acid, and 5% use fresh frozen plasma...For long-term prophylaxis (LTP), 37% use subcutaneous pdC1-INH, 35% use intravenous pdC1-INH, and 30% use lanadelumab...About one quarter of physicians reported not prescribing medications for treatment of HAE attacks and STP in pregnancy and not all physicians prescribed STP for deliveries. An upcoming qualitative study will aim to further explain these findings."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Understanding the Impact of Long-Term Prophylaxis Switches for Patients With Hereditary Angioedema (AAAAI-WAO 2025) - "Methods Patients with HAE aged >18 years in the US who initiated lanadelumab LTP before switching to berotralstat then back to lanadelumab were eligible and enrolled through centers that completed a linked chart-review, or directly through a patient research register. Almost half of patients reported complete satisfaction with lanadelumab. Given selection criteria and small sample, findings may not be generalizable to all patients with HAE."

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

Evolution Of Long-Term Prophylaxis In Patients With Hereditary Angioedema With C1 Inhibitor Deficiency, A 10-year Real-Life Experience In A Spanish Cohort. (AAAAI-WAO 2025) - "During 2018, mean (SD) annual attack rate was 7.4(10.9), 35(26.3%) patients were receiving LTP, danazol being the most common, median (IQR) HAE-QoL and AE-QoL were 101.5(82.5-122) and 30.9(11-47.8) respectively. • During 2013 and 2018, the most common LTP was an attenuated androgen but in 2023 was lanadelumab. • Quality of life, as measured by HAE-QoL and AE-QoL, improved between 2018 and 2023."

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

Lanadelumab for treatment of acquired angioedema triggered by ACE inhibitor (AAAAI-WAO 2025) - No abstract available

Cardiovascular

Longitudinal Study of Adult Patients with Hereditary Angioedema Receiving Long-Term Prophylaxis for 3 Years or Longer with Lanadelumab: Baseline Findings (AAAAI-WAO 2025) - "Compared with pre-lanadelumab, patients at index experienced reduced HAE severity (mean [SD] score 3.57/10.0 [1.93] vs 6.96/10.0 [1.36]; P <0.001), improved QoL (mean [SD] score 4.15/5.0 [0.76] vs 2.51/5.0 [0.75]; P <0.001), and higher likelihood of daily living activities being unaffected (88% vs 7%). Conclusions In this patient-centric study, lanadelumab LTP for ≥3 years was associated with reduced HAE attack frequency/severity, improved QoL, and high treatment satisfaction."

Clinical • Longitudinal study • Observational data • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema