Takhzyro (lanadelumab-flyo) / Takeda - LARVOL DELTA

FAERS Pharmacovigilance Disproportionality Analysis: Serious Adverse Events Associated with Lanadelumab Administration for Hereditary Angioedema Prophylaxis (EADV 2026) - No abstract available

Adverse events • Serious adverse event • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Isolated gastrointestinal hereditary angioedema associated with a pathogenic SERPING1 variant arising de novo: a case report. (PubMed, Front Med (Lausanne)) - "An abdominal attack in May 2025 resolved rapidly after icatibant administration. Long-term prophylaxis (LTP) with lanadelumab was subsequently initiated following an individualized shared decision-making process...This report highlights that HAE should be considered in patients with recurrent, unexplained, self-limiting abdominal pain, and C4 should be included in the initial evaluation. A negative family history does not exclude HAE."

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

Impact of Lanadelumab Prophylaxis on Quality of Life In Pediatric Hereditary Angioedema Patients and Caregivers (ACAAI 2026) - No abstract available

Clinical • HEOR • Late-breaking abstract • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Beyond the Bowel: Recurrent Abdominal Pain Unmasking Hereditary Angioedema (ACG 2026) - "Therapy for acute attacks include plasma-derived C1-INH and long-term prophylaxis with lanadelumab...HAE should be on the differential for recurrent, episodic abdominal pain, particularly when imaging shows a migratory pattern of inflammation with unrevealing gastrointestinal evaluations, even in the absence of cutaneous swelling. Early recognition is essential to prevent unnecessary procedures, reduce morbidity, and mitigate the risk of life-threatening laryngeal attacks, especially given the availability of highly effective targeted therapies."

Cardiovascular • Complement-mediated Rare Disorders • Gastroenterology • Gastrointestinal Disorder • Hereditary Angioedema • Immunology • Inflammation • Inflammatory Bowel Disease • Pain • Urticaria

Chronic Spontaneous Urticaria with Biochemical C1 Inhibitor Deficiency: A Case Report of Suspected Overlap with Hereditary Angioedema. (PubMed, Clin Cosmet Investig Dermatol) - "Documented treatment included H1 antihistamines, systemic corticosteroids, cyclosporine, and omalizumab. Subcutaneous icatibant and lanadelumab were also administered, but the retrospective records did not permit reliable assessment of icatibant response, and lanadelumab exposure was too brief to assess prophylactic efficacy...In patients with CSU, recurrent throat tightness or abdominal pain should prompt complement testing and episode-level assessment. Objective airway findings, abdominal evaluation, attack timing, complement results, and response to on-demand therapy should be documented before symptoms are attributed to a specific mechanism."

Journal • Cardiovascular • Chronic Spontaneous Urticaria • Complement-mediated Rare Disorders • Dermatology • Hereditary Angioedema • Immunology • Pain • Pruritus • Urticaria

Diagnostic delay, SERPING1 allelic heterogeneity, and real-world lanadelumab prophylaxis in Chinese patients with hereditary angioedema due to C1 inhibitor deficiency. (PubMed, Front Allergy) - "Earlier complement testing and access to appropriate genetic testing may shorten diagnostic delay. Larger prospective studies are needed to define individualized long-term prophylaxis strategies in Chinese patients."

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

Pediatric hereditary angioedema presenting as recurrent episodic abdominal pain and vomiting: Challenges in diagnosis and management. (PubMed, JPGN Rep) - "We present the challenging diagnostic odyssey of a pediatric patient (symptom onset at age 4 years; diagnosis at age 8), culminating in an unexpected diagnosis of hereditary angioedema, a critical etiology often omitted from differentials. This case underscores a key gap in current clinical pathways and serves to expand the diagnostic consideration for specialists managing unexplained recurrent episodic abdominal symptoms."

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

The evolving treatment landscape for hereditary angioedema in Sweden. (PubMed, Front Immunol) - "Among patients with ≥12 months on modern LTP (n=69) who had at least one ODT dispensation event during their most recent 12 months of treatment (n=46/59 for lanadelumab; n=8/9 for berotralstat), the mean number of ODT packages dispensed-in addition to emergency stock ODT-was 4.3 per patient on lanadelumab and 6.5 per patient on berotralstat. This study provides valuable insights into real-world dispensation patterns of HAE drugs in Sweden. Despite the availability of modern LTP therapies, there remains relatively high ODT dispensation events, suggesting ongoing unmet need and underscoring the importance of optimizing prophylactic strategies to achieve total disease control."

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

Lanadelumab Use for Hereditary Angioedema Long-Term Prophylaxis Over the Last 7 Years: A Narrative Review of Clinical and Real-World Data. (PubMed, Clin Rev Allergy Immunol) - "Future research should address real-world outcomes in pediatric patients, extended dosing intervals in well-controlled disease, transitions from other LTP therapies, and cost-effectiveness. Overall, sustained effectiveness, safety, and quality-of-life benefits of lanadelumab reinforce its role as a cornerstone of HAE management."

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

Sustained Effectiveness of Lanadelumab in Preventing Hereditary Angioedema Attacks: The ENABLE Study. (PubMed, Clin Transl Allergy) - P | "Real-world data from ENABLE demonstrated long-term effectiveness of lanadelumab in patients with HAE aged ≥ 12 years and a safety profile consistent with previous clinical studies."

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

Current and future therapies for bradykinin-mediated angioedema (PubMed, Dermatologie (Heidelb)) - "On-demand treatment options include plasma-derived and recombinant C1 inhibitor (C1INH) concentrates, the bradykinin B2 receptor antagonist icatibant, and, more recently, the first orally available plasma kallikrein inhibitor, sebetralstat...LTP therapies include subcutaneous and intravenous C1INH preparations, the oral kallikrein inhibitor berotralstat, the anti-kallikrein monoclonal antibody lanadelumab, the factor XIIa inhibitor garadacimab, and the antisense oligonucleotide donidalorsen. Currently under development are the oral bradykinin B2 receptor antagonist deucrictibant, which is intended for both on-demand treatment and long-term prophylaxis in different formulations, long-acting antibodies, such as navenibart, and CRISPR/Cas9-based gene-editing therapies, such as NTLA-2002 with potential functional curative properties. In particular, orally available and long-acting therapies are expected to improve adherence, self-management, and quality of life in..."

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

A Survey of Lanadelumab in Participants With Hereditary Angioedema (clinicaltrials.gov) - P=N/A | N=155 | Active, not recruiting | Sponsor: Takeda | Trial completion date: Jun 2026 ➔ Oct 2026 | Trial primary completion date: Jun 2026 ➔ Oct 2026

Trial completion date • Trial primary completion date • 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=37 | Completed | Sponsor: Takeda | Recruiting ➔ Completed

Real-world evidence • Trial completion • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics

Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S. (PubMed, J Allergy Clin Immunol Glob) - P3 | "This interim analysis of the KONFIDENT-S study evaluated long-term safety and effectiveness of oral sebetralstat 600 mg for attacks of hereditary angioedema with C1-inhibitor deficiency in participants receiving LTP with lanadelumab, berotralstat, or C1 inhibitor. Sebetralstat was well tolerated and enabled early on-demand treatment of attacks in patients with hereditary angioedema with C1-inhibitor deficiency receiving LTP. Treatment of breakthrough attacks with sebetralstat resulted in rapid symptom relief, reduction in attack severity, and complete attack resolution, regardless of LTP mechanism of action."

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

Donidalorsen for the Treatment of Hereditary Angioedema: A Review of Clinical Studies. (PubMed, Drug Des Devel Ther) - "A notable study in the clinical program included a cohort of patients who switched from berotralstat, C1 inhibitor, or lanadelumab to donidalorsen for up to 1 year; mean attack rates were reduced by 68% vs baseline (on prior HAE prophylaxis). Across studies, donidalorsen had an acceptable safety and tolerability profile, with mostly mild to moderate adverse events reported. Overall, the clinical data are promising for donidalorsen as a long-term prophylactic medication for HAE."

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

Clinical experience with berotralstat in patients with hereditary angioedema: an Italian case series from the ITACA cohort. (PubMed, Eur Ann Allergy Clin Immunol) - "The latest international guidelines recommend as first-line options for LTP plasma-derived C1-INH, lanadelumab, and berotralstat. Conclusions. Patient engagement, awareness of androgen risks, transition strategies and drug-interaction evaluation are essential to guide individualized LTP choices."

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

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

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

Hereditary angioedema attack trends among patients maintained on lanadelumab long-term prophylaxis. (PubMed, J Allergy Clin Immunol Glob) - "Patients on lanadelumab for at least 6 months had low discontinuation rates. Attack rates were low, and the proportion of attack-free patients increased across intervals, indicating attack-free status was achievable in the later years on treatment."

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

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 | Initiation date: Jul 2026 ➔ Oct 2026

Trial initiation date • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Characterization of hereditary angioedema population in Argentina: A nationwide study. (PubMed, World Allergy Organ J) - "Icatibant was the most prescribed drug (76.91%). Long-term prophylaxis (LTP) was used in 28.34% of subjects, with lanadelumab being the most prescribed agent (58.45%). Argentine HAE patients share several characteristics with patients from real-world studies, but some findings (time to diagnosis, low LTP rates, relatively high rates of HAE-FXII and a novel, recently characterized form of HAE-DAB2IP) require further research."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • DAB2IP

Recurrent abdominal attacks as a presentation of hereditary angioedema type I: a multigenerational family series. (PubMed, Rev Esp Enferm Dig) - "After diagnosis, the index case received on-demand icatibant and initiated long-term prophylaxis with lanadelumab, remaining asymptomatic at the most recent follow-up. This series highlights the importance of considering HAE in patients with unexplained recurrent abdominal pain, edema without urticaria, and a suggestive family history, particularly when imaging suggests reversible small-bowel involvement, in order to reduce diagnostic delay and prevent potentially avoidable interventions."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Hereditary Angioedema • Immunology • Pain • Urticaria

Lanadelumab in Hereditary Angioedema: Extending Dose Intervals from Every 2 Weeks to Every 4 Weeks. (PubMed, Int Arch Allergy Immunol) - "Although there are inherent limitations related to analyzing small sample sizes for sub-groups of patients with a rare disease and for data derived from real-world studies, these data can inform physicians and patients about the characteristics and outcomes of patients who have successfully extended dosing intervals of lanadelumab LTP. This may in turn encourage regular monitoring to facilitate optimization of treatment and outcomes for each patient."

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

A novel SERPING1 splice-site variant (c.1029 + 2T > A) causing hereditary angioedema type I: functional characterization and clinical analysis. (PubMed, Orphanet J Rare Dis) - "We identified and functionally characterized a novel SERPING1 splice-site variant, c.1029 + 2T > A, which disrupts canonical mRNA splicing, primarily through exon 6 skipping, leading to C1-INH haploinsufficiency and type I HAE. Our retrospective analysis expands the mutational spectrum of SERPING1 and demonstrates the clinical efficacy of Lanadelumab as a long-term prophylactic therapy. These findings underscore the importance of integrating genetic testing and functional validation in the diagnosis and management of HAE."

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

The Neglected Older Adults in Hereditary Angioedema: Insights From the ITACA Registry. (PubMed, Clin Transl Allergy) - "Older patients with HAE-C1INH constitute a relevant subgroup, characterized by persistent disease activity and comorbidities. The availability of new therapies and guideline recommendations are driving an increase in LTP use, although shifting from older non-specific treatments, especially androgens, is still incomplete."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Coronary Artery Disease • Diabetes • Dyslipidemia • Endocrine Disorders • Hepatitis B • Hepatitis C • Hereditary Angioedema • Hypertension • Metabolic Disorders • Oncology • Rare Diseases

Real-world treatment patterns and clinical burden of patients with hereditary angioedema treated with long-term prophylaxis. (PubMed, Orphanet J Rare Dis) - "Although recently available LTP can partly reduce the clinical burden of patients with HAE, novel therapies are needed to further alleviate this burden."

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