Ekterly (sebetralstat) / KalVista, Kaken Pharma - LARVOL DELTA

Impact of Oral Sebetralstat on Anxiety Associated With Hereditary Angioedema Attacks. (PubMed, Clin Exp Allergy) - P3 | "Moderate-to-extreme anxiety was common in HAE attacks. Reduction in anxiety was significantly greater in attacks treated with sebetralstat compared with placebo."

Journal • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Mood Disorders • Psychiatry • STAT3

COST-EFFECTIVENESS OF RECOMBINANT HUMAN C1 ESTERASE INHIBITOR (RHC1-INH) VS SEBETRALSTAT FOR THE TREATMENT OF SEVERE OR VERY SEVERE HEREDITARY ANGIOEDEMA (HAE) ATTACKS (ISPOR 2026) - P2/3, P3 | "For severe or very severe HAE attacks, rhC1-INH treatment offers more attack-free time, cost savings, and QALH gains driven by faster time to complete resolution and reduced need for redosing compared with sebetralstat."

Cost effectiveness • HEOR • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Rare Diseases

On-demand Treatment Patterns of Hereditary Angioedema (HAE) Attacks with Sebetralstat in the KONFIDENT-S Study (AAAAI 2026) - "Conclusions Given that participants could utilize conventional treatment for attacks, the preference for sebetralstat for on-demand treatment of most attacks in KONFIDENT-S was evident. A greater proportion of attacks were treated in KONFIDENT-S compared to real-world observational studies, possibly due to reduced barriers to treatment with sebetralstat, leading to a higher willingness of participants to treat attacks; particularly attacks that were mild in severity."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Response Drivers in Sebetralstat Placebo-controlled Clinical Trials (AAAAI 2026) - "Using the final model, a simulation (n=5000) of a single sebetralstat 600mg dose administered within 30 minutes of attack onset provided symptom relief for 88.5% (95% CI: 81.2% - 93.9%) of simulation subjects within 12 hours. Conclusions In this modeling and simulation using placebo-controlled clinical trial data, time to treatment (<30 minutes) was the most robust driver of endpoint achievement, highlighting the importance of early treatment in accordance with treatment guidelines, and underscoring the value of oral on-demand treatment."

Clinical • Dermatology

The power of a pill: Ekterly (Sebetralstat): first oral on-demand therapy for hereditary angioedema-a paradigm shift in treatment. (PubMed, Ann Med Surg (Lond)) - No abstract available

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

Sebetralstat (Ekterly) for treatment of hereditary angioedema attacks. (PubMed, Med Lett Drugs Ther) - No abstract available

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

KONFIDENT-KID: Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II (clinicaltrials.gov) - P3 | N=36 | Completed | Sponsor: KalVista Pharmaceuticals, Ltd. | Recruiting ➔ Completed | Trial completion date: Aug 2027 ➔ Jan 2026 | Trial primary completion date: Aug 2027 ➔ Jan 2026

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

Novel drug approval by the FDA: Ekterly (Sebetralstat), oral plasma kallikrein inhibitor for treating acute attacks of hereditary angioedema. (PubMed, Ann Med Surg (Lond)) - "Through a detailed appraisal of its therapeutic efficacy, safety profile, and pharmacologic constraints -including clinically relevant interactions with CYP3A4 modulators and the lack of evidence in pregnant patients or those with hepatic impairment ,this editorial emphasizes Ekterly's emerging role as an important and potentially transformative addition to HAE management.The approval of sebetralstat signals a new era of convenient, rapid-acting, accessible treatment for HAE, with the potential to reduce emergency visits, enhance patient autonomy, and improve quality of life. Continued surveillance, long-term safety studies, and global access initiatives will be essential to maximize its public-health impact and ensure equitable availability of this breakthrough oral therapy."

FDA event • Journal • Cardiovascular • Complement-mediated Rare Disorders • Hepatology • Hereditary Angioedema • Rare Diseases • CYP3A4

KalVista Pharmaceuticals Announces Approval of EKTERLY (sebetralstat) in Japan, First and Only Oral On-demand Treatment for Hereditary Angioedema (Businesswire) - "KalVista Pharmaceuticals, Inc...announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing approval for EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older...EKTERLY will be commercialized in Japan by KalVista’s partner, Kaken Pharmaceutical Co., Ltd. Kaken will launch EKTERLY shortly after it is listed on the Japanese National Health Insurance System (NHI)...The approval is based on results from the phase 3 KONFIDENT clinical trial..."

Japan approval • Launch Japan • Hereditary Angioedema

Sebetralstat as a patient-centered alternative to lanadelumab: Insights from recent real-world evidence. (PubMed, Ann Allergy Asthma Immunol) - No abstract available

HEOR • Journal • Real-world evidence

Assessing the Adoption of Novel Therapies in a Private/Public Payer System: A Study in Fitusiran, Sebetralstat, and Nipocalimab (ASHP 2025) - No abstract available

Sebetralstat: a paradigm shift in hereditary angioedema management. (PubMed, Ann Med Surg (Lond)) - No abstract available

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

Sebetralstat for On-Demand Treatment of Mucosal Hereditary Angioedema Attacks in KONFIDENT-S. (PubMed, Clin Transl Allergy) - P3 | "Oral sebetralstat enabled rapid treatment of laryngeal and abdominal attacks of all severities, was well tolerated, and provided early symptom relief."

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

IL-1β Induce CAR-T-Associated Coagulopathy Via the Hsp90/Kallikrein/uPA Pathway (ASH 2024) - "These manifestations can be improved by IL-1β receptor inhibitor (Anakinra), Hsp90 inhibitor (17-DMAG hydrochloride), and kallikrein inhibitor (sebetralstat). Conclusion CARAC is characterized by bleeding and hypofibrinogenemia and is closely related to the inflammation-immune-endothelium-coagulation-fibrinolysis network. IL-1β of CRS promotes the release of Hsp90 by endothelial cells, which subsequently induces hyperfibrinolysis through the kallikrein-uPA pathway."

IO biomarker • Gastroenterology • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Oncology • CDC37 • HSP90AA1 • HSP90AB1 • IL1B • IL6 • VCAM1

Bradykinin-Mediated Angioedema Complicating Hospitalization For Behcet's-like Disease (ACAAI 2025) - "Prodromal throat discomfort began >24 hours prior to peak swelling, preceded by menstruation, ACE-inhibitor use one week earlier, and infliximab initiation five days prior...After extubation, she had recurrent angioedema that responded well to icatibant...She was discharged with sebetralstat, an oral plasma kallikrein inhibitor, for rescue. This case underscores mechanism-guided therapy to angioedema, regardless of molecular diagnosis, especially when laboratory findings are pending or inconclusive."

Clinical • Cardiovascular • Dermatology • Immunology • Pruritus • Urticaria

Satisfaction with Sebetralstat for HAE Attacks in Patients Switching from Parenteral On-demand Treatments in KONFIDENT-S (ACAAI 2025) - P3 | "Abstract Introduction Here, we present interim efficacy and treatment satisfaction data from patients who switched from icatibant, pdC1INH, rC1INH, or multiple parenteral on-demand treatments to oral sebetralstat to participate in KONFIDENT-S (NCT05505916). Among the 989 attacks rated as neutral (0) or satisfied (1-3), 79 were neutral and 715 were rated as very (2) or extremely satisfied (3). Conclusion Most participants who switched from parenteral treatment were very satisfied or extremely satisfied with oral sebetralstat as an on-demand treatment."

Clinical

Cost-Effectiveness of Recombinant C1 Inhibitor (rhC1-INH) Versus Sebetralstat for Treatment of Hereditary Angioedema (HAE) Attacks (ACAAI 2025) - "Treatment with rhC1-INH led to 10.3 additional attack-free days, 9.4 fewer doses, 79.0 QALHs gained, and cost savings of $183,293 annually, suggesting rhC1-INH is cost-effectively dominant. Conclusion Compared with sebetralstat, rhC1-INH is associated with more attack-free time, cost savings, and a gain in QALHs due to faster time to complete resolution and less redosing for treating HAE attacks."

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

Indirect Treatment Comparison (ITC) of Recombinant C1 Inhibitor and Sebetralstat for Hereditary Angioedema On-Demand Therapy (ACAAI 2025) - P2/3, P3 | "Results were comparable across unanchored/anchored analyses. Conclusion This ITC suggests rhC1-INH may significantly improve time to resolution and reduce the need for redosing compared with sebetralstat 300 mg and 600 mg in patients with HAE."

Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • STAT3

Sebetralstat for On-demand Treatment of Hereditary Angioedema in Pediatric (2-11y) Patients: Interim Results from KONFIDENT-KID (ACAAI 2025) - P3 | "Conclusions Observed pharmacokinetic sebetralstat concentrations align with popPK-based simulations. Sebetralstat enabled early treatment and was well tolerated and effective among pediatric patients with HAE-C1INH."

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

Sebetralstat: FDA approves first oral on-demand therapy for hereditary angioedema. (PubMed, Ir J Med Sci) - No abstract available

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

Self-administered oral sebetralstat: a game-changer in treatment of hereditary angioedema. (PubMed, Ann Med Surg (Lond)) - No abstract available

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

Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat (clinicaltrials.gov) - P=N/A | N=0 | Available | Sponsor: KalVista Pharmaceuticals, Ltd.

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

Sebetralstat: First Approval. (PubMed, Drugs) - "Sebetralstat has also received a positive opinion in the EU for the symptomatic treatment of acute attacks of HAE in adults and adolescents aged 12 years and older. This article summarizes the milestones in the development of sebetralstat leading to this first approval for HAE."

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

KalVista Pharmaceuticals Announces European Commission and Swissmedic Approval of EKTERLY (sebetralstat), First and Only Oral On-Demand Treatment for Hereditary Angioedema (Yahoo Finance) - "The EC and Swissmedic marketing authorizations for EKTERLY are based on results from the phase 3 KONFIDENT clinical trial..."

Approval • EMA approval • Hereditary Angioedema

KONFIDENT-KID: Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II (clinicaltrials.gov) - P3 | N=48 | Recruiting | Sponsor: KalVista Pharmaceuticals, Ltd. | N=36 ➔ 48

Enrollment change • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema • Pediatrics