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

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

EKTERLY (sebetralstat) (Businesswire) - "Initiated US commercial launch on July 7, 2025 and received 460 patient start forms for the period ended August 29, 2025; Cost of revenue was $0.6 million for the three months ended July 31, 2025 which included EKTERLY manufacturing and inventory overhead costs incurred after US approval."

Commercial • Hereditary Angioedema

The Committee for Orphan Medicinal Products (COMP) of the EMA confirmed maintenance of orphan designation for sebetralstat. (Businesswire) - "The decision was based on a finding of comparable efficacy to injectable on-demand treatments with a major contribution to patient care by reducing the HAE attack morbidity."

European regulatory • Orphan drug • Hereditary Angioedema

Long-term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension. (PubMed, J Allergy Clin Immunol Pract) - P3 | "Oral sebetralstat enabled compliance with treatment guidelines. No new safety signals were observed, and effectiveness for repeated attacks was consistent with the KONFIDENT trial results."

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

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial (clinicaltrials.gov) - P3 | N=11 | Active, not recruiting | Sponsor: KalVista Pharmaceuticals, Ltd. | Recruiting ➔ Active, not recruiting

Enrollment closed • Cardiovascular • Complement-mediated Rare Disorders • Hereditary Angioedema

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 July 2025 (European Medicines Agency) - "On 24 July 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Ekterly, intended for the symptomatic treatment of acute attacks of hereditary angioedema in adults and adolescents from 12 years of age. The applicant for this medicinal product is KalVista Pharmaceuticals (Ireland) Ltd."

CHMP • Hereditary Angioedema

MHRA approves sebetralstat (Ekterly) to treat hereditary angioedema (HAE) attacks in patients aged 12 and over (GOV.UK) - "The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 15 July 2025, approved sebetralstat (Ekterly) for the treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older....Sebetralstat’s approval is supported by the KONFIDENT clinical trial, in which 110 patients experienced and treated 264 hereditary angioedema attacks."

MHRA approval • Hereditary Angioedema

Sebetralstat. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Orsini Selected as Specialty Pharmacy Partner for KalVista's EKTERLY (sebetralstat) (PRNewswire) - "Orsini, a leader in rare disease pharmacy solutions, has been chosen by KalVista Pharmaceuticals as a specialty pharmacy partner for EKTERLY (sebetralstat), an FDA-approved oral therapy for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older."

Commercial • Hereditary Angioedema

KalVista Pharmaceuticals Announces FDA Approval of EKTERLY (sebetralstat), First and Only Oral On-demand Treatment for Hereditary Angioedema (Businesswire) - "KalVista Pharmaceuticals, Inc...announced that the U.S. Food and Drug Administration (FDA) has approved EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. EKTERLY is the first and only oral on-demand treatment for HAE...The efficacy and safety of EKTERLY was established by the results from KalVista’s phase 3 KONFIDENT clinical trial, which was the largest clinical trial program ever conducted in HAE...These results were further supported by the more real-world KONFIDENT-S open-label extension trial..."

FDA approval • Hereditary Angioedema

IL-1ß INDUCE CAR-T-ASSOCIATED COAGULOPATHY VIA THE HSP90/KALLIKREIN/UPA PATHWAY (EHA 2025) - "These manifestations could be mitigated by IL-1β receptor inhibitor (Anakinra), Hsp90 inhibitor (17-DMAG), and kallikrein inhibitor (Sebetralstat) (Fig1 G-J). IL-1β is responsible for CARAC, through promoting 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

IL-1ß Induce CAR-T-associated coagulopathy via the Hsp90/Kallikrein/uPA Pathway (ISTH 2025) - "Aims To explore the potential pathogenesis and treatment strategy of CARAC...These manifestations could be mitigated by IL-1β receptor inhibitor (Anakinra), Hsp90 inhibitor (17-DMAG), and kallikrein inhibitor (Sebetralstat) (Fig1 G-J). Additionally, anakinra suppressed the expression of Hsp90, 17-DMAG decreased kallikrein production, ultimately decreasing uPA levels and ameliorating coagulopathy (Figure1 K-M). Table or Figure Upload"

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