Alhemo (concizumab-mtci) / Novo Nordisk - LARVOL DELTA

Rebalancing therapy for congenital hemophilia (PubMed, Rinsho Ketsueki) - "The anti-TFPI agents concizumab and marstacimab are administered subcutaneously and have been approved for use in Japan. The siRNA drug fitusiran is used as an anti-AT agent that reduces the synthesis of AT, and SerpinPC as an anti-APC agent that specifically inhibits APC. This article will outline the concept of rebalancing therapy and the results of clinical trials, as well as precautions and potential issues during treatment."

Journal • Review • Hematological Disorders • Hemophilia • Rare Diseases

Fitusiran (Qfitlia) for hemophilia A and B. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Pharmacokinetic evaluation of concizumab for the treatment of hemophilia. (PubMed, Expert Opin Drug Metab Toxicol) - "While the daily injection may seem demanding, it does not compromise the optimal benefits of concizumab prophylaxis. Moreover, the acceptable safe profile and efficacy of concizumab in bleed prevention in hemophilia A or B with and without inhibitors provide reassurance that it may be a therapeutic option in managing all hemophilia patients."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

PHYSICIANS OPTIMISTIC ABOUT NEXT EVOLUTION IN HEMOPHILIA PATIENT CARE (EHA 2025) - "Advancements in therapy are moving beyond factor replacement, with novel approaches such as gene therapy and non-factor therapies, including emicizumab, marstacimab, concizumab, fitusiran, and Mim8...The greatest challenges were cited as selecting the best treatment option (30%), patient adherence (28%) and securing insurance coverage (20%) - especially for newer options.45% of hematologists have made changes to the way they manage hemophilia A patients in the past year with more adoption of emicizumab and efanesoctocog alfa, for which they report high satisfaction... Managing hemophilia patients throughout their lifetime is challenging for hematologists as they aim to improve patient quality of life and lifestyle. Newer options are providing better efficacy for different patient types and with favorable administration. As these options are approved and launched, hematologists expect many patients will be candidates and they will quickly begin prescribing these agents as..."

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Pain • Pain • Rare Diseases

TFPI slows prothombinase assembly when concizumab is bound to its second Kunitz domain (ISTH 2025) - "Concizumab reversed TFPIα inhibition of fully assembled FXa-FV’ prothrombinase. However, the TFPIα-concizumab complex retains some anticoagulant activity towards prothrombinase because the TFPIα C-terminal tail remains available to slow assembly of FXa and FV’ into prothrombinase. Methods Enzyme kinetic studies using 15nM TFPIα, 0.1nM FXa, 16nM FV, 1.4µM prothrombin, phospholipids, 2.5mM calcium and 5-100nM concizumab."

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

The effect of concizumab on thrombin generation in FVII deficient plasma (ISTH 2025) - "Similar data were obtained in normal human plasma where FVII activity was neutralized with antibodies. Table or Figure Upload"

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Efficacy and convenience of concizumab prophylaxis in two patients affected by severe HAwI (ISTH 2025) - P3 | "Bleeding episodes required a total 351 mg rFVIIa, 100 mg for breakthrough bleedings and 251 mg for 2 surgeries. Table or Figure Upload"

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Non-joint bleeds in patients with hemophilia A or B with inhibitors: Concizumab explorer7 study (ISTH 2025) - P3 | "At the 56-week cut-off (Table 1), low numbers of non-joint bleeds were maintained with concizumab. Table or Figure Upload"

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Real-world efficacy of concizumab prophylaxis in a patient with hemophilia B and inhibitors (ISTH 2025) - "The patient’s quality of life improved significantly, with no bleeding-related hospitalizations. This case, the first in Taiwan, demonstrates concizumab’s transformative potential for hemophilia B with inhibitors, particularly in patients excluded from clinical trials."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Diabetes • Fibrosis • Hematological Disorders • Hemophilia • Hemophilia B • Hepatology • Immunology • Inflammation • Metabolic Disorders • Rare Diseases

Efficacy of concizumab prophylaxis in patients with Hemophilia:A systematic review and meta-analysis (ISTH 2025) - "No significant heterogeneity was detected (I² = 0%). Table or Figure Upload"

Retrospective data • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Annualized bleeding rates in hemophilia A/B and target joints: Concizumab explorer8 study (ISTH 2025) - P3 | "Median ABRs [interquartile range] for treated spontaneous and traumatic bleeding episodes during concizumab prophylaxis (arms 2–4) were low at 32 weeks for patients with (2.9 [0.4–6.4]) and without (1.6 [0.0–4.5]) target joints, remaining low at 56 weeks (Table 2). Table or Figure Upload"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

EVOLVING TREATMENTS IN HAEMOPHILIA: IMPACT OF BIOLOGIC THERAPY AND PROPHYLAXIS ON HEMARTHROSIS REDUCTION: REAL-WORLD EVIDENCE OF REDUCED HEMARTHROSIS BURDEN (EULAR 2025) - "To assess the association between switching to emicizumab (EM) treatment and the reduction in the number of H per year...Following the high prevalence of H and with the advances in recent years, 40 patients switched to biologic therapy (37 EM and 3 concizumab) and 10 changed the CF regimen from on-demand to prophylaxis regimen... The progress in the treatment of HA/HB in recent years has been exponential. Patients who switched their treatment to EM in our cohort had a significant reduction in H episodes. Our results confirm what has been observed in other studies and reinforce the need for a change in treatment to prevent haemophilic arthropathy, which causes so much comorbidity and detriment to quality of life."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Osteoarthritis • Rare Diseases • Rheumatology

Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations? (PubMed, Expert Rev Hematol) - "Anti-tissue factor pathway inhibitors (anti-TFPIs) that have completed Phase 3 clinical studies are concizumab and marstacimab. As hemophilia treatment goals continue to evolve, the role of currently developed anti-TFPIs is still not fully defined. This review comprehensively summarizes the clinical trial data, which shows that anti-TFPIs are not intended to replace the standard of care CFCs but to expand the therapeutic arsenal for patients with hemophilia treated with these therapeutic agents."

Journal • Review • Hematological Disorders • Hemophilia • Musculoskeletal Diseases • Rare Diseases

Novo Nordisk to present phase 3 trials across hemophilia portfolio, reinforcing commitment to research in rare blood disorders, at ISTH 2025 (PRNewswire) - "Key presentations include two updates from a phase 3 trial evaluating investigational treatment with Mim8 (denecimig) and five assessing treatment outcomes with concizumab in hemophilia; A phase 3 trial analysis from FRONTIER5 will evaluate the safety of switching directly from emicizumab to Mim8 (denecimig) in people living with hemophilia A/B; Findings from explorer7 and explorer8 phase 3 trials will assess data including non-joint bleeds, annualized bleeding rates and additional studies including thrombin generation with concizumab in hemophilia A/B."

P3 data • Hemophilia A • Hemophilia B

Explorer10: A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors (clinicaltrials.gov) - P3 | N=153 | Recruiting | Sponsor: Novo Nordisk A/S | N=90 ➔ 153 | Trial primary completion date: Jun 2026 ➔ Sep 2029

Enrollment change • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Concizumab, a Non-Replacement Therapy for Persons with Hemophilia with Inhibitors. (PubMed, J Clin Med) - "Whereas persons with HA with inhibitors can receive prophylaxis with a factor-mimicking agent, emicizumab, there is no recommendation for the agents to use as prophylaxis in persons with HB with inhibitors as there are no available molecules. Concizumab is a novel, subcutaneous prophylaxis option in persons with HA or HB with inhibitors that can potentially improve long-term outcomes. Here, we review the available data on concizumab and discuss its possible positioning in the armamentarium to treat hemophilia with inhibitors."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Evaluating the Safety and Efficacy of Concizumab in Hemophilia A/B Patients: A Systematic Review. (PubMed, Clin Appl Thromb Hemost) - "No thromboembolic events were reported.ConclusionConcizumab appears to be an effective and safe prophylactic treatment for patients with hemophilia A and B, demonstrating consistent reductions in bleeding rates and enhanced thrombin generation. Further long-term studies are warranted to establish its sustained safety and efficacy."

Journal • Review • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Case Report: Severe hemophilia B patient with inhibitor and anaphylaxis reaction to FIX, successfully managed with concizumab prophylaxis therapy. (PubMed, Front Pediatr) - "This case highlights concizumab's transformative role in managing hemophilia B with inhibitors, demonstrating its potential to address unmet clinical needs and improve outcomes, as corroborated by pivotal clinical trials. Comprehensive multidisciplinary care remains essential for optimizing long-term results."

Journal • Allergy • Hematological Disorders • Hemophilia • Hemophilia B • Pediatrics • Rare Diseases

Concizumab (Alhemo) for hemophilia A and B with inhibitors. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

explorer8: Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (clinicaltrials.gov) - P3 | N=158 | Active, not recruiting | Sponsor: Novo Nordisk A/S | Trial completion date: Jun 2026 ➔ Feb 2028

Trial completion date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Revolutionizing Treatment Strategies through Inhibition of Tissue Factor Pathway Inhibitor: A Promising Therapeutic Approach for Hemophilia Management. (PubMed, J Assoc Physicians India) - "Novel monoclonal antibodies (concizumab and marstacimab) enhance thrombin generation by blocking TFPI to restore hemostasis. These innovative therapies have the potential to enhance the quality of life (QoL) of people with hemophilia. This review provides a comprehensive overview of the clinical development, therapeutic potential, challenges, and prospects of anti-TFPI in the management of hemophilia."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. (PubMed, Res Pract Thromb Haemost) - P3 | "Estimated treatment difference at week 24 between patients in arms 1 and 2 was -18.0 points (95% CI, -26.4 to -9.5) for Haemophilia Quality of Life Questionnaire for Adults "total score" and -16.8 points (95% CI, -32.2 to -1.4) for "physical health." Hemophilia Treatment Experience Measure and Haemophilia Patient Preference Questionnaire results favored concizumab prophylaxis over no prophylaxis or previous treatment. PRO data from the phase 3 explorer8 study provided additional support for concizumab prophylaxis compared with no prophylaxis as a treatment option for patients with HA/HB."

Journal • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Pain • Rare Diseases

Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo (clinicaltrials.gov) - P=N/A | N=30 | Not yet recruiting | Sponsor: Novo Nordisk A/S

New trial • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

CDSCO Approves Novo Nordisk Protocol Amendment Proposal For Concizumab Study (Medical Dialogues) - "The Subject Expert Committee (SEC) functional under the Central Drug Standard Control Organisation (CDSCO) has approved the protocol amendment proposal presented by Novo Nordisk to study Concizumab in patients with hemophilia A or B with inhibitors...This came after Novo Nordisk presented protocol amendment version 11.0 dated 07 October 2024 protocol no. NN7415-4311 (EXPLORER 7)....After detailed deliberation, the committee recommended the approval of the protocol amendment as presented by the firm."

Clinical protocol • Hemophilia A • Hemophilia B

Explorer10: A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors (clinicaltrials.gov) - P3 | N=90 | Recruiting | Sponsor: Novo Nordisk A/S | Trial primary completion date: Dec 2024 ➔ Jun 2026

Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases