Hympavzi (marstacimab-hncq) / Pfizer - LARVOL DELTA

Clinical Pharmacokinetics and Pharmacodynamics of Marstacimab, an Anti-tissue Factor Pathway Inhibitor Monoclonal Antibody, in Adolescent and Adult Participants with Hemophilia. (PubMed, Clin Pharmacokinet) - P1, P2, P3 | "A TMDD model with first-order absorption and quasi-steady-state approximation adequately characterized marstacimab PK and total TFPI concentrations. An Emax model adequately described the relationship between model-predicted free TFPI and peak thrombin. The PK/PD simulation indicated that no changes in dosing regimen based on age or body weight were warranted."

Journal • PK/PD data • Hematological Disorders • Hemophilia • Hepatology • Rare Diseases

Transforming Hemophilia Treatment With Novel Rebalancing Agents: Clinical Studies and Practical Perspectives. (PubMed, Clin Appl Thromb Hemost) - "Rebalancing agents, including fitusiran (antithrombin-lowering small interfering ribonucleic acid), concizumab and marstacimab (tissue factor pathway inhibitor antagonists), are novel nonfactor therapies that restore hemostatic balance by targeting natural anticoagulants rather than replacing missing clotting factors. In addition, the use of rebalancing agents in pediatric patients (<12 years old) remains under investigation. While promising, successful implementation depends on individualized treatment approaches, appropriate monitoring protocols, and thorough patient education to improve current care for PwH."

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

Bridging the Gap: A Systematic Review of Modern Hemophilia Therapies and Global Inequities in Clinical Trial Participation. (PubMed, Haemophilia) - "Modern therapies demonstrate strong efficacy and acceptable safety, with several approved agents. However, persistent global inequities in trial participation and access highlight the need for more inclusive research and equitable implementation strategies."

Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Pediatrics • Rare Diseases

New and novel pharmacotherapies for hemophilia A: an update. (PubMed, Expert Opin Pharmacother) - "In replacement therapy, efanesoctocog alfa maintains normal-to-near-normal factor VIII (FVIII) levels weekly by bypassing endogenous von Willebrand factor dependence. In non-replacement therapies, the focus centers on rebalancing agents - the anti-tissue factor pathway inhibitor (TFPI) monoclonal antibodies concizumab and marstacimab, and the antithrombin-targeting small interfering RNA (siRNA) fitusiran - as well as next-generation FVIII-mimetics like denecimig...Rebalancing therapies present potential thromboembolic risks, complex breakthrough bleed protocols, and standard laboratory assay interference (requiring antithrombin monitoring or specialized assays). Critical goals for contemporary hemophilia management include tailoring therapies through multidisciplinary collaboration, monitoring subclinical joint disease via point-of-care ultrasound, and implementing standardized, real-world protocols for emergency hemostasis."

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

BASIS KIDS Phase 3 Trial Releases Encouraging Interim Data on Marstacimab for Hemophilia A, B (DocWire) - "The interim data were from 92 patients in these two age groups with hemophilia A or B; among these patients, 73% comprised a without-inhibitors subcohort, and 27%, a with-inhibitors subcohort. At the time that the interim data were compiled, 58.7% of the total cohort had completed the study, 38.0% remained in the study, and 3.3% had discontinued the study."

P3 data • Hemophilia A • Hemophilia B

Advances in treatments for hemophilia A/B with inhibitors: current treatment gaps and promising therapies. (PubMed, Expert Rev Hematol) - "A literature search was conducted using PubMed/MEDLINE, Embase, and Google Scholar for publications from January 2000 to March 2026 using combinations of the terms 'hemophilia A,' 'hemophilia B,' 'inhibitors,' 'immune tolerance induction,' 'bypassing agents,' 'emicizumab,' 'concizumab,' 'marstacimab,' 'fitusiran,' and 'gene therapy.' Relevant clinical trials, observational studies, guidelines, and review articles were evaluated. Despite transformative progress, important unmet needs remain, including incomplete hemostatic control, lack of predictive biomarkers, limited laboratory standardization, and inequitable global access. Future advances will depend on biomarker-guided personalized therapy, rational combination strategies, and integration of immune-modulatory and gene-based approaches to achieve durable tolerance and potentially curative outcomes."

Journal • Review • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Preventive care • Rare Diseases

Indirect Comparison of Marstacimab, Emicizumab, and Efanesoctocog Alfa in Hemophilia A Prophylaxis (ISTH 2026) - "Formal network meta-analysis needed for definitive conclusions. Table or Figure Upload (1) table 1 Page 2 Table or Figure Upload (2) Figure 1 DOI*10.1016/j.rpth.2026.104831"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Marstacimab prophylaxis in pediatric participants with hemophilia A or B with or without inhibitors: interim results from the phase 3 BASIS KIDS trial (ISTH 2026) - No abstract available

Clinical • Late-breaking abstract • P3 data • P3 data: top line • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Pediatrics • Rare Diseases

A Real world evidence on patients' preference among Chinese Adults and Adolescent Patients with Hemophilia on Marstacimab in Bo'ao pilot zone (ISTH 2026) - "Marstacimab injections frequency of hemophilia patients. DOI*10.1016/j.rpth.2026.104878"

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

Pharmacokinetic Variability and Dosing Strategy Heterogeneity in Anti‑TFPI Therapies: Implications for Plasma Monitoring (ISTH 2026) - "Aims To explore inter-patient PK variability between marstacimab and concizumab in patients WITHOUT inhibitors, evaluate dose adjustment requirements, and assess whether PK variability characteristics support the rationale for plasma monitoring in anti ‑ TFPI therapies. These observations are hypothesis ‑ generating and warrant prospective validation. Table or Figure Upload (1) Table 1 Page 2 Table or Figure Upload (2) Table 2 DOI*10.1016/j.rpth.2026.104814"

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

Marstacimab in Severe Haemophilia B without inhibitors: First UK Real‑World Experience – A Case Series (ISTH 2026) - "Ongoing national data will clarify long ‑ term safety, outcomes and implementation across UK haemophilia centres. DOI*10.1016/j.rpth.2026.104911"

Clinical • Real-world • Real-world evidence • Autism Spectrum Disorder • Cardiovascular • Developmental Disorders • Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases • Renal Disease

Anti-Tissue Factor Pathway Inhibitor Prophylaxis with Marstacimab in a Severely Obese Adolescent with Hemophilia B (ISTH 2026) - "Conclusions Anti-TFPI prophylaxis with Marstacimab enabled sustained bleed prevention, preserved joint health, supported safe sports participation and improved the quality of life significantly in a severely obese adolescent with severe hemophilia B who also suffered from i.v. needle phobia. DOI*10.1016/j.rpth.2026.104888"

Hematological Disorders • Hemophilia • Hemophilia B • Obesity • Rare Diseases

UK Laboratory Consensus on Coagulation testing of patients receiving marstacimab: Findings from the first structured review of BASIS clinical trial data (ISTH 2026) - "Spiking studies, external quality assurance (EQA) participation, and field studies are recommended to verify assay performance. Further research into combined therapy approaches and the management of urgent clinical scenarios is needed Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104882"

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

Marstacimab versus Concizumab in Hemophilia A/B Without Inhibitors: A Descriptive Comparison of Pivotal Trials (ISTH 2026) - "With comparable final ABR despite heterogeneous trial designs, treatment choice between anti-TFPI agents should prioritize patient-specific thrombotic risk, dosing preference, and monitoring capacity rather than cross-trial efficacy contrasts. Active thrombotic surveillance remains essential for both therapies Table or Figure Upload (1) Page 2 Table or Figure Upload (2) Page 3 DOI*10.1016/j.rpth.2026.104872"

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A Novel Functional Assay Using a Two-Step Diluted Prothrombin Time for Monitoring Marstacimab Therapy (ISTH 2026) - "The TS-dPT assay may serve as a practical method for monitoring marstacimab therapy; however, further clinical validation is warranted. Page 2 DOI*10.1016/j.rpth.2026.104807"

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

Marstacimab Prophylaxis in Participants With Hemophilia A or B With Inhibitors With Prior Routine Prophylaxis Use: Results From the Phase 3 BASIS Trial (ISTH 2026) - P3 | "Conclusions In participants with hemophilia with inhibitors and prior RP, marstacimab demonstrated a favorable efficacy and safety profile, consistent with the inhibitor cohort with prior OD and the non-inhibitor cohort of the BASIS trial. Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104305"

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

Pharmacokinetics and Pharmacodynamics of Marstacimab in Adults and Adolescents With Hemophilia With and Without Inhibitors (ISTH 2026) - P3 | "Conclusions No clinically relevant differences were seen in marstacimab PK and PD between participants with hemophilia (adult and adolescent) with and without inhibitors. Table or Figure Upload (1) Page 2 DOI*10.1016/j.rpth.2026.104297"

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

A plain language summary of the BASIS study looking at people living with severe hemophilia A or B with inhibitors and being treated with marstacimab. (PubMed, Ther Adv Hematol) - "What do the results mean? The study showed that marstacimab could help prevent bleeding in people living with hemophilia A or B with inhibitors."

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

EVOLVING TREATMENT PRIORITIES IN HEMOPHILIA: PHYSICIAN AND PATIENT PERSPECTIVES ON CONVENIENCE, QUALITY OF LIFE, AND THE EMERGING ROLE OF REBALANCING AGENTS ACROSS THE US AND EU5 (EHA 2026) - "Rebalancing agents such as fitusiran (Qfitlia) , marstacimab (Hympavzi) , and concizumab (Alhemo) have introduced opportunities to reduce treatment burden and better align with patient lifestyle and preference, marking a fundamental evolution in disease management. Summary/Conclusion The emergence of rebalancing agents has accelerated a global paradigm shift toward shared decision-making and individualized care in hemophilia. As treatment expectations expand beyond bleed control to encompass lifestyle and mental well-being, convenience and patient voice have become defining measures of therapeutic success."

Clinical • HEOR • Hematological Disorders • Hemophilia • Rare Diseases

ELICITING EXPERT RECOMMENDATIONS ON THE DOSING OF FACTOR REPLACEMENT THERAPY WITH MARSTACIMAB FOR BREAKTHROUGH BLEEDING & PERIOPERATIVE SITUATIONS IN HAEMOPHILIA: INTERIM RESULTS OF A DELPHI STUDY (EHA 2026) - "Summary/Conclusion Preliminary findings provide early insights into dosing recommendations for SHL FRT with marstacimab in the management of BTB and perioperative situations. The dosing ranges recommended in the first-round survey align with management practices observed in marstacimab clinical studies."

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

MATCHING-ADJUSTED INDIRECT COMPARISON OF RIX-FP VERSUS MARSTACIMAB FOR THE PROPHYLAXIS OF SEVERE HEMOPHILIA B (EHA 2026) - "Results were consistent in the secondary analysis. This suggests rIX-FP may be associated with reduced bleeding compared to marstacimab in patients with severe hem B."

Hematological Disorders • Hemophilia • Hemophilia B • Rare Diseases

Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and B. (PubMed, MAbs) - "TFPI-106 elicited enhanced hemostasis in hemophilic plasma, shortened clotting time, and enhanced thrombin generation velocity. Marstacimab is now approved for patients with hemophilia A or B with or without inhibitors."

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

U.S. FDA Approves Pfizer’s HYMPAVZI for the Treatment of Two Additional Hemophilia A or B Patient Populations with Significant Medical Need (Pfizer Press Release) - "Results from the Phase 3 BASIS trial (NCT03938792) supported the approval of HYMPAVZI in adults and adolescents 12 years and older with hemophilia A or B with inhibitors...Interim results from the Phase 3 BASIS KIDS trial (NCT05611801) supported the approval of HYMPAVZI in children ages 6 to 17 years with hemophilia A or B with or without inhibitors."

FDA approval • Hemophilia A • Hemophilia B

Effect of marstacimab in young people living with severe hemophilia A or moderate to severe hemophilia B without inhibitors: A plain language summary from the BASIS study. (PubMed, Ther Adv Hematol) - "What do the results mean? This study showed that a once weekly injection of marstacimab could help reduce bleeding episodes in young people living with hemophilia A or B, without inhibitors."

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

Dental Surgery without Clotting Factor Replacement Therapy during Continued Prophylaxis with Marstacimab, an Inhibitory TFPI Monoclonal Antibody. (PubMed, Hamostaseologie) - No abstract available

Journal