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Disease
Hemophilia A
Late-stage therapeutic developmentEmerging research
1
Publications
23
Clinical trials
11
Associated genes
4
Related proteins
2017
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Clinical trial2026-08-12Status changed to Active, not recruiting · ClinicalTrials.gov
Topical and Local TXA in Facelifts - A Randomized Controlled Double Blinded Study
Clinical trial2026-07-23Status changed to Withdrawn · ClinicalTrials.gov
Clinical trial2026-05-18Results posted · ClinicalTrials.gov
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 5 clinical trials expected to report results, the earliest in Q4 2026.
- 1 industry development reported.
Upcoming Milestones
- Q4 2026A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia A
- Q1 2027A Single-Arm, Open-Label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, Immunogenicity and Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Therapy in Patients With Severe Hemophilia A (Adults and Adolescents)
- Q4 2027A Multicenter, Open-Label Phase IV Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes, in Participants Aged ≥13 and <70 Years With Severe or Moderate Hemophilia A Without FVIII Inhibitors on Emicizumab Prophylaxis
- Q1 2028A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia A
- Q3 2030AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY <1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS
Major developments
Major trial resultsHigh impact
Results posted2026-07-07
Upcoming trial readoutHigh impact
Results expected Q1 20282026-09-04
Clinical MilestonesView all 12Hide
Results posted
- 2026-07-07A Phase IIIb, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients From Birth to 12 Months of Age With Hemophilia A Without InhibitorsResults posted
- 2026-04-01A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A PatientsResults posted
- 2025-10-08A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A PatientsResults posted
Results expected
- 2026-09-04A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia AResults expected Q1 2028
- 2026-09-04A Single-Arm, Open-Label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, Immunogenicity and Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Therapy in Patients With Severe Hemophilia A (Adults and Adolescents)Results expected Q1 2027
- 2026-07-27A Multicenter, Open-Label Phase IV Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes, in Participants Aged ≥13 and <70 Years With Severe or Moderate Hemophilia A Without FVIII Inhibitors on Emicizumab ProphylaxisResults expected Q4 2027
- 2026-07-01AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY <1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORSResults expected Q3 2030
- 2025-12-09A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia AResults expected Q4 2026
Recently completed
- 2026-06-02A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 MonthsCompleted
- 2025-12-19A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII InhibitorsCompleted
Other updates
- 2027-07-01POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement TherapiesWithdrawn
- 2027-07-01Phase III INHIBIT Platform: Prevention Trial, Eloctate vs Emicizumab to Prevent Inhibitors; Eradication Trial: Eloctate Immune Tolerance (ITI) Plus Emicizumab vs vs Eloctate ITI Alone to Eradicate Inhibitors in Severe Hemophilia AWithdrawn
+4 more in the activity timeline below
Industry & MarketViewHide
- 2026-09-18CHMP backs Novo’s hemophilia A drug and seven other medicinesIndustry · Endpoints News
Activity timeline13
- 2027-07-01ClinicalPOCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement TherapiesWithdrawn
- 2027-07-01ClinicalPhase III INHIBIT Platform: Prevention Trial, Eloctate vs Emicizumab to Prevent Inhibitors; Eradication Trial: Eloctate Immune Tolerance (ITI) Plus Emicizumab vs vs Eloctate ITI Alone to Eradicate Inhibitors in Severe Hemophilia AWithdrawn
- 2026-09-18IndustryCHMP backs Novo’s hemophilia A drug and seven other medicinesIndustry · Endpoints News
- 2026-09-04ClinicalA Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Emicizumab Prophylaxis in People With Hemophilia AResults expected Q1 2028
- 2026-09-04ClinicalA Single-Arm, Open-Label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, Immunogenicity and Pharmacokinetics of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Therapy in Patients With Severe Hemophilia A (Adults and Adolescents)Results expected Q1 2027
- 2026-07-27ClinicalA Multicenter, Open-Label Phase IV Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes, in Participants Aged ≥13 and <70 Years With Severe or Moderate Hemophilia A Without FVIII Inhibitors on Emicizumab ProphylaxisResults expected Q4 2027
- 2026-07-07ClinicalA Phase IIIb, Multicenter, Open-Label, Single-Arm Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Patients From Birth to 12 Months of Age With Hemophilia A Without InhibitorsResults posted
- 2026-07-01ClinicalAN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY <1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORSResults expected Q3 2030
- 2026-06-02ClinicalA Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 MonthsCompleted
- 2026-04-01ClinicalA Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A PatientsResults posted
Clinical trials
The current development programme across all trial phases.
Clinical programme
23
6
18
6
Late-stage studies
Recruiting
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Most influential
Recent publications
Major themes3
- Dependovirus1
- Genetic Therapy1
- Genetic Vectors1
Leading journals1
- The New England journal of medicine1
Leading researchers8
- Laffan M1
- Lester W1
- Madan B1
- Pasi KJ1
- Perry D1
- Pierce GF1
- Rangarajan S1
- Vettermann C1
Affiliations (unnormalised)1
- From Hampshire Hospitals NHS Foundation Trust1
Associated genes
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Disease biology
Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.