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Disease

Hemophilia A

Late-stage therapeutic developmentEmerging research
MeSHHemophilia A
1
Publications
23
Clinical trials
2017
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

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.
Upcoming Milestones
Clinical Milestones12View all 12
Results posted 3
Results expected 5
Recently completed 2
Other updates 2
+4 more in the activity timeline below
Activity timeline12

Clinical trials

14 sponsors · 2 new · 3 completed in the last 12 months (net +2)

The current development programme across all trial phases.

Clinical programme
23
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

A 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

Phase 3 · Recruiting · Hoffmann-La Roche

A 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 Inhibitors

Phase 3 · Active, not recruiting · Hoffmann-La Roche

A 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

Phase 3 · Recruiting · Gritgen Therapeutics Co., Ltd.

AN 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

Phase 3 · Recruiting · Pfizer

A 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)

Phase 3 · Recruiting · Hangzhou Gensciences Biopharmaceutical Co., Ltd.

An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors

Phase 4 · Recruiting · Sanofi
Recruiting

A 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

Phase 3 · Recruiting · Hoffmann-La Roche

A 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

Phase 3 · Recruiting · Gritgen Therapeutics Co., Ltd.

AN 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

Phase 3 · Recruiting · Pfizer

A 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)

Phase 3 · Recruiting · Hangzhou Gensciences Biopharmaceutical Co., Ltd.

An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors

Phase 4 · Recruiting · Sanofi
Recently completed

A 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 Months

Phase 3 · Completed · Swedish Orphan Biovitrum

A Randomized, Two-armed, Double-blind, Single-dose, Crossover, Two-sequence, Bioequivalence Clinical Trial to Compare PK Parameters and Safety of rFVIII-Fc (AryoGen Pharmed Co.) Versus Elocta® in PTPs With Severe Hemophilia A

Phase 3 · Completed · AryoGen Pharmed Co.

A 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 Patients

Phase 3 · Completed · BioMarin Pharmaceutical

A Phase 2/3, Open-Label, Non-controlled Study to Evaluate the Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (rpFVIII, TAK-672), in the Treatment of Serious Bleeding Episode in Japanese Subjects With Acquired Hemophilia A (AHA)

Phase 2/3 · Completed · Takeda

A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Patients

Phase 3 · Completed · Hoffmann-La Roche

A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Phase 3 · Completed · Hoffmann-La Roche

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Most influential

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

The New England journal of medicine · 2017 · 524 cites
Recent publications

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

The New England journal of medicine · 2017 · 524 cites
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

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
MeSHHemophilia A
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.