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Disease

Hemophilia A

Late-stage therapeutic developmentEmerging research
MeSHHemophilia A
1
Publications
23
Clinical trials
11
Associated genes
4
Related proteins
2017
Latest publication
Latest activity
beta

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

ATLAS-PEDS: An Open-label, Multinational Study of Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Clinical trial2026-09-28Status changed to Completed · ClinicalTrials.gov

A Phase III Clinical Study on the Efficacy and Safety of STSP-0601 for Injection in Patients With Hemophilia Associated With Inhibitors, With a Multicenter, Open Evaluation Approach

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Clinical trial2026-07-23Status changed to Withdrawn · ClinicalTrials.gov

Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants With Moderately Severe to Severe Hemophilia A(FVIII:C≤1%)

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.
  • 3 industry developments reported.
Upcoming Milestones
Clinical Milestones11View all 11
Results posted 2
Results expected 5
Recently completed 2
Other updates 2
+3 more in the activity timeline below
Industry & Market3View
Activity timeline14

Clinical trials

12 sponsors · 2 new · 2 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 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

Phase 4 · Active, not recruiting · Hoffmann-La Roche

A Single-arm, Open-label, Multicenter Phase III Clinical Study Evaluating the Efficacy, Safety, and Immunogenicity of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) On-demand Treatment in Patients With Severe Hemophilia A (Adults and Adolescents) .

Phase 3 · Active, not recruiting · Hangzhou Gensciences Biopharmaceutical Co., Ltd.
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
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

Multicenter Study to Evaluate the Efficacy, Safety, Immunogenicity, and Pharmacokinetics of Recombinant Human Coagulation Factor Ⅷ-Fc Fusion Protein (FRSW117) for Injection in Patients With Severe Hemophilia A (Adults and Adolescents)

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

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 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 paper

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

Associated genes

11 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

F80.91F90.76F100.59F70.55TFPI0.51FUNDC20.33H2AB10.14LMAN10.12SERPINA100.03F8A10.03HSFX10.00

Disease biology

4 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Coagulation factor VIIIvia F80.91Coagulation factor IXvia F90.76Coagulation factor Xvia F100.59Tissue factor pathway inhibitorvia TFPI0.51

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.