VivoSeq
Rare Disease

Hemophilia a

74

Pipeline Programs↗

39

Active Trials↗

26

Companies Involved↗

Hemophilia A is a moderately active landscape with 74 programs across 26 companies. The most active players are rEVO Biologics, Eli Lilly, Staidson (Beijing) Biopharmaceuticals Co., Ltd. 9 therapies are approved with 23 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

68/100

Pipeline Velocity

27/100

Mech. Concentration

13/100

Completion Rate

84/100

Attrition Rate

10/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

R

rEVO Biologics

1 program

Phase 1Eli Lilly logo

Eli Lilly

1 program

Phase 1S

Staidson (Beijing) Biopharmaceuticals Co., Ltd

1 program

Phase 2P

PTC Therapeutics

1 program

Phase 2A

ASC Therapeutics

1 program

Phase 2CSL Behring logo

CSL Behring

3 programs

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

88%

Small molecule

52 of 59 classified programs — the dominant modality here.

12%ALL OTHERSMonoclonal antibody58%RNA therapy12%Gene therapy12%

Small molecule leads; the ring shows all 59 classified programs, each share of the total. Modality classified for 96% of 94 pipeline programs.

Analysis

Trial Status

134

clinical trials

Completed70 · 52%
Recruiting24 · 18%
Terminated12 · 9%
Unknown status11 · 8%
Active, not recruiting11 · 8%
Not yet recruiting4 · 3%
Other2 · 1%

Clinical Trials

Trial Activity

134 trials

ASC618 Gene Therapy in Hemophilia A Patients
Recruiting·AASC Therapeutics·Phase 1/2·NCT04676048·12 enrolled
Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)
Recruiting·RRunhui WU·Phase 4·NCT06864975·110 enrolled
Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products
Recruiting·CSL Behring logoCSL Behring·Phase 3·NCT06738485·60 enrolled
Treatment of Hemophilia A Patients With FVIII Inhibitors
Recruiting·EEmory University·N/A·NCT04023019·120 enrolled
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
Recruiting·GGritgen Therapeutics Co., Ltd.·Phase 3·NCT06833983·50 enrolled
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 1·NCT06238908·6 enrolled
Hemlibra in Mild Hemophilia A
Recruiting·IIndiana Hemophilia &Thrombosis Center, Inc.·Phase 4·NCT04567511·20 enrolled
ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 4·NCT05888870·50 enrolled
Efficacy and Safety Evaluation Study of SCT800 in Previously Untreated Hemophilia A Patients.
Recruiting·SSinocelltech Ltd.·Phase 3·NCT07437404·36 enrolled
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Recruiting·Pfizer logoPfizer·Phase 3·NCT05611801·100 enrolled
Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
Recruiting·Sanofi logoSanofi·Phase 4·NCT06941870·35 enrolled
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
Recruiting·Sanofi logoSanofi·N/A·NCT06684314·100 enrolled
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
Recruiting·Roche logoRoche·Phase 3·NCT07416604·360 enrolled
A Multicenter, Randomized, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of NXT007 Prophylaxis Versus Factor VIII Prophylaxis in People With Hemophilia A Without Inhibitors
Recruiting·Roche logoRoche·Phase 3·NCT07416526·126 enrolled
A Phase I/II Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Recruiting·Roche logoRoche·Preclinical·NCT05987449·60 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)