VivoSeq
Hematology

Severe Hemophilia a

48

Pipeline Programs↗

13

Active Trials↗

17

Companies Involved↗

Severe Hemophilia a is a moderately active landscape with 48 programs across 17 companies. The most active players are Capsida Biotherapeutics, Inc., Bioverativ, a Sanofi company, BioMarin Pharmaceutical. 6 therapies are approved with 22 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

60/100

Pipeline Velocity

19/100

Mech. Concentration

33/100

Completion Rate

84/100

Attrition Rate

12/100

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.

C

Capsida Biotherapeutics, Inc.

1 program

Phase 1B

Bioverativ, a Sanofi company

1 program

Phase 2BioMarin Pharmaceutical logo

BioMarin Pharmaceutical

1 program

Phase 2CSL Behring logo

CSL Behring

1 program

Phase 3Chia Tai Tianqing Pharmaceutical Group Co., Ltd. logo

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

1 program

Phase 3C

Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.

1 program

Phase 3

Pipeline

Programs by Phase

Preclinical4

Octocog Alfa

Sanofi

FVIII/VWF Concentrates

City of Hope Medical Center

FVIII Concentrates

City of Hope Medical Center

Prophylaxis With Emicizumab

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Phase 14

Gene Therapy

Capsida Biotherapeutics, Inc.

Marstacimab

Pfizer

BAY81-8973

Bayer

Rurioctocog Alfa Pegol

Sanofi

Phase 212

Rituximab

Roche

Valoctocogene Roxaparvovec

BioMarin Pharmaceutical

Human-Cl Rhfviii

Octapharma

SB-525 (PF-07055480)

Pfizer

Serpinpc

ApcinteX Ltd

Phase 322

Fitusiran

Genzyme, a Sanofi Company

Emicizumab

Roche

PF-06741086

Pfizer

Rfviii (BAY81-8973) on Demand

Bayer

Greengene™ F

Green Cross Corporation

Approved6

Rfviiifc

Bioverativ Therapeutics Inc.

Adynovate

Takeda

Kogenate (BAY14-2222)

Bayer

FEIBA- Activated Prothrombin Complexes

Sheba Medical Center

Eloctate

University of Texas Southwestern Medical Center

Analysis

Modality Breakdown

78%

Small molecule

25 of 32 classified programs — the dominant modality here.

22%ALL OTHERSMonoclonal antibody413%Gene therapy26%RNA therapy13%

Small molecule leads; the ring shows all 32 classified programs, each share of the total. Modality classified for 100% of 60 pipeline programs.

Analysis

Trial Status

68

clinical trials

Completed42 · 62%
Terminated7 · 10%
Unknown status5 · 7%
Not yet recruiting5 · 7%
Recruiting5 · 7%
Active, not recruiting3 · 4%
Other1 · 1%

Clinical Trials

Trial Activity

68 trials

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.
Recruiting·Pfizer logoPfizer·Phase 1·NCT06703606·15 enrolled
POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
Recruiting·UUniversity of Texas Southwestern Medical Center·Phase 4·NCT04690322·28 enrolled
Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A
Recruiting·—·Phase 2/3·NCT06155955·15 enrolled
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)
Recruiting·HHangzhou Gensciences Biopharmaceutical Co., Ltd.·Phase 3·NCT07684898·60 enrolled
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) .
Recruiting·HHangzhou Gensciences Biopharmaceutical Co., Ltd.·Phase 3·NCT07663903·60 enrolled
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Active, not recruiting·Pfizer logoPfizer·Phase 3·NCT04370054·77 enrolled
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT06224907·6 enrolled
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
Active, not recruiting·Roche logoRoche·Phase 3·NCT04431726·55 enrolled
Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India
Not yet recruiting·Bayer logoBayer·N/A·NCT07446010·33 enrolled
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
Not yet recruiting·—·Phase 3·NCT06136507·76 enrolled
A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A
Not yet recruiting·CChia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.·Phase 3·NCT06297655·60 enrolled
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Not yet recruiting·EExpression Therapeutics, LLC·Phase 1·NCT04418414·7 enrolled
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
Not yet recruiting·SSK Plasma Co., Ltd.·Phase 3·NCT06738901·55 enrolled
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
Completed·Bayer logoBayer·Phase 3·NCT01233258·80 enrolled
A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A
Completed·Takeda logoTakeda·Phase 3·NCT05707351·37 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)