Hemophilia A With Inhibitors

Rare Diseases
4
Pipeline Programs
4
Companies
3
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
1
1
2
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

4 companies ranked by most advanced pipeline stage

Swedish Orphan Biovitrum
1 program
1
rFVIIIFcPhase 4
Sanofi
SanofiPARIS, France
1 program
1
rFVIIIFcPhase 41 trial
Active Trials
NCT03093480Completed16Est. Feb 2021
LFB
LFBFrance - Paris
1 program
1
Coagulation FVIIaPhase 31 trial
Active Trials
NCT02448680Completed25Est. Aug 2017
CSL Behring
CSL BehringIL - Bradley
1 program
1
CSL689Phase 2/31 trial
Active Trials
NCT02484638Terminated25Est. Mar 2018

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
SanofirFVIIIFc
LFBCoagulation FVIIa
CSL BehringCSL689

Clinical Trials (3)

Total enrollment: 66 patients across 3 trials

NCT03093480SanofirFVIIIFc

A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)

Start: Dec 2017Est. completion: Feb 202116 patients
Phase 4Completed
NCT02448680LFBCoagulation FVIIa

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa

Start: Dec 2015Est. completion: Aug 201725 patients
Phase 3Completed
NCT02484638CSL BehringCSL689

Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors

Start: Jul 2015Est. completion: Mar 201825 patients
Phase 2/3Terminated

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
4 companies competing in this space

Related Indications

Cystic Fibrosis(244)Hemophilia A(139)Sickle Cell Disease(64)Fabry Disease(55)Duchenne Muscular Dystrophy(53)Hemophilia B(47)Spinal Muscular Atrophy(34)Phenylketonuria(32)Gaucher Disease(29)Severe Hemophilia A(26)