Hemophilia A With Inhibitor

Rare Diseases
6
Pipeline Programs
8
Companies
11
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Gene Therapy
1100%
+ 7 programs with unclassified modality

Competitive Landscape

7 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
1 program
1
FeibaPhase 44 trials
Active Trials
NCT04563520Recruiting5Est. Mar 2027
NCT04205175Active Not Recruiting20Est. Oct 2022
NCT02764489Completed45Est. Dec 2021
+1 more trials
LFB
LFBFrance - Paris
1 program
1
coagulation factor VIIa [recombinant]-jncwPhase 41 trial
Active Trials
NCT04647227Recruiting55Est. Mar 2027
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
1
1
MarzAAPhase 31 trial
Coagulation Factor VIIa variantPhase 21 trial
Active Trials
NCT03407651CompletedEst. Apr 2019
NCT04489537TerminatedEst. Dec 2021
BioMarin Pharmaceutical
1 program
1
Valoctocogene roxaparvovecPhase 1/2Gene Therapy1 trial
Active Trials
NCT04684940Active Not Recruiting10Est. Apr 2029
CT
1 program
1
TQG203Phase 11 trial
Active Trials
NCT04768699UnknownEst. Dec 2021
Genentech
GenentechCA - Oceanside
1 program
ATHN 7: Hemophilia Natural History StudyN/A1 trial
Active Trials
NCT03619863Completed395Est. Mar 2024
Swedish Orphan Biovitrum
1 program
rFVIIIFcN/A1 trial
Active Trials
NCT03951103CompletedEst. Sep 2022

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
LFBcoagulation factor VIIa [recombinant]-jncw
TakedaFeiba
TakedaFeiba
Catalyst PharmaceuticalsMarzAA
TakedaFeiba
TakedaFeiba
Catalyst PharmaceuticalsCoagulation Factor VIIa variant
BioMarin PharmaceuticalValoctocogene roxaparvovec
Chia Tai TianQing Pharmaceutical GroupTQG203
Swedish Orphan BiovitrumrFVIIIFc
GenentechATHN 7: Hemophilia Natural History Study

Clinical Trials (11)

Total enrollment: 582 patients across 11 trials

NCT04647227LFBcoagulation factor VIIa [recombinant]-jncw

SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

Start: Jun 2021Est. completion: Mar 202755 patients
Phase 4Recruiting
NCT04205175TakedaFeiba

A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab

Start: Jul 2020Est. completion: Oct 202220 patients
Phase 4Active Not Recruiting
NCT04563520TakedaFeiba

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

Start: Mar 2026Est. completion: Mar 20275 patients
Phase 3Recruiting
NCT04489537Catalyst PharmaceuticalsMarzAA

Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B

Start: May 2021Est. completion: Dec 2021
Phase 3Terminated
NCT02764489TakedaFeiba

FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)

Start: Feb 2019Est. completion: Dec 202145 patients
Phase 3Completed
NCT00851721TakedaFeiba

Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

Start: Mar 2009Est. completion: Oct 201252 patients
Phase 3Completed
NCT03407651Catalyst PharmaceuticalsCoagulation Factor VIIa variant

Study of Coagulation Factor VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia A and B

Start: Dec 2017Est. completion: Apr 2019
Phase 2Completed
NCT04684940BioMarin PharmaceuticalValoctocogene roxaparvovec

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

Start: Dec 2020Est. completion: Apr 202910 patients
Phase 1/2Active Not Recruiting
NCT04768699Chia Tai TianQing Pharmaceutical GroupTQG203

Study of Recombinant Human Coagulation Factor VIIa for Injection (FⅦa) in Patients With Hemophilia.

Start: Dec 2020Est. completion: Dec 2021
Phase 1Unknown
NCT03951103Swedish Orphan BiovitrumrFVIIIFc

rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A

Start: Nov 2018Est. completion: Sep 2022
N/ACompleted
NCT03619863GenentechATHN 7: Hemophilia Natural History Study

ATHN 7: Hemophilia Natural History Study

Start: Oct 2018Est. completion: Mar 2024395 patients
N/ACompleted

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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
2 actively recruiting trials targeting 582 patients
8 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)