Hemophilia

Rare Diseases
7
Pipeline Programs
10
Companies
23
Clinical Trials
3 recruiting
1
Approved Products

Pipeline by Development Stage

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

On Market (1)

Approved therapies currently available

Sanofi
QFITLIAApproved
fitusiran
Sanofi
Antithrombin-directed RNA Interaction [EPC]subcutaneous2025

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Sanofi
SanofiPARIS, France
1 program
1
1
QFITLIA(Fitusiran)Phase 35 trials
Active Trials
NCT07285460Recruiting85Est. Dec 2031
NCT06145373Recruiting20Est. Nov 2029
NCT05662319Active Not Recruiting91Est. Jan 2029
+2 more trials
Bayer
BayerLEVERKUSEN, Germany
5 programs
1
1
KogenatePhase 31 trial
BAY1093884Phase 11 trial
Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013N/A
Joint Outcome Study Continuation for Children With Severe Factor VIII DeficiencyN/A1 trial
SureSource Engage applicationN/A1 trial
Active Trials
NCT01000844Completed11Est. Oct 2017
NCT03168685Unknown20Est. May 2018
NCT03996486Withdrawn0Est. Jul 2020
+1 more trials
LFB
LFBFrance - Paris
1 program
1
Coagulation Factor VIIaPhase 31 trial
Active Trials
NCT02548143Completed12Est. Aug 2017
GC Biopharma
GC BiopharmaKorea - Yongin
2 programs
2
MG1113Phase 11 trial
MG1113Phase 11 trial
Active Trials
NCT05493631CompletedEst. Dec 2024
NCT03855696CompletedEst. Aug 2021
CSL Behring
CSL BehringIL - Bradley
3 programs
ATHN 8: Previously Untreated Patients (PUPs) Matter StudyN/A
Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013N/A1 trial
Fibrogammin PN/A1 trial
Active Trials
NCT02546622Completed310Est. Jun 2020
NCT00640289Completed72Est. Dec 2011
Takeda
TakedaTOKYO, Japan
2 programs
ATHN 8: Previously Untreated Patients (PUPs) Matter StudyN/A
Virtual-realityN/A1 trial
Active Trials
NCT05437211Completed24Est. Feb 2024
Pfizer
PfizerNEW YORK, NY
2 programs
Epidemiological Non interventionalN/A1 trial
blood specimenN/A1 trial
Active Trials
NCT00927992Completed11Est. Jun 2010
NCT02540187Completed164Est. Feb 2016
Octapharma
OctapharmaAustria - Vienna
1 program
ATHN 8: Previously Untreated Patients (PUPs) Matter StudyN/A1 trial
Active Trials
NCT03818529Completed237Est. Dec 2022
Merit Medical
Merit MedicalSOUTH JORDAN, UT
1 program
embolization with spherical microparticles embosphereN/A1 trial
Active Trials
NCT05629130RecruitingEst. Dec 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
SanofiFitusiran
LFBCoagulation Factor VIIa
BayerKogenate
SanofiFitusiran
GC BiopharmaMG1113
BayerBAY1093884
GC BiopharmaMG1113
TakedaVirtual-reality

Showing 15 of 23 trials with date data

Clinical Trials (23)

Total enrollment: 1,679 patients across 23 trials

NCT06145373SanofiFitusiran

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Start: Mar 2024Est. completion: Nov 202920 patients
Phase 4Recruiting
NCT07285460SanofiFitusiran

A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Start: Dec 2025Est. completion: Dec 203185 patients
Phase 3Recruiting
NCT05662319SanofiFitusiran

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

Start: Feb 2023Est. completion: Jan 202991 patients
Phase 3Active Not Recruiting
NCT03974113SanofiFitusiran

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Start: Jan 2020Est. completion: Dec 202632 patients
Phase 3Active Not Recruiting
NCT03754790SanofiFitusiran

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Start: Jan 2019Est. completion: Nov 2026281 patients
Phase 3Active Not Recruiting
NCT03549871SanofiFitusiran

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

Start: Jul 2018Est. completion: Mar 202280 patients
Phase 3Completed
NCT03417245SanofiFitusiran

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Start: Mar 2018Est. completion: Jul 2021120 patients
Phase 3Completed
NCT03417102SanofiFitusiran

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Start: Feb 2018Est. completion: Jun 202160 patients
Phase 3Completed
NCT02548143LFBCoagulation Factor VIIa

LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures

Start: Aug 2016Est. completion: Aug 201712 patients
Phase 3Completed
NCT00606060BayerKogenate

BAY14-2222 Continuous Infusion in Surgeries

Start: Jul 2004Est. completion: May 200515 patients
Phase 3Completed
NCT02554773SanofiFitusiran

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Start: Sep 2015Est. completion: Mar 202334 patients
Phase 1/2Completed
NCT05493631GC BiopharmaMG1113

A Phase 1b Study to Assess the Safety, Tolerability, PK and PD of MG1113 in Hemophilia Patient

Start: Aug 2022Est. completion: Dec 2024
Phase 1Completed
NCT03996486BayerBAY1093884

Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia

Start: Oct 2019Est. completion: Jul 20200
Phase 1Withdrawn
NCT03855696GC BiopharmaMG1113

A Study to Investigate the Safety, Tolerability, PK and PD of MG1113 in Healthy Subjects and Hemophilia Patients

Start: Jan 2019Est. completion: Aug 2021
Phase 1Completed
NCT05437211TakedaVirtual-reality

A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions

Start: Jun 2023Est. completion: Feb 202424 patients
N/ACompleted
NCT05629130Merit Medicalembolization with spherical microparticles embosphere

Embolization in Hereditary Coagulopathies

Start: Jan 2023Est. completion: Dec 2029
N/ARecruiting
NCT03818529OctapharmaATHN 8: Previously Untreated Patients (PUPs) Matter Study

ATHN 8: Previously Untreated Patients (PUPs) Matter Study

Start: Oct 2018Est. completion: Dec 2022237 patients
N/ACompleted
NCT03168685BayerSureSource Engage application

Hemophilia Mobile App Usability Pilot

Start: May 2017Est. completion: May 201820 patients
N/AUnknown
NCT02546622CSL BehringFactor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013

ATHN 2: Factor Switching Study

Start: Sep 2015Est. completion: Jun 2020310 patients
N/ACompleted
NCT02540187Pfizerblood specimen

Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients

Start: Feb 2012Est. completion: Feb 2016164 patients
N/ACompleted
NCT01000844BayerJoint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

Start: Nov 2009Est. completion: Oct 201711 patients
N/ACompleted
NCT00927992PfizerEpidemiological Non interventional

Study Evaluating Liver Transplantation in Haemophilia Patients in Spain

Start: Jul 2009Est. completion: Jun 201011 patients
N/ACompleted
NCT00640289CSL BehringFibrogammin P

Clinical Trial of Factor XIII (FXIII) Concentrate

Start: Jan 2000Est. completion: Dec 201172 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

3 late-stage (Phase 3) programs — potential near-term approvals
3 actively recruiting trials targeting 1,679 patients
10 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)