Hemophilia A

Rare Diseases
89
Pipeline Programs
23
Companies
50
Clinical Trials
5 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
15
6
7
4
42
15
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
754%
Monoclonal Antibody
646%
+ 117 programs with unclassified modality

Competitive Landscape

23 companies ranked by most advanced pipeline stage

Takeda
TakedaTOKYO, Japan
23 programs
1
1
2
6
3
ADVATEPhase 41 trial
Antihemophilic factor, recombinant, manufactured protein-freePhase 41 trial
Recombinant Protein-Free Factor VIIIPhase 41 trial
AdynovatePhase 31 trial
Antihemophilic FactorPhase 35 trials
+18 more programs
Active Trials
NCT02078427Completed951Est. Jan 2024
NCT03824522Completed341Est. Jan 2024
NCT03169972Completed135Est. Sep 2023
+35 more trials
Bayer
BayerLEVERKUSEN, Germany
20 programs
4
1
1
1
1
2
KogenatePhase 41 trial
rFVIII-FSPhase 41 trial
BAY94-9027Phase 31 trial
BAY94-9027Phase 2/31 trial
rFVIII-FS/pegylated liposomesPhase 21 trial
+15 more programs
Active Trials
NCT02396862Completed272Est. Mar 2018
NCT02263066Completed181Est. Jul 2015
NCT02971930Completed30Est. Dec 2017
+29 more trials
Pfizer
PfizerNEW YORK, NY
17 programs
2
6
6
Intravenous infusions of XynthaPhase 41 trial
Laboratory TestsPhase 43 trials
Moroctocog alfaPhase 41 trial
Moroctocog-alfaPhase 41 trial
ReFactoPhase 41 trial
+12 more programs
Active Trials
NCT03248141Terminated11Est. Mar 2018
NCT00037544CompletedEst. Aug 2004
NCT00038935CompletedEst. Feb 2003
+20 more trials
Swedish Orphan Biovitrum
9 programs
1
2
2
Efanesoctocog alfaPhase 41 trial
Recombinant coagulation factorPhase 41 trial
BIIB031Phase 3
rFVIIIFcPhase 3
Efanesoctocog alfaPhase 11 trial
+4 more programs
Active Trials
NCT04293523Active Not RecruitingEst. Apr 2026
NCT04731701CompletedEst. Jul 2022
NCT02976753CompletedEst. Nov 2021
+3 more trials
Octapharma
OctapharmaAustria - Vienna
5 programs
1
1
NuwiqPhase 41 trial
Human-cl rhFVIIIPhase 21 trial
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of N/A1 trial
NuwiqN/A1 trial
Wilate or NuwiqN/A1 trial
Active Trials
NCT02962765Completed80Est. Aug 2020
NCT04023019Recruiting120Est. Jun 2029
NCT03344003Terminated14Est. Nov 2020
+2 more trials
Grifols
GrifolsNEW YORK, NY
1 program
1
FVIIIPhase 41 trial
Active Trials
NCT04621916UnknownEst. Apr 2025
Sanofi
SanofiPARIS, France
11 programs
2
1
5
BIIB031Phase 31 trial
efanesoctocog alfaPhase 31 trial
efanesoctocog alfaPhase 31 trial
rFVIIIFcPhase 31 trial
rFVIIIFcPhase 31 trial
+6 more programs
Active Trials
NCT05911763Active Not Recruiting200Est. Sep 2030
NCT06684314Recruiting100Est. Aug 2031
NCT06530030Terminated3Est. Sep 2025
+7 more trials
CSL Behring
CSL BehringIL - Bradley
8 programs
1
1
3
BiostatePhase 31 trial
BiostatePhase 31 trial
rVIII-SingleChainPhase 31 trial
rVIII-SingleChainPhase 2/31 trial
Biostate® [SP]Phase 21 trial
+3 more programs
Active Trials
NCT00936845Completed22Est. Jan 2010
NCT01034904Completed48Est. Nov 2013
NCT01053715Completed78Est. Jan 2015
+5 more trials
BioMarin Pharmaceutical
6 programs
1
4
Valoctocogene RoxaparvovecPhase 3Gene Therapy1 trial
Valoctocogene roxaparvovecPhase 3Gene Therapy1 trial
valoctocogene roxaparvovecPhase 3Gene Therapy1 trial
valoctocogene roxaparvovecPhase 3Gene Therapy1 trial
Valoctocogene RoxaparvovecPhase 1/2Gene Therapy1 trial
+1 more programs
Active Trials
NCT05580692Terminated321Est. Jul 2024
NCT03520712Terminated3Est. Aug 2024
NCT03392974Completed1Est. Jun 2023
+3 more trials
CP
Chugai PharmaJapan - Tokyo
6 programs
6
EmicizumabPhase 3Monoclonal Antibody1 trial
EmicizumabPhase 3Monoclonal Antibody1 trial
EmicizumabPhase 3Monoclonal Antibody1 trial
EmicizumabPhase 3Monoclonal Antibody1 trial
NXT007Phase 31 trial
+1 more programs
Active Trials
NCT02622321Completed113Est. Dec 2020
NCT02847637Completed152Est. May 2022
NCT02795767Completed88Est. Nov 2020
+3 more trials
CT
5 programs
1
4
Recombinant Factor VIIIPhase 31 trial
Recombinant Human Coagulation FVIIIPhase 31 trial
Recombinant human activated coagulation factor VII for injectionPhase 31 trial
Recombinant human activated coagulation factor VIII for injectionPhase 31 trial
XynthaPhase 11 trial
Active Trials
NCT04060836UnknownEst. Mar 2020
NCT02930317UnknownEst. Dec 2017
NCT04061109UnknownEst. Mar 2020
+2 more trials
Roche
RocheSTAVANGER NORWAY, Norway
3 programs
1
1
NXT007Phase 3
SPK-8011QQPhase 1/21 trial
Bypassing AgentsN/A1 trial
Active Trials
NCT02476942Completed221Est. Mar 2017
NCT07226206Recruiting5Est. Jul 2031
Centessa Pharmaceuticals
3 programs
1
1
1
SerpinPCPhase 31 trial
SerpinPCPhase 21 trial
SerpinPCPhase 1/21 trial
Active Trials
NCT04073498CompletedEst. Sep 2024
NCT05789524TerminatedEst. Feb 2025
NCT06568302TerminatedEst. Jan 2025
GC Biopharma
GC BiopharmaKorea - Yongin
3 programs
3
GreenGenePhase 31 trial
GreenGene™ FPhase 31 trial
GreenGene™ F and an approved recombinant Factor VIII productPhase 31 trial
Active Trials
NCT01568580CompletedEst. Sep 2006
NCT02027779UnknownEst. Feb 2016
NCT01619046UnknownEst. Sep 2015
PTC Therapeutics
1 program
1
AtalurenPhase 21 trial
Active Trials
NCT00947193Terminated13Est. Aug 2011
Ipsen
IpsenChina - Tianjin
1 program
1
recombinant porcine coagulation factor VIIIPhase 21 trial
Active Trials
NCT00245245Completed12Est. Jun 2007
Eli Lilly and Company
Eli Lilly and CompanyINDIANAPOLIS, IN
1 program
1
SIG-001Phase 1/21 trial
Active Trials
NCT04541628Terminated3Est. Jan 2023
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
2
MarzAAPhase 11 trial
PF-05280602Phase 11 trial
Active Trials
NCT04072237CompletedEst. Jun 2020
NCT01439971CompletedEst. Oct 2015
Expression Therapeutics
1 program
1
Gene therapyPhase 1Gene Therapy1 trial
Active Trials
NCT04418414Not Yet Recruiting7Est. Aug 2039
Opko Health
Opko HealthMIAMI, FL
1 program
1
MOD-5014Phase 11 trial
Active Trials
NCT02418793CompletedEst. Sep 2018
Genentech
GenentechCA - Oceanside
1 program
EmicizumabN/AMonoclonal Antibody1 trial
Active Trials
NCT03154437Approved For Marketing
Providence Therapeutics
1 program
Ideal Body Weight FirstN/A1 trial
Active Trials
NCT03286153UnknownEst. Dec 2018
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
1 program
Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet FunctionN/A1 trial
Active Trials
NCT02225483Completed42Est. Dec 2016

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
OctapharmaNuwiq
Swedish Orphan BiovitrumEfanesoctocog alfa
BayerDamoctocog
TakedaADVATE
GrifolsFVIII
PfizerMoroctocog-alfa
BayerDamoctocog alfa pegol
Swedish Orphan BiovitrumRecombinant coagulation factor
PfizerIntravenous infusions of Xyntha
TakedaOctocog alfa
BayerRecombinant Factor VIII
PfizerLaboratory Tests
PfizerLaboratory Tests
TakedaOctocog alfa
PfizerLaboratory Tests

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 2,823 patients across 50 trials

Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery

Start: Apr 2026Est. completion: Feb 202728 patients
Phase 4Recruiting

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

Start: May 2025Est. completion: Dec 2028
Phase 4Recruiting

Prophylaxis Regimen for Hemophilia A Patients

Start: Jul 2022Est. completion: Sep 202421 patients
Phase 4Completed

A Study of ADVATE in People With Hemophilia A in India

Start: Jan 2022Est. completion: Feb 202350 patients
Phase 4Completed

Preventing Inhibitor Recurrence Indefinitely

Start: Sep 2020Est. completion: Apr 2025
Phase 4Unknown
NCT04396639PfizerMoroctocog-alfa

Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients

Start: Jan 2020Est. completion: Sep 202050 patients
Phase 4Completed
NCT04085458BayerDamoctocog alfa pegol

Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)

Start: Sep 2019Est. completion: Aug 202232 patients
Phase 4Completed
NCT03103542Swedish Orphan BiovitrumRecombinant coagulation factor

Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies

Start: Aug 2017Est. completion: Aug 2020
Phase 4Completed
NCT02492984PfizerIntravenous infusions of Xyntha

PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A

Start: Apr 2015Est. completion: Aug 201685 patients
Phase 4Completed
NCT02170402TakedaOctocog alfa

China ADVATE PTP Study

Start: Jun 2014Est. completion: May 201682 patients
Phase 4Completed
NCT01810666BayerRecombinant Factor VIII

Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

Start: Mar 2013Est. completion: Jan 201430 patients
Phase 4Completed
NCT00950170PfizerLaboratory Tests

Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting

Start: Feb 2010Est. completion: Nov 201623 patients
Phase 4Completed
NCT00914459PfizerLaboratory Tests

Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients

Start: Dec 2009Est. completion: Apr 201637 patients
Phase 4Completed
NCT00916032TakedaOctocog alfa

Pharmacokinetic Study of ADVATE 3000 IU in Previously Treated Patients With Severe Hemophilia A

Start: Jun 2009Est. completion: Apr 201029 patients
Phase 4Completed
NCT00884390PfizerLaboratory Tests

Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings

Start: May 2009Est. completion: Mar 2013208 patients
Phase 4Terminated
NCT00765726PfizerMoroctocog alfa

Study Evaluating The Safety Of Xyntha In Usual Care Settings

Start: Feb 2009Est. completion: Aug 201112 patients
Phase 4Terminated
NCT00666406TakedaAntihemophilic Factor

Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A

Start: Mar 2008Est. completion: Feb 20099 patients
Phase 4Completed

Russian Kogenate Pediatric Study

Start: Jun 2007Est. completion: Sep 200932 patients
Phase 4Completed
NCT00357656TakedaRecombinant Protein-Free Factor VIII

Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery

Start: May 2006Est. completion: Dec 201585 patients
Phase 4Completed

Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A

Start: May 2006Est. completion: Oct 20061 patients
Phase 4Terminated
NCT00289536TakedaAntihemophilic factor, recombinant, manufactured protein-free

Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

Start: Feb 2006Est. completion: Apr 200738 patients
Phase 4Completed
NCT00243386TakedaAntihemophilic factor, recombinant, manufactured protein-free

Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

Start: Jan 2006Est. completion: Jun 201082 patients
Phase 4Completed
NCT00151385Pfizersingle blood draw

Study Evaluating Inhibitor Specificity in Hemophilia A

Start: Nov 2005Est. completion: Oct 20070
Phase 4Withdrawn

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

Start: Feb 2003Est. completion: Jul 200715 patients
Phase 4Completed

Study Evaluating ReFacto AF in Severe Hemophilia A

Est. completion: Aug 2004
Phase 3Completed

Study Evaluating BDDRFVIII and ReFacto AF in Hemophilia A

Est. completion: Feb 2003
Phase 3Completed

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

Start: May 2026Est. completion: Sep 2031126 patients
Phase 3Not Yet Recruiting

A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

Start: Apr 2026Est. completion: Jan 2032360 patients
Phase 3Not Yet Recruiting

The Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial

Start: Jul 2024Est. completion: Jan 2025
Phase 3Terminated
NCT06297655Chia Tai TianQing Pharmaceutical GroupRecombinant human activated coagulation factor VIII for injection

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

Start: Apr 2024Est. completion: Apr 2025
Phase 3Not Yet Recruiting
NCT06224907BioMarin PharmaceuticalValoctocogene roxaparvovec

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Start: Dec 2023Est. completion: Mar 20296 patients
Phase 3Active Not Recruiting

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

Start: Mar 2023Est. completion: Sep 202437 patients
Phase 3Completed
NCT05568719PfizerTesting of hepatic AAV Vector integration

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Start: Dec 2022Est. completion: Feb 2040173 patients
Phase 3Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Start: Dec 2022Est. completion: Sep 2028100 patients
Phase 3Recruiting
NCT05487976Chia Tai TianQing Pharmaceutical GroupRecombinant human activated coagulation factor VII for injection

Clinical Study of Recombinant Human Activated Coagulation Factor VII for Injection in Patients With Hemophilia With Inhibitor

Start: Apr 2022Est. completion: Apr 2023
Phase 3Unknown
NCT05147662BayerDamoctocog alfa pegol

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

Start: Mar 2022Est. completion: Jun 202536 patients
Phase 3Completed
NCT04644575Sanofiefanesoctocog alfa

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

Start: Feb 2021Est. completion: Jan 2027261 patients
Phase 3Active Not Recruiting
NCT04759131Sanofiefanesoctocog alfa

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

Start: Feb 2021Est. completion: Jan 202374 patients
Phase 3Completed
NCT04323098BioMarin Pharmaceuticalvaloctocogene roxaparvovec

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Start: Dec 2020Est. completion: May 202522 patients
Phase 3Completed

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Start: Aug 2020Est. completion: Oct 202877 patients
Phase 3Active Not Recruiting

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Start: Mar 2020Est. completion: Apr 2025189 patients
Phase 3Completed
NCT04061109Chia Tai TianQing Pharmaceutical GroupRecombinant Human Coagulation FVIII

Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.

Start: May 2019Est. completion: Mar 2020
Phase 3Unknown
NCT03392974BioMarin PharmaceuticalValoctocogene Roxaparvovec

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Start: Mar 2018Est. completion: Jun 20231 patients
Phase 3Completed
NCT03370913BioMarin Pharmaceuticalvaloctocogene roxaparvovec

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Start: Dec 2017Est. completion: Nov 2024144 patients
Phase 3Completed
NCT02973087TakedaAntihemophilic Factor

rVWF IN PROPHYLAXIS

Start: Nov 2017Est. completion: Jul 202029 patients
Phase 3Completed
NCT02932618TakedaAntihemophilic Factor

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)

Start: Nov 2017Est. completion: Mar 202631 patients
Phase 3Recruiting

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A

Start: Jan 2017Est. completion: Jun 202248 patients
Phase 3Completed
NCT02895945TakedaAntihemophilic Factor

BAX 802 in CHA With Inhibitors

Start: Dec 2016Est. completion: Jan 20218 patients
Phase 3Terminated

A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors

Start: Sep 2016Est. completion: May 2022152 patients
Phase 3Completed

Evaluate Efficacy and Safety of Recombinant Factor VIII (rFVIII)Treatment of Severe or Moderately Severe Hemophilia A

Start: Aug 2016Est. completion: Dec 2017
Phase 3Unknown

Related Jobs in Rare Diseases

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

42 late-stage (Phase 3) programs — potential near-term approvals
5 actively recruiting trials targeting 2,823 patients
23 companies competing in this space