Hemophilia B

Rare Diseases
33
Pipeline Programs
9
Companies
46
Clinical Trials
4 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
3
7
5
1
13
4
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
1100%
+ 43 programs with unclassified modality

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Pfizer
PfizerNEW YORK, NY
11 programs
2
4
2
Nonacog alfaPhase 42 trials
Recombinant Factor IX CoagulationPhase 41 trial
PF-06838435/ fidanacogene elaparvovecPhase 3Gene Therapy1 trial
Recombinant Coagulation Factor IXPhase 31 trial
rFIXPhase 31 trial
+6 more programs
Active Trials
NCT00714415Completed80Est. Oct 2016
NCT00167973Completed218Est. Jun 2009
NCT00244114Completed20Est. Dec 2009
+9 more trials
Takeda
TakedaTOKYO, Japan
7 programs
2
1
2
Factor IX ConcentratePhase 41 trial
RIXUBIS: On-DemandPhase 41 trial
BAX 326Phase 34 trials
AskBio009Phase 1/21 trial
SHP648Phase 1/21 trial
+2 more programs
Active Trials
NCT02937831Completed6Est. May 2022
NCT02922231Completed57Est. Apr 2019
NCT01687608Active Not Recruiting30Est. Jan 2030
+7 more trials
CSL Behring
CSL BehringIL - Bradley
15 programs
1
2
1
1
6
AAV5-hFIXco-PaduaPhase 31 trial
CSL222Phase 31 trial
CSL222Phase 31 trial
Recombinant fusion protein linking coagulation factor IX with albuminPhase 31 trial
rIX-FPPhase 31 trial
+10 more programs
Active Trials
NCT05360706Active Not Recruiting9Est. May 2026
NCT05962398Enrolling By Invitation56Est. Mar 2035
NCT06008938Recruiting500Est. Aug 2043
+12 more trials
Swedish Orphan Biovitrum
4 programs
1
2
rFIXFcPhase 3
rFIXFcPhase 3
rFIXFcPhase 1
AlprolixN/A1 trial
Active Trials
NCT03901755CompletedEst. Mar 2024
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
1
1
Coagulation Factor IX variantPhase 21 trial
ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kgPhase 11 trial
Active Trials
NCT03186677CompletedEst. Feb 2019
NCT03995784CompletedEst. Apr 2020
Grifols
GrifolsNEW YORK, NY
1 program
1
BeneFIXPhase 21 trial
Active Trials
NCT03091751CompletedEst. Oct 2009
Regeneron
RegeneronTARRYTOWN, NY
2 programs
1
REGV131Phase 1/2
Non-InterventionalN/A3 trials
Active Trials
NCT05568459Active Not Recruiting11Est. Jan 2026
NCT05673967Completed62Est. Mar 2025
NCT05338892Completed595Est. Oct 2023
Ultragenyx Pharmaceutical
1 program
1
DTX101Phase 1/21 trial
Active Trials
NCT02618915Terminated6Est. Oct 2017
Intellia Therapeutics
1 program
1
REGV131Phase 1/21 trial
Active Trials
NCT06379789Recruiting130Est. Dec 2032

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
PfizerNonacog alfa
TakedaRIXUBIS: On-Demand
TakedaFactor IX Concentrate
PfizerRecombinant Factor IX Coagulation
PfizerrFIX
PfizerrFIX
CSL BehringCSL222
CSL BehringRecombinant fusion protein linking coagulation factor IX with albumin
CSL BehringCSL222
PfizerPF-06838435/ fidanacogene elaparvovec
CSL BehringAAV5-hFIXco-Padua
CSL BehringrIX-FP
CSL BehringrIX-FP
TakedaBAX 326
PfizerNonacog alfa

Showing 15 of 46 trials with date data

Clinical Trials (46)

Total enrollment: 2,714 patients across 46 trials

NCT04286412PfizerNonacog alfa

Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B

Start: Feb 2020Est. completion: Sep 2020
Phase 4Completed
NCT03565237TakedaRIXUBIS: On-Demand

RIXUBIS PMS India (RIXUBIS PMS)

Start: Dec 2018Est. completion: Aug 202125 patients
Phase 4Completed
NCT01128881TakedaFactor IX Concentrate

IMMUNINE Pre-Treatment Study

Start: May 2010Est. completion: Aug 201257 patients
Phase 4Completed
NCT00581126PfizerRecombinant Factor IX Coagulation

Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B

Start: Dec 2001Est. completion: Jul 200414 patients
Phase 4Completed

Study Evaluating rFIX; BeneFIX® in Hemophilia B

Est. completion: May 200523 patients
Phase 3Completed

Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B

Est. completion: Sep 2005
Phase 3Completed

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Start: Jul 2025Est. completion: Oct 203320 patients
Phase 3Recruiting
NCT06399289CSL BehringRecombinant fusion protein linking coagulation factor IX with albumin

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Start: Jul 2024Est. completion: Dec 202623 patients
Phase 3Active Not Recruiting

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

Start: Jan 2024Est. completion: Apr 203235 patients
Phase 3Recruiting
NCT03861273PfizerPF-06838435/ fidanacogene elaparvovec

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

Start: Jul 2019Est. completion: Feb 203151 patients
Phase 3Active Not Recruiting
NCT03569891CSL BehringAAV5-hFIXco-Padua

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Start: Jun 2018Est. completion: Mar 202567 patients
Phase 3Completed

A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

Start: Feb 2014Est. completion: Jun 202197 patients
Phase 3Completed

A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

Start: Jan 201327 patients
Phase 3Completed

BAX 326 Surgery Study in Hemophilia B Patients

Start: Dec 2011Est. completion: May 201430 patients
Phase 3Completed
NCT01335061PfizerNonacog alfa

Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

Start: Sep 2011Est. completion: Apr 201425 patients
Phase 3Completed

BAX 326 (rFIX) Continuation Study

Start: Apr 2011Est. completion: Jun 2017117 patients
Phase 3Completed

Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients

Start: Jul 2010Est. completion: May 201286 patients
Phase 3Completed
NCT00364182PfizerRecombinant Coagulation Factor IX

Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B

Start: May 2007Est. completion: Oct 201050 patients
Phase 3Completed

A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B

Start: Feb 201263 patients
Phase 2/3Completed

BAX 326 Pediatric Study

Start: Dec 2011Est. completion: May 201323 patients
Phase 2/3Completed
NCT03995784Catalyst PharmaceuticalsCoagulation Factor IX variant

Study of Next-Generation Recombinant Factor IX Variant in Adult Subjects With Hemophilia B

Start: Jun 2019Est. completion: Apr 2020
Phase 2Completed
NCT03489291CSL BehringAAV5-hFIXco-Padua

Dose Confirmation Trial of AAV5-hFIXco-Padua

Start: Jul 2018Est. completion: Sep 20233 patients
Phase 2Completed

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Start: Jun 2017Est. completion: Oct 202921 patients
Phase 2Active Not Recruiting

A Gene Therapy Study for Hemophilia B

Start: Nov 2015Est. completion: Apr 201915 patients
Phase 2Completed

Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B

Start: Aug 2005Est. completion: Oct 2009
Phase 2Completed

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Start: Sep 2024Est. completion: Dec 2032130 patients
Phase 1/2Recruiting

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

Start: May 2020Est. completion: May 20212 patients
Phase 1/2Terminated

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Start: Dec 2015Est. completion: Oct 20176 patients
Phase 1/2Terminated

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Start: Jun 2015Est. completion: Apr 202110 patients
Phase 1/2Completed

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Start: Feb 2013Est. completion: Jan 203030 patients
Phase 1/2Active Not Recruiting
NCT01361126CSL BehringRecombinant Coagulation Factor IX Albumin Fusion Protein

A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

Start: Jul 2011Est. completion: Jul 201217 patients
Phase 1/2Completed
NCT03186677Catalyst PharmaceuticalsISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kg

Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients

Start: Jun 2017Est. completion: Feb 2019
Phase 1Completed
NCT01233440CSL BehringRecombinant Coagulation Factor IX Albumin Fusion Protein

Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B

Start: Oct 2010Est. completion: Jul 201125 patients
Phase 1Completed
NCT05568459RegeneronNon-Interventional

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

Start: Jan 2024Est. completion: Jan 202611 patients
N/AActive Not Recruiting
NCT05962398CSL BehringAAV5-hFIXco-Padua

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

Start: Aug 2023Est. completion: Mar 203556 patients
N/AEnrolling By Invitation

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

Start: Jun 2023Est. completion: Aug 2043500 patients
N/ARecruiting
NCT05673967RegeneronNon-Interventional

Real-World Clinical Outcomes in Patients With Relapsed/Refractory Multiple Myeloma

Start: Jan 2023Est. completion: Mar 202562 patients
N/ACompleted
NCT05338892RegeneronNon-Interventional

Real-World Clinical Outcomes in Adult Patients Who Initiate Systemic Treatment for Relapsed/Refractory Diffuse Large B-Cell Lymphoma

Start: Jun 2022Est. completion: Oct 2023595 patients
N/ACompleted

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Start: Mar 2021Est. completion: May 20269 patients
N/AActive Not Recruiting

An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B

Start: Sep 2019Est. completion: Mar 2024
N/ACompleted
NCT03358836CSL BehringJoint Health Study

Joint Health Study

Start: Nov 2017Est. completion: Aug 20208 patients
N/ACompleted

Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea

Start: Jan 2017Est. completion: Apr 201957 patients
N/ACompleted

RIXUBIS Drug Use-Result Survey (Japan)

Start: Nov 2016Est. completion: May 20226 patients
N/ACompleted

Registry For Patients Treated With BeneFix In Usual Care Setting In Germany

Start: Jan 2008Est. completion: Oct 201680 patients
N/ACompleted

Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients

Start: Feb 2006Est. completion: Dec 200920 patients
N/ACompleted
NCT00167973PfizerProspective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use

Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use

Start: Jan 2002Est. completion: Jun 2009218 patients
N/ACompleted

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

13 late-stage (Phase 3) programs — potential near-term approvals
4 actively recruiting trials targeting 2,714 patients
9 companies competing in this space