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Hemophilia B

Rare Diseases
34
Pipeline Programs
19
Companies
50
Clinical Trials
6 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
4
6
2
3
16
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
2100%
+ 76 programs with unclassified modality

Rare Diseases is a $21.9B market dominated by a single blockbuster (ELIQUIS), with moderate growth and high consolidation among top-tier pharma players.

$21.9B marketMature→ Stable30 products15 companies

Key Trends

  • Market concentration: One product (ELIQUIS) represents 83% of market spending
  • Complement inhibitors and enzyme replacement therapies gaining momentum
  • Active pipeline with 1,790 trials, predominantly Phase 2-3 stage

Career Verdict

Yes, if you seek stability and commercial opportunity with major pharma; proceed cautiously if you want pipeline upside or innovation-driven roles.

AI-generated market analysis based on FDA, CMS, ClinicalTrials.gov, and hiring data

Market Leaders

#1ELIQUISStable
$18.3B
Bristol Myers Squibb·LAUNCH15.0yr
#2XIFAXANDeclining
$1.1B
Bausch Health·LOE_APPROACHING
#3BRILINTAStable
$692M
AstraZeneca·PEAK10.2yr
#4TYVASO DPIStable
$437M
#5STRENSIQStable
$259M

Drug Class Breakdown

FXa Inhibitors (Anticoagulants)
$18.3B(83%)

Mature, patent-protected until 2041

Unknown Mechanism
$1.4B(6%)

Mixed—includes LOE-approaching products

P2Y12 Receptor Antagonists
$692M(3%)

Peak sales, LOE 2036

Prostaglandins/Vasodilators
$437M(2%)

Stable, long patent protection

Complement Inhibitors
$408M(2%)

Emerging class, no LOE data available

Enzyme Replacement Therapies
$40M(0%)

Niche market, specialized patient populations

Career Outlook

Stable

The rare disease market is stable but mature, with job growth concentrated in commercial and medical affairs rather than R&D innovation. Consolidation among top pharma (AstraZeneca, BMS, Amgen) limits opportunities for mid-tier companies, but specialist biotech firms show hiring momentum. Patent cliff risk for ELIQUIS (2041) will create restructuring opportunities and potential job flux in downstream years.

Breaking In

Target commercial or quality assurance roles at AstraZeneca, Capricor, or Amgen to build market knowledge; rare disease expertise is portable and valued across therapeutic areas.

For Experienced Professionals

Seek regulatory or medical affairs leadership roles where salary ceiling is highest ($243K-$257K); consider biotech firms like Insmed or Krystal for upside if pipeline approvals accelerate.

In-Demand Skills

Market access and HCP engagement (medical affairs)Regulatory and compliance expertisePatient stratification and genetic testing knowledgeCommercial analytics and lifecycle management

Best For

Medical Science Liaison (high salary, growing demand)Commercial Manager/Product Manager (39% of jobs)Regulatory Affairs Specialist (premium salary $257K)Health Economics & Outcomes Research (unmet need in data)

Hiring Landscape

$114K-$257K

Rare disease hiring is concentrated in commercial roles (75 jobs at $215K avg) and medical affairs (16 jobs at $243K avg), reflecting a mature market's focus on market access and HCP engagement. Top hiring companies include AstraZeneca (38 jobs) and emerging biotech players like Capricor Therapeutics (30 jobs). Department salaries range from $114K-$257K, with regulatory affairs commanding premium compensation.

195
Open Roles
5
Companies Hiring
5
Departments

Top Hiring Companies

38Growing
18Stable
17Growing
14Stable

By Department

Commercial(39%)
$215K
Medical Affairs(8%)
$243K
Research & Development(10%)
Regulatory Affairs(4%)
$257K
Quality Assurance(4%)
$168K

Commercial and regulatory roles offer strong salaries and growth; R&D hiring is minimal (10%), reflecting a consolidation and maturity phase in the market.

Competitive Landscape

19 companies ranked by most advanced pipeline stage

Pfizer
PfizerNEW YORK, NY
15 programs
1
7
2
BenefixPhase 41 trial
Factor IX recoveryPhase 41 trial
BenefixPhase 31 trial
Nonacog alfaPhase 33 trials
PF-06838435/ fidanacogene elaparvovecPhase 3Gene Therapy1 trial
+10 more programs
Active Trials
NCT00484185Completed183Est. Jun 2012
NCT00037557Completed20Est. Nov 2007
NCT00581126Completed14Est. Jul 2004
+16 more trials
Prothya Biosolutions
Prothya BiosolutionsBelgium - Brussels
1 program
1
human coagulation Factor IXPhase 41 trial
Active Trials
NCT00139828Completed12Est. Feb 2007
CSL Seqirus
CSL SeqirusUK - Maidenhead
15 programs
1
2
1
1
6
AAV5-hFIXco-PaduaPhase 3
CSL222Phase 3
CSL222Phase 3
Recombinant fusion protein linking coagulation factor IX with albuminPhase 3
rIX-FPPhase 3
+10 more programs
Medexus Pharmaceuticals
3 programs
1
2
APVO101Phase 31 trial
IB1001Phase 31 trial
IB1001Phase 2/31 trial
Active Trials
NCT00768287Completed77Est. Dec 2016
NCT03855280Completed21Est. Jul 2022
NCT01271868Terminated9Est. Dec 2016
Biocad
BiocadRussia - St. Petersburg
2 programs
1
1
ANB-002Phase 31 trial
ANB-002, dose 1Phase 1/21 trial
Active Trials
NCT06120582Recruiting28Est. Nov 2029
NCT06700096Recruiting24Est. Mar 2032
Biomed
BiomedAustralia - Sydney
1 program
1
Single dose intravenous injection of BBM-H901Phase 2/31 trial
Active Trials
NCT05203679Active Not Recruiting32Est. Jun 2028
Spur Therapeutics
Spur TherapeuticsUK - Stevenage
2 programs
2
FLT180aPhase 1/21 trial
verbrinacogene setparvovecPhase 1/2Gene Therapy1 trial
Active Trials
NCT03641703Terminated10Est. May 2023
NCT05164471Terminated6Est. May 2023
Be Biopharma
Be BiopharmaMA - Cambridge
1 program
1
BE-101Phase 1/21 trial
Active Trials
NCT06611436Recruiting24Est. Jul 2027
SPARK Biopharma
SPARK BiopharmaKorea - Seoul
2 programs
1
AAV8-hFIX19Phase 11 trial
LTFU for Gene Transfer Subjects With Hemophilia BN/A1 trial
Active Trials
NCT00515710Completed4Est. Dec 2017
NCT01620801Terminated4Est. Mar 2016
Sangamo Therapeutics
2 programs
1
SB-FIXPhase 11 trial
SB-318N/A1 trial
Active Trials
NCT04628871Active Not Recruiting13Est. Jan 2030
NCT02695160Terminated1Est. Apr 2021
ISU Abxis
ISU AbxisKorea - Seongnam
1 program
1
ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kgPhase 11 trial
Active Trials
NCT03186677Completed11Est. Feb 2019
CSL Behring
CSL BehringIL - Bradley
15 programs
AAV5-hFIXN/A1 trial
AAV5-hFIXco-PaduaN/A1 trial
HEMGENIXN/A1 trial
Joint Health StudyN/A1 trial
Recombinant Coagulation Factor IX Albumin Fusion ProteinPHASE_11 trial
+10 more programs
Active Trials
NCT05360706Active Not Recruiting9Est. May 2026
NCT05962398Enrolling By Invitation56Est. Mar 2035
NCT06008938Recruiting500Est. Aug 2043
+12 more trials
Takeda
TakedaTOKYO, Japan
7 programs
RIXUBISN/A1 trial
RIXUBISN/A2 trials
AskBio009PHASE_1_21 trial
SHP648PHASE_1_21 trial
BAX 326PHASE_34 trials
+2 more programs
Active Trials
NCT02922231Completed57Est. Apr 2019
NCT02937831Completed6Est. May 2022
NCT02190149Completed64Est. Mar 2016
+8 more trials
Swedish Orphan Biovitrum
4 programs
AlprolixN/A1 trial
rFIXFcPHASE_11 trial
rFIXFcPHASE_31 trial
rFIXFcPHASE_31 trial
Active Trials
NCT03901755Completed151Est. Mar 2024
NCT00716716Completed10Est. Oct 2009
NCT01440946Completed30Est. Nov 2014
+1 more trials
Regeneron
RegeneronTARRYTOWN, NY
2 programs
Non-InterventionalN/A1 trial
REGV131PHASE_1_21 trial
Active Trials
NCT05568459Active Not Recruiting11Est. May 2026
NCT06379789Recruiting130Est. Aug 2047
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
ISU304/CB2679d/Dalcinonacog alfa 75~150 IU/kgPHASE_1
Coagulation Factor IX variantPHASE_21 trial
Active Trials
NCT03995784CompletedEst. Apr 2020
Ultragenyx Pharmaceutical
1 program
DTX101PHASE_1_21 trial
Active Trials
NCT02618915Terminated6Est. Oct 2017
Intellia Therapeutics
1 program
REGV131PHASE_1_2
Grifols
GrifolsNEW YORK, NY
1 program
BeneFIXPHASE_21 trial
Active Trials
NCT03091751Completed13Est. Oct 2009

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
PfizerNonacog alfa
TakedaRIXUBIS: On-Demand
PfizerBenefix
TakedaFactor IX Concentrate
PfizerFactor IX recovery
Prothya Biosolutionshuman coagulation Factor IX
PfizerBeneFIX
PfizerrFIX
PfizerrFIX
CSL BehringCSL222
BiocadANB-002
CSL BehringRecombinant fusion protein linking coagulation factor IX with albumin
CSL BehringCSL222
Medexus PharmaceuticalsAPVO101
PfizerPF-06838435/ fidanacogene elaparvovec

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 1,550 patients across 50 trials

NCT04286412PfizerNonacog alfa

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

Start: Feb 2020Est. completion: Sep 202025 patients
Phase 4Completed
NCT03565237TakedaRIXUBIS: On-Demand

RIXUBIS PMS India (RIXUBIS PMS)

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

Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China

Start: Jan 2015Est. completion: Aug 201670 patients
Phase 4Completed
NCT01128881TakedaFactor IX Concentrate

IMMUNINE Pre-Treatment Study

Start: May 2010Est. completion: Aug 201257 patients
Phase 4Completed
NCT00749476PfizerFactor IX recovery

Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX

Start: Apr 2008Est. completion: Jan 20091 patients
Phase 4Completed
NCT00139828Prothya Biosolutionshuman coagulation Factor IX

Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX)

Start: May 2003Est. completion: Feb 200712 patients
Phase 4Completed

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

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Start: Nov 2024Est. completion: Mar 203224 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

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Start: Jan 2020Est. completion: Jul 202221 patients
Phase 3Completed
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

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B

Start: Nov 2014Est. completion: Aug 201933 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

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

Start: Jun 2012Est. completion: Nov 201430 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

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Start: Nov 2010Est. completion: Dec 20169 patients
Phase 3Terminated

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

Start: Jul 2010Est. completion: May 201286 patients
Phase 3Completed

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Start: Feb 2009Est. completion: Dec 200935 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

Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

Start: Sep 2002Est. completion: Nov 200720 patients
Phase 3Completed
NCT05203679BiomedSingle dose intravenous injection of BBM-H901

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Start: Dec 2021Est. completion: Jun 202832 patients
Phase 2/3Active Not Recruiting

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

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Start: Jan 2009Est. completion: Dec 201677 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

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

Start: Aug 2005Est. completion: Oct 200913 patients
Phase 2Completed

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Start: Dec 2024Est. completion: Jul 202724 patients
Phase 1/2Recruiting

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: Aug 2047130 patients
Phase 1/2Recruiting
NCT06120582BiocadANB-002, dose 1

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Start: May 2023Est. completion: Nov 202928 patients
Phase 1/2Recruiting
NCT05164471Spur Therapeuticsverbrinacogene setparvovec

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

Start: Dec 2021Est. completion: May 20236 patients
Phase 1/2Terminated

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

A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Start: Jul 2018Est. completion: May 202310 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
NCT03186677ISU AbxisISU304/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 201911 patients
Phase 1Completed

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

Start: Nov 2016Est. completion: Apr 20211 patients
Phase 1Terminated
NCT02213250Pfizerrecombinant factor IX

An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

Start: Mar 2015Est. completion: Apr 201512 patients
Phase 1Completed

Hemophilia B Gene Therapy With AAV8 Vector

Start: Oct 2012Est. completion: Mar 20164 patients
Phase 1Terminated

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

16 late-stage (Phase 3) programs, potential near-term approvals
6 actively recruiting trials targeting 1,550 patients
19 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.