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Hemophilia A With Inhibitor

Rare Diseases
3
Pipeline Programs
12
Companies
16
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
0
0
0
1
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
1100%
+ 12 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

11 companies ranked by most advanced pipeline stage

Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
1
FeibaPhase 41 trial
Active Trials
NCT04205175Active Not Recruiting20Est. Oct 2022
AryoGen Pharmed
AryoGen PharmedIran - Tehran
2 programs
1
eptacog alfa, activatedPhase 32 trials
Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSevenN/A1 trial
Active Trials
NCT03372993Completed200Est. Apr 2021
NCT04789954Completed14Est. Jul 2022
NCT03935334Completed48Est. Feb 2021
Gensciences
GensciencesChina - Nantong
1 program
1
SS109Phase 11 trial
Active Trials
NCT05651061Completed27Est. Aug 2023
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
Coagulation Factor VIIa variantPHASE_21 trial
MarzAAPHASE_31 trial
Active Trials
NCT03407651CompletedEst. Apr 2019
NCT04489537TerminatedEst. Dec 2021
Genentech
GenentechCA - Oceanside
1 program
ATHN 7: Hemophilia Natural History StudyN/A1 trial
Active Trials
NCT03619863Completed395Est. Mar 2024
JW Pharmaceutical
JW PharmaceuticalKorea - Seoul
1 program
Emicizumab subcutaneous injectionN/A1 trial
Active Trials
NCT04805801Unknown17Est. Feb 2025
Swedish Orphan Biovitrum
1 program
rFVIIIFcN/A1 trial
Active Trials
NCT03951103Completed44Est. Sep 2022
Chia Tai TianQing Pharmaceutical Group
1 program
TQG203PHASE_11 trial
Active Trials
NCT04768699Unknown24Est. Dec 2021
BioMarin Pharmaceutical
1 program
Valoctocogene roxaparvovecPHASE_1_2Gene Therapy1 trial
Active Trials
NCT04684940Active Not Recruiting10Est. Apr 2029
Takeda
TakedaTOKYO, Japan
1 program
FeibaPHASE_43 trials
Active Trials
NCT04563520Recruiting5Est. Mar 2027
NCT02764489Completed45Est. Dec 2021
NCT00851721Completed52Est. Oct 2012
LFB
LFBFrance - Paris
1 program
coagulation factor VIIa [recombinant]-jncwPHASE_41 trial
Active Trials
NCT04647227Recruiting55Est. Mar 2027

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
LFBcoagulation factor VIIa [recombinant]-jncw
Angeles TherapeuticsFeiba
TakedaFeiba
Catalyst PharmaceuticalsMarzAA
TakedaFeiba
AryoGen Pharmedeptacog alfa, activated
TakedaFeiba
Catalyst PharmaceuticalsCoagulation Factor VIIa variant
BioMarin PharmaceuticalValoctocogene roxaparvovec
GensciencesSS109
AryoGen Pharmedeptacog alfa, activated
Chia Tai TianQing Pharmaceutical GroupTQG203
JW PharmaceuticalEmicizumab subcutaneous injection
Swedish Orphan BiovitrumrFVIIIFc
GenentechATHN 7: Hemophilia Natural History Study

Showing 15 of 16 trials with date data

Clinical Trials (16)

Total enrollment: 956 patients across 16 trials

NCT04647227LFBcoagulation factor VIIa [recombinant]-jncw

SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

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

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

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

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

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

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

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

Start: Feb 2019Est. completion: Dec 202145 patients
Phase 3Completed
NCT03935334AryoGen Pharmedeptacog alfa, activated

Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors

Start: Jul 2018Est. completion: Feb 202148 patients
Phase 3Completed

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

A Phase I of SS109 in Hemophilia A or and B With Inhibitors

Start: Dec 2022Est. completion: Aug 202327 patients
Phase 1Completed
NCT04789954AryoGen Pharmedeptacog alfa, activated

Study on the Dose-response Relationship of Pharmacodynamic Parameters in Patients With Hemophilia With Inhibitors

Start: Dec 2020Est. completion: Jul 202214 patients
Phase 1Completed

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

Start: Dec 2020Est. completion: Dec 202124 patients
Phase 1Unknown
NCT04805801JW PharmaceuticalEmicizumab subcutaneous injection

The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors

Start: Aug 2019Est. completion: Feb 202517 patients
N/AUnknown

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

Start: Nov 2018Est. completion: Sep 202244 patients
N/ACompleted
NCT03619863GenentechATHN 7: Hemophilia Natural History Study

ATHN 7: Hemophilia Natural History Study

Start: Oct 2018Est. completion: Mar 2024395 patients
N/ACompleted
NCT03372993AryoGen PharmedProspective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven

Prospective, Non-interventional Study to Evaluate Immunogenicity of AryoSeven

Start: Jun 2016Est. completion: Apr 2021200 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

1 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 956 patients
12 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.