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Hemophilia A

Rare Diseases
64
Pipeline Programs
30
Companies
50
Clinical Trials
7 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
12
6
2
3
26
15
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Gene Therapy
754%
Monoclonal Antibody
646%
+ 188 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

37 companies ranked by most advanced pipeline stage

Bayer
BayerLEVERKUSEN, Germany
35 programs
7
1
1
1
3
5
KogenatePhase 41 trial
No DrugPhase 41 trial
Recombinant Factor VIIIPhase 41 trial
Recombinant Factor VIIIPhase 41 trial
rFVIII-FSPhase 41 trial
+30 more programs
Active Trials
NCT02396862Completed272Est. Mar 2018
NCT02263066Completed181Est. Jul 2015
NCT02971930Completed30Est. Dec 2017
+37 more trials
Pfizer
PfizerNEW YORK, NY
29 programs
2
12
9
Intravenous infusions of XynthaPhase 41 trial
Laboratory TestsPhase 41 trial
Moroctocog alfaPhase 41 trial
Moroctocog alfaPhase 41 trial
Moroctocog-alfaPhase 41 trial
+24 more programs
Active Trials
NCT03248141Terminated11Est. Mar 2018
NCT00895037Completed101Est. Oct 2016
NCT02718677Withdrawn0Est. Jan 2022
+28 more trials
Angeles Therapeutics
1
FVIIIPhase 41 trial
FVIII immuno-assayN/A1 trial
Personalized Prediction of Tolerance and Immunogenicity in HemophiliaN/A1 trial
Thromboelastography/Thromboelastometry-Guided Treatment RegimenN/A1 trial
Active Trials
NCT00839202Completed7Est. Feb 2009
NCT01626105Unknown55Est. Jun 2014
NCT02582060Completed18Est. Dec 2017
+1 more trials
CSL Seqirus
8 programs
1
1
3
BiostatePhase 3
BiostatePhase 3
rVIII-SingleChainPhase 3
rVIII-SingleChainPhase 2/3
Biostate® [SP]Phase 2
+3 more programs
Baxter
BaxterCosta Rica - Cartago
7 programs
1
1
Recombinant antihemophilic factor, plasma/albumin-free methodPhase 3
Antihemophilic FactorPhase 1
FVIII concentrateN/A
MyPKFiTN/A
Personalized Prediction of Tolerance and Immunogenicity in HemophiliaN/A
+2 more programs
Genentech
5 programs
2
1
NXT007Phase 31 trial
NXT007Phase 1/21 trial
SPK-8011QQPhase 1/21 trial
Bypassing AgentsN/A1 trial
EmicizumabN/AMonoclonal Antibody1 trial
Active Trials
NCT02476942Completed221Est. Mar 2017
NCT03154437Approved For Marketing
NCT05987449Recruiting60Est. Jun 2030
+2 more trials
SPARK Biopharma
SPARK BiopharmaKorea - Seoul
3 programs
1
1
SPK-8011Phase 31 trial
SPK-8011Phase 1/21 trial
SPK-8011N/A1 trial
Active Trials
NCT03432520Active Not Recruiting29Est. Dec 2032
NCT03003533Completed25Est. Dec 2023
NCT06297486Withdrawn0Est. Sep 2035
Gensciences
GensciencesChina - Nantong
3 programs
1
2
Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionPhase 31 trial
Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for InjectionPhase 31 trial
ADVATEPhase 11 trial
Active Trials
NCT05251090Completed13Est. May 2022
NCT04456387Completed119Est. Sep 2021
NCT04845399Completed101Est. Jun 2021
Biopharma Group
Biopharma GroupUK - Winchester
2 programs
2
GreenGene™ FPhase 31 trial
GreenGene™ F and an approved recombinant Factor VIII productPhase 31 trial
Active Trials
NCT02027779Unknown150Est. Feb 2016
NCT01619046Unknown124Est. Sep 2015
Gritgen Therapeutics
1
GS1191-0445 injectionPhase 32 trials
Active Trials
NCT06833983Recruiting50Est. Nov 2030
NCT07548411Active Not Recruiting7Est. Dec 2029
Biomed
BiomedAustralia - Sydney
1 program
1
Single dose intravenous injection of BBM-H803Phase 2/31 trial
Active Trials
NCT06111638Recruiting55Est. May 2031
Biocad
1 program
1
ANB-010, dose 1Phase 1/21 trial
Active Trials
NCT06185335Active Not Recruiting50Est. Jun 2033
Prevail Therapeutics
1
SIG-001Phase 1/21 trial
Active Trials
NCT04541628Terminated3Est. Jan 2023
TiumBio
TiumBioKorea - Seongnam
1 program
1
TU7710Phase 11 trial
Active Trials
NCT06025552Completed40Est. Aug 2024
Takeda
TakedaTOKYO, Japan
23 programs
ADVATEN/A1 trial
ADYNOVATEN/A1 trial
ADYNOVATEN/A1 trial
ADYNOVI/ADYNOVATEN/A1 trial
Factor VIIIN/A1 trial
+18 more programs
Active Trials
NCT02078427Completed951Est. Jan 2024
NCT03169972Completed135Est. Sep 2023
NCT03824522Completed341Est. Jan 2024
+35 more trials
Sanofi
SanofiPARIS, France
11 programs
Efanesoctocog Alfa BIVV001N/A1 trial
Efanesoctocog alfaN/A1 trial
efanesoctocog alfaN/A1 trial
ALN-AT3SCPHASE_13 trials
Efanesoctocog alfaPHASE_11 trial
+6 more programs
Active Trials
NCT05911763Active Not Recruiting200Est. Sep 2030
NCT06684314Recruiting100Est. Aug 2031
NCT06530030Terminated3Est. Sep 2025
+7 more trials
Swedish Orphan Biovitrum
9 programs
ELOCTAN/A1 trial
Hemophilia Joint Health ScoreN/A1 trial
efmoroctocog alfaN/A1 trial
rFVIIIFcN/A1 trial
Efanesoctocog alfaPHASE_11 trial
+4 more programs
Active Trials
NCT04293523Active Not Recruiting427Est. Jul 2026
NCT04731701Completed180Est. Jul 2022
NCT02976753Completed361Est. Nov 2021
+6 more trials
CSL Behring
CSL BehringIL - Bradley
8 programs
Females With Severe or Moderate Hemophilia A or B: A Multi-Center StudyN/A1 trial
Health Related Quality of Life of Youth and Young Adults With Hemophilia AN/A1 trial
Quality of Life Study of Helixate NexGenN/A1 trial
Biostate® [SP]PHASE_2
rVIII-SingleChainPHASE_2_31 trial
+3 more programs
Active Trials
NCT00936845Completed22Est. Jan 2010
NCT01034904Completed48Est. Nov 2013
NCT01053715Completed78Est. Jan 2015
+3 more trials
BioMarin Pharmaceutical
6 programs
Biospecimen CollectionN/A1 trial
Valoctocogene RoxaparvovecPHASE_1_2Gene Therapy1 trial
Valoctocogene RoxaparvovecPHASE_3Gene Therapy1 trial
Valoctocogene roxaparvovecPHASE_3Gene Therapy1 trial
valoctocogene roxaparvovecPHASE_3Gene Therapy1 trial
+1 more programs
Active Trials
NCT05580692Terminated321Est. Jul 2024
NCT03520712Terminated3Est. Aug 2024
NCT03392974Completed1Est. Jun 2023
+3 more trials
Chugai Pharma
Chugai PharmaJapan - Tokyo
6 programs
EmicizumabPHASE_3Monoclonal Antibody
EmicizumabPHASE_3Monoclonal Antibody
EmicizumabPHASE_3Monoclonal Antibody
EmicizumabPHASE_3Monoclonal Antibody
NXT007PHASE_3
+1 more programs
Octapharma
OctapharmaAustria - Vienna
5 programs
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of N/A1 trial
NuwiqN/A1 trial
Wilate or NuwiqN/A1 trial
Human-cl rhFVIIIPHASE_21 trial
NuwiqPHASE_41 trial
Active Trials
NCT02962765Completed80Est. Aug 2020
NCT04023019Recruiting120Est. Jun 2029
NCT03344003Terminated14Est. Nov 2020
+2 more trials
Chia Tai TianQing Pharmaceutical Group
5 programs
XynthaPHASE_11 trial
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
Active Trials
NCT04060836Unknown24Est. Mar 2020
NCT02930317Unknown60Est. Dec 2017
NCT04061109Unknown64Est. Mar 2020
+2 more trials
Baxter International
4 programs
FVIII concentrateN/A1 trial
MyPKFiTN/A1 trial
Personalized Prediction of Tolerance and Immunogenicity in HemophiliaN/A
Platelet Function in Patients With Hemophilia AN/A1 trial
Active Trials
NCT01652027Completed25Est. Mar 2020
NCT02634424Terminated12Est. May 2016
NCT02093065Completed36Est. Feb 2020
Roche
RocheSTAVANGER NORWAY, Norway
3 programs
Bypassing AgentsN/A
SPK-8011QQPHASE_1_2
NXT007PHASE_3
Centessa Pharmaceuticals
3 programs
SerpinPCPHASE_1_21 trial
SerpinPCPHASE_21 trial
SerpinPCPHASE_31 trial
Active Trials
NCT04073498Completed39Est. Sep 2024
NCT05789524Terminated60Est. Feb 2025
NCT06568302Terminated15Est. Jan 2025
Parexel
ParexelMA - Boston
3 programs
Biostate® [SP]PHASE_21 trial
BiostatePHASE_31 trial
Recombinant Human Coagulation FVIIIPHASE_31 trial
Active Trials
NCT00879541Completed81Est. Oct 2010
NCT01229007Completed35Est. Jul 2014
NCT03815318Completed73Est. Jan 2020
GC Biopharma
3 programs
GreenGenePHASE_31 trial
GreenGene™ FPHASE_3
GreenGene™ F and an approved recombinant Factor VIII productPHASE_3
Active Trials
NCT01568580Completed71Est. Sep 2006
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
2 programs
MarzAAPHASE_11 trial
PF-05280602PHASE_11 trial
Active Trials
NCT04072237CompletedEst. Jun 2020
NCT01439971CompletedEst. Oct 2015
Providence Therapeutics
1 program
Ideal Body Weight FirstN/A1 trial
Active Trials
NCT03286153Unknown20Est. Dec 2018
Oregon Therapeutics
Ideal Body Weight FirstN/A

+7 more companies

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
BayerRecombinant Factor VIII
Angeles TherapeuticsFVIII
PfizerMoroctocog-alfa
BayerBAY94-9027
Swedish Orphan BiovitrumRecombinant coagulation factor
PfizerIntravenous infusions of Xyntha
TakedaOctocog alfa
BayerRecombinant Factor VIII
PfizerLaboratory Tests
PfizerMoroctocog alfa
TakedaOctocog alfa

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 3,346 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 2028250 patients
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
NCT04565236BayerRecombinant Factor VIII

A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A

Start: Sep 2020Est. completion: Mar 202445 patients
Phase 4Completed

Preventing Inhibitor Recurrence Indefinitely

Start: Sep 2020Est. completion: Apr 202552 patients
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

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 202016 patients
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
NCT00914459PfizerMoroctocog alfa

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

Joint Status in Subjects With Severe Hemophilia A in Relation to Different Treatment Regimens

Start: Jun 2009Est. completion: Dec 2010156 patients
Phase 4Completed
NCT00884390Pfizermoroctocog alfa

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 Comparing Blood Levels of ReFacto and Advante in Hemophilia A

Start: Apr 2005Est. completion: Nov 20050
Phase 4Withdrawn

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

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

Study Evaluating BDDRFVIII and ReFacto AF in Hemophilia A

Est. completion: Feb 2003
Phase 3Completed

Study Evaluating ReFacto AF in Severe Hemophilia A

Est. completion: Aug 2004
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 3Recruiting
NCT06833983Gritgen TherapeuticsGS1191-0445 injection

To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A

Start: Mar 2025Est. completion: Nov 203050 patients
Phase 3Recruiting

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

Start: Jul 2024Est. completion: Jan 202515 patients
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 202560 patients
Phase 3Unknown

Study of a Gene Therapy Treatment for Hemophilia A

Start: Mar 2024Est. completion: Sep 20350
Phase 3Withdrawn
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 202350 patients
Phase 3Unknown

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

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

Start: Nov 2021Est. completion: Jul 2030245 patients
Phase 3Recruiting
NCT04845399GensciencesRecombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A

Start: Apr 2021Est. completion: Jun 2021101 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
NCT04456387GensciencesRecombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A.

Start: Oct 2020Est. completion: Sep 2021119 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 202064 patients
Phase 3Unknown
NCT03815318ParexelRecombinant Human Coagulation FVIII

Evaluation of SCT800 in Prophylaxis Therapy on Previous Treated Patients With Severe Hemophilia A

Start: Jan 2019Est. completion: Jan 202073 patients
Phase 3Completed

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

26 late-stage (Phase 3) programs, potential near-term approvals
7 actively recruiting trials targeting 3,346 patients
30 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.