Severe Hemophilia A

Rare Diseases
18
Pipeline Programs
11
Companies
22
Clinical Trials
2 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
3
1
0
0
10
4
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
2100%
+ 23 programs with unclassified modality

On Market (1)

Approved therapies currently available

Roche
HEMLIBRAApproved
emicizumab
Roche
injection2017

Competitive Landscape

10 companies ranked by most advanced pipeline stage

Octapharma
OctapharmaAustria - Vienna
8 programs
1
6
1
NuwiqPhase 41 trial
Human cl rhFVIIIPhase 31 trial
Human-cl rhFVIIIPhase 31 trial
Human-cl rhFVIIIPhase 31 trial
WilatePhase 31 trial
+3 more programs
Active Trials
NCT04046848Terminated36Est. Feb 2022
NCT01712438Completed110Est. Dec 2019
NCT01341912Completed3Est. Aug 2012
+5 more trials
Grifols
GrifolsNEW YORK, NY
3 programs
1
Alphanate SD/HTPhase 41 trial
FVIII ConcentratesN/A
VWF/FVIII concentratesN/A
Active Trials
NCT00323856CompletedEst. Dec 2018
Roche
RocheSTAVANGER NORWAY, Norway
1 program
1
HEMLIBRA(Emicizumab)Phase 4Monoclonal Antibody5 trials
Active Trials
NCT05181618Active Not Recruiting136Est. May 2027
NCT04188639Completed47Est. Jan 2023
NCT04158648Completed73Est. Dec 2025
+2 more trials
Sanofi
SanofiPARIS, France
4 programs
2
2
Factor VIIIPhase 31 trial
rFVIIIFcPhase 31 trial
rFVIIIFcPhase 11 trial
rFVIIIFcPhase 11 trial
Active Trials
NCT02083965Completed19Est. May 2015
NCT01027377Completed16Est. May 2011
NCT01181128Completed165Est. Aug 2012
+1 more trials
Swedish Orphan Biovitrum
2 programs
2
Factor VIIIPhase 3
rFVIIIFcPhase 3
Pfizer
PfizerNEW YORK, NY
1 program
1
MARSTACIMABPhase 1Monoclonal Antibody1 trial
Active Trials
NCT06703606Recruiting15Est. May 2027
Biotest Pharmaceuticals
Biotest PharmaceuticalsGermany - Dreieich
2 programs
FVIII ConcentratesN/A
VWF/FVIII concentratesN/A
CSL Behring
CSL BehringIL - Bradley
2 programs
FVIII ConcentratesN/A
VWF/FVIII concentratesN/A
City Therapeutics
City TherapeuticsMA - Cambridge
1 program
FVIII ConcentratesN/A2 trials
Active Trials
NCT01051076Completed3Est. Oct 2020
NCT01051544Withdrawn0Est. Jun 2020
Bayer
BayerLEVERKUSEN, Germany
1 program
Octocog alfaN/A1 trial
Active Trials
NCT07446010Not Yet Recruiting33Est. Dec 2026

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
OctapharmaNuwiq
RocheEmicizumab
GrifolsAlphanate SD/HT
RocheEmicizumab
RocheEmicizumab
OctapharmaWilate
OctapharmaWilate
SanofirFVIIIFc
OctapharmaHuman-cl rhFVIII
OctapharmaHuman cl rhFVIII
OctapharmaHuman-cl rhFVIII
SanofiFactor VIII
Octapharmarecombinant Factor VIII
RocheEmicizumab
OctapharmaOCTA101

Showing 15 of 22 trials with date data

Clinical Trials (22)

Total enrollment: 957 patients across 22 trials

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

Start: Nov 2023Est. completion: Aug 202628 patients
Phase 4Recruiting

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis

Start: Jun 2022Est. completion: May 2027136 patients
Phase 4Active Not Recruiting
NCT00323856GrifolsAlphanate SD/HT

Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A

Start: Apr 2003Est. completion: Dec 2018
Phase 4Completed

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

Start: Feb 2020Est. completion: Dec 202573 patients
Phase 3Completed

Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants

Start: Apr 2018Est. completion: Aug 202585 patients
Phase 3Completed

Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A

Start: Nov 2017Est. completion: Nov 201811 patients
Phase 3Completed

Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A

Start: Dec 2016Est. completion: Mar 201857 patients
Phase 3Completed

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale

Start: Aug 2015Est. completion: Jun 201724 patients
Phase 3Completed
NCT01992549OctapharmaHuman-cl rhFVIII

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Start: Apr 2014Est. completion: Dec 201848 patients
Phase 3Completed
NCT01712438OctapharmaHuman cl rhFVIII

Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated Patients

Start: Feb 2013Est. completion: Dec 2019110 patients
Phase 3Completed
NCT01341912OctapharmaHuman-cl rhFVIII

Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

Start: Jun 2011Est. completion: Aug 20123 patients
Phase 3Completed

Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A

Start: Nov 2010Est. completion: Aug 2012165 patients
Phase 3Completed
NCT01125813Octapharmarecombinant Factor VIII

Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A

Start: Jun 2010Est. completion: Jan 201232 patients
Phase 3Completed

Emicizumab in Acquired Hemophilia A

Start: Mar 2021Est. completion: Jan 202347 patients
Phase 2Completed

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A

Start: Jul 2019Est. completion: Feb 202236 patients
Phase 1/2Terminated

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.

Start: May 2025Est. completion: May 202715 patients
Phase 1Recruiting

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

Start: Mar 2018Est. completion: Sep 201816 patients
Phase 1Completed

Pharmacokinetics of rFVIIIFc at Two Vial Strengths

Start: Mar 2014Est. completion: May 201519 patients
Phase 1Completed

Study of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects With Severe Hemophilia A

Start: Dec 2009Est. completion: May 201116 patients
Phase 1Completed
NCT07446010BayerOctocog alfa

Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India

Start: Mar 2026Est. completion: Dec 202633 patients
N/ANot Yet Recruiting

Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)

Start: Nov 2009Est. completion: Oct 20203 patients
N/ACompleted

Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive

Start: Sep 2009Est. completion: Jun 20200
N/AWithdrawn

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

10 late-stage (Phase 3) programs — potential near-term approvals
2 actively recruiting trials targeting 957 patients
11 companies competing in this space