Severe Haemophilia A
4
Pipeline Programs
3
Companies
4
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
3
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
1100%
+ 3 programs with unclassified modality
Competitive Landscape
3 companies ranked by most advanced pipeline stage
OctapharmaAustria - Vienna
2 programs2
Human cl rhFVIIIPhase 31 trial
Human-cl rhFVIIIPhase 31 trial
Active Trials
BioMarin PharmaceuticalNOVATO, CA
1 program1
valoctocogene roxaparvovecPhase 1/2Gene Therapy1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Swedish Orphan BiovitrumEfanesoctocog alfa
OctapharmaHuman cl rhFVIII
OctapharmaHuman-cl rhFVIII
BioMarin Pharmaceuticalvaloctocogene roxaparvovec
Clinical Trials (4)
Total enrollment: 139 patients across 4 trials
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
Start: Mar 2025Est. completion: Mar 2027
Phase 3Recruiting
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Start: May 2015Est. completion: Sep 201858 patients
Phase 3Completed
Assess the Safety and Efficacy of Individually Tailored Prophylaxis With Human-cl rhFVIII in Patients With Severe Haemophilia A
Start: Aug 2013Est. completion: Jan 201566 patients
Phase 3Completed
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
Start: Sep 2015Est. completion: Feb 202415 patients
Phase 1/2Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
3 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 139 patients
3 companies competing in this space