Summary
Efanesoctocog Alfa has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Haemophilia A / B would be drug repurposing rather than first-in-human development. This does not mean it is approved for Haemophilia A / B. ClinicalTrials.gov lists 8 registered trials linking Efanesoctocog Alfa to Haemophilia A / B: 4 are currently recruiting; 1 is active or not yet recruiting; 3 have completed. The most advanced is Phase 4 (NCT06940830). The largest enrolment is 261 participants (NCT04644575). Registration activity spans 2019 to 2025. No published literature item is linked to Efanesoctocog Alfa and Haemophilia A / B in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Hemophilia ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: No spontaneous remission; lifelong clotting factor deficiency; joint damage progressive with each bleed without treatment; with prophylaxis, normal lifespan achievable.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 50.0 (trials 30.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 4 recruiting, 1 active / not yet recruiting, 3 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | F8 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT06941870 | Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy 2025 | recruiting | Phase 4 | 35 |
| NCT06940830 | Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa 2025 | recruiting | Phase 4 | 250 |
| NCT04644575 | Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A 2021 | active, not recruiting | Phase 3 | 261 |
| NCT04161495 | A Phase 3 Open-label Interventional Study of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, Efanesoctocog Alfa (BIVV001), in Patients With Severe Hemophilia A 2019 | completed | Phase 3 | 159 |
| NCT04759131 | Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A 2021 | completed | Phase 3 | 74 |
| NCT06716814 | Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa 2025 | recruiting | Phase 3 | 104 |
| NCT06579144 | Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A 2025 | completed | Phase 1 | 24 |
| NCT06684314 | A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan 2024 | recruiting | Not applicable | 100 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target F8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Efanesoctocog Alfa approved for Haemophilia A / B?
Efanesoctocog Alfa has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Haemophilia A / B would be drug repurposing rather than first-in-human development. This does not mean it is approved for Haemophilia A / B. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Efanesoctocog Alfa in clinical trials for Haemophilia A / B?
ClinicalTrials.gov lists 8 registered trials linking Efanesoctocog Alfa to Haemophilia A / B: 4 are currently recruiting; 1 is active or not yet recruiting; 3 have completed. The most advanced is Phase 4 (NCT06940830). The largest enrolment is 261 participants (NCT04644575). Registration activity spans 2019 to 2025.
What does the evidence show for Efanesoctocog Alfa in Haemophilia A / B?
Efanesoctocog Alfa has 3 completed registered trials for Haemophilia A / B but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target F8. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.