Summary
Efmoroctocog 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linking Efmoroctocog Alfa to Haemophilia A / B: 1 is active or not yet recruiting; 3 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03103542). The largest enrolment is 427 participants (NCT04293523). Registration activity spans 2015 to 2023. The literature layer holds 2 publications for this pair: 2 clinical trial publications. Publication years run from 2024 to 2025. These are primary trial reports rather than syntheses, so results have not yet been pooled or graded independently.
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 | 43.5 (trials 19.5, literature 4.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 1 active / not yet recruiting, 3 completed, 4 other |
| Linked publications | 2 (2 clinical trial publications) |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT03103542 | Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies 2017 | completed | Phase 4 | 16 |
| NCT05856266 | An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa 2023 | terminated | Phase 4 | 10 |
| NCT02196207 | Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform 2020 | withdrawn | Phase 3 | — |
| NCT02586012 | Weight-based Dosing in Hemophilia A 2015 | terminated | Phase 2 | 30 |
| NCT04293523 | A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health 2020 | active, not recruiting | Not applicable | 427 |
| NCT02976753 | Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products 2016 | completed | Not applicable | 361 |
| NCT02546622 | ATHN 2: Factor Switching Study 2015 | completed | Not applicable | 310 |
| NCT02392156 | Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes 2015 | terminated | Not applicable | 3 |
Published literature
- Clinical trial publication A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia AOldenburg J, Hay C, Peyvandi F et al. · Hematology (Amsterdam, Netherlands) · 2025 · PMID 40616274
- Clinical trial publication A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trialCarcao M, Schiavulli M, Kulkarni R et al. · Blood advances · 2024 · PMID 38266154
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 Efmoroctocog Alfa approved for Haemophilia A / B?
Efmoroctocog 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 Efmoroctocog Alfa in clinical trials for Haemophilia A / B?
ClinicalTrials.gov lists 8 registered trials linking Efmoroctocog Alfa to Haemophilia A / B: 1 is active or not yet recruiting; 3 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03103542). The largest enrolment is 427 participants (NCT04293523). Registration activity spans 2015 to 2023.
What does the evidence show for Efmoroctocog Alfa in Haemophilia A / B?
Efmoroctocog Alfa has 3 completed trials and 2 linked publications for Haemophilia A / B. Completed trials may or may not have posted results; follow the NCT links to check. 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.