Overview
Efanesoctocog alfa (BIVV001) is a recombinant factor VIII (FVIII) analogue fusion protein used for the routine prophylaxis, perioperative management of bleeding and on-demand treatment and control of bleeding episodes in patients with hemophilia A. The use of FVIII replacement products is beneficial in patients with hemophilia A; however, their quality of life can be affected due to frequent doses. Efanesoctocog alfa was designed to have an extended half-life, and to surpass the half-life ceiling to which other forms of recombinant FVIII are subjected due to their association with von Willebrand factor (VWF). Endogenous VWF protects FVIII from degradation but also sets a half-life of approximately 15 to 19 h. To extend the half-life of FVIII, efanesoctocog alfa is fused to dimeric Fc, a D'D3 domain of VWF, and two XTEN polypeptides. In February 2023, efanesoctocog alfa was approved by the FDA as a new class of factor VIII therapy for hemophilia A.
Indication
Efanesoctocog alfa is indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding.
Associated Conditions
- Bleeding
- Perioperative Blood Loss
- Bleeding episodes
Research Report
Efanesoctocog Alfa: A Comprehensive Clinical and Pharmacological Analysis of a First-in-Class Factor VIII Replacement Therapy
Executive Summary
Efanesoctocog alfa represents a paradigm shift in the management of Hemophilia A. It is a first-in-class, high-sustained Factor VIII (FVIII) replacement therapy engineered to overcome the primary biological limitation of previous treatments: the von Willebrand factor (vWF) ceiling on FVIII half-life.[1] Through a sophisticated fusion protein design, efanesoctocog alfa circulates independently of endogenous vWF, achieving a 3- to 4-fold extension in half-life compared to conventional therapies.[3] This novel pharmacokinetic profile allows for a simplified once-weekly prophylactic dosing regimen that maintains FVIII activity in the normal to near-normal range for the majority of the week.[5]
The pivotal Phase 3 clinical trials, XTEND-1 in adults and adolescents and XTEND-Kids in children, demonstrated profound clinical efficacy. Once-weekly prophylaxis with efanesoctocog alfa resulted in a statistically significant and clinically meaningful reduction in annualized bleeding rates (ABR), proving superior to prior FVIII prophylaxis in an intra-patient comparison.[6] The median ABR was zero across all age groups, and treatment was associated with significant improvements in joint health, pain, and physical functioning.[6] The therapy was well-tolerated, with a favorable safety profile notable for the absence of inhibitor development in the pivotal studies.[5]
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/04/24 | Phase 4 | Recruiting | |||
2025/04/23 | Phase 4 | Recruiting | |||
2024/12/31 | Phase 4 | Recruiting | |||
2024/12/04 | Phase 3 | Recruiting | |||
2024/11/12 | N/A | Recruiting | |||
2024/08/30 | Phase 1 | Recruiting | |||
2024/07/31 | N/A | Recruiting | |||
2023/04/18 | Phase 3 | Active, not recruiting | |||
2021/09/13 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| No FDA approvals found for this drug. | |||||
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 6/17/2024 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| ALTUVIIIO POWDER AND SOLVENT FOR SOLUTION FOR INJECTION 500IU | N/A | N/A | N/A | 10/10/2024 | |
| ALTUVIIIO POWDER AND SOLVENT FOR SOLUTION FOR INJECTION 3000IU | N/A | N/A | N/A | 10/10/2024 |
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| No Health Canada approvals found for this drug. | |||||
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| ALTUVOCT 1 000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824004 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized | |
| ALTUVOCT 500 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824002 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized | |
| ALTUVOCT 250 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824001 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized | |
| ALTUVOCT 750 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824003 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Not Commercialized | |
| ALTUVOCT 3 000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824006 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized | |
| ALTUVOCT 2 000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824005 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized | |
| ALTUVOCT 4 000 UI POLVO Y DISOLVENTE PARA SOLUCION INYECTABLE | 1241824007 | POLVO Y DISOLVENTE PARA SOLUCIÓN INYECTABLE | Uso Hospitalario | Commercialized |
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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