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Factor VIII (FVIII)
Factor VIII is a clotting factor that replaces deficient or dysfunctional coagulation factor VIII to restore blood clotting ability in hemophilia A patients.
Factor VIII is a clotting factor that replaces deficient or dysfunctional coagulation factor VIII to restore blood clotting ability in hemophilia A patients. Used for Hemophilia A (congenital Factor VIII deficiency), Acquired hemophilia A.
At a glance
| Generic name | Factor VIII (FVIII) |
|---|---|
| Also known as | Afstyla, Kovaltry |
| Sponsor | Hoffmann-La Roche |
| Drug class | Coagulation factor replacement therapy |
| Target | Factor VIII (coagulation factor VIII) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
Factor VIII is a critical cofactor in the intrinsic coagulation pathway that works with von Willebrand factor and other factors to activate Factor X, ultimately leading to thrombin generation and fibrin clot formation. In hemophilia A, Factor VIII is either absent or non-functional, leading to impaired clotting. Replacement therapy restores the ability to form stable blood clots and prevent or treat bleeding episodes.
Approved indications
- Hemophilia A (congenital Factor VIII deficiency)
- Acquired hemophilia A
Common side effects
- Inhibitor development (anti-Factor VIII antibodies)
- Thrombotic events
- Hypersensitivity reactions
- Injection site reactions
Key clinical trials
- A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B (PHASE3)
- Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa (PHASE4)
- A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
- Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (PHASE3)
- Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle" (PHASE1, PHASE2)
- An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
- A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease (PHASE3)
- An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |