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Coagulation Factor VIII
Coagulation Factor VIII replaces deficient or dysfunctional clotting factor VIII to restore the intrinsic coagulation pathway and enable normal blood clot formation.
Coagulation Factor VIII replaces deficient or dysfunctional clotting factor VIII to restore the intrinsic coagulation pathway and enable normal blood clot formation. Used for Hemophilia A (congenital Factor VIII deficiency), Acquired hemophilia A.
At a glance
| Generic name | Coagulation Factor VIII |
|---|---|
| Sponsor | Beijing Children's Hospital |
| Drug class | Coagulation factor replacement |
| Target | Coagulation Factor VIII (antihemophilic factor) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | FDA-approved |
Mechanism of action
Factor VIII is a critical cofactor in the intrinsic coagulation cascade that works with Factor IX to activate Factor X, ultimately leading to thrombin generation and fibrin clot formation. In hemophilia A, Factor VIII is either absent or non-functional, causing severe bleeding. Replacement therapy restores this enzymatic activity and corrects the coagulation defect.
Approved indications
- Hemophilia A (congenital Factor VIII deficiency)
- Acquired hemophilia A
Common side effects
- Inhibitor development (alloimmunization)
- Infusion reactions
- Thrombosis
- Headache
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Coagulation Factor VIII CI brief — competitive landscape report
- Coagulation Factor VIII updates RSS · CI watch RSS
- Beijing Children's Hospital portfolio CI