Last reviewed · How we verify
factor concentrates
Factor concentrates replace or supplement deficient clotting factors to restore hemostatic function in patients with bleeding disorders.
Factor concentrates replace or supplement deficient clotting factors to restore hemostatic function in patients with bleeding disorders. Used for Hemophilia A (Factor VIII deficiency), Hemophilia B (Factor IX deficiency), Other inherited factor deficiencies.
At a glance
| Generic name | factor concentrates |
|---|---|
| Sponsor | Genzyme, a Sanofi Company |
| Drug class | Coagulation factor replacement therapy |
| Target | Multiple coagulation factors (Factor VIII, Factor IX, Factor V, fibrinogen, prothrombin complex components) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
Factor concentrates are purified or recombinant blood coagulation factors (such as Factor VIII, Factor IX, Factor V, fibrinogen, or prothrombin complex concentrates) that are administered intravenously to patients with inherited or acquired deficiencies. They work by restoring the missing or dysfunctional clotting factors in the coagulation cascade, enabling normal thrombin generation and fibrin clot formation to prevent or treat bleeding episodes.
Approved indications
- Hemophilia A (Factor VIII deficiency)
- Hemophilia B (Factor IX deficiency)
- Other inherited factor deficiencies
- Acquired coagulation factor deficiencies
Common side effects
- Inhibitor development (alloimmunization)
- Thrombosis
- Infusion reactions
- Headache
- Fever
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)
- Monitoring of Anti-TFPI in Hemophilia
- 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
- Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass (CPB) (PHASE3)
- Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A (PHASE2, PHASE3)
- Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate
- A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (PHASE3)
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 |