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Factor IX (FIX)
Factor IX is a vitamin K-dependent clotting factor that replaces deficient or dysfunctional Factor IX to restore the intrinsic coagulation pathway and enable normal blood clot formation.
Factor IX is a vitamin K-dependent clotting factor that replaces deficient or dysfunctional Factor IX to restore the intrinsic coagulation pathway and enable normal blood clot formation. Used for Hemophilia B (Factor IX deficiency) — prevention and treatment of bleeding episodes, Hemophilia B — perioperative management.
At a glance
| Generic name | Factor IX (FIX) |
|---|---|
| Sponsor | CSL Behring |
| Drug class | Clotting factor replacement therapy |
| Target | Factor IX (coagulation factor IX) |
| Modality | Biologic |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
Factor IX is a serine protease that functions as part of the tenase complex in the intrinsic coagulation cascade. By replacing missing or defective endogenous Factor IX, the drug restores the ability to generate thrombin and form stable fibrin clots. This is essential for patients with Factor IX deficiency (hemophilia B) who otherwise have impaired coagulation and are at risk of spontaneous or excessive bleeding.
Approved indications
- Hemophilia B (Factor IX deficiency) — prevention and treatment of bleeding episodes
- Hemophilia B — perioperative management
Common side effects
- Thrombosis (deep vein thrombosis, pulmonary embolism)
- Inhibitor development (anti-Factor IX antibodies)
- Injection site reactions
- Headache
- Fever
Key clinical trials
- HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients (PHASE3)
- Evaluation of 4-Factor PCC in DOAC-associated Intracranial Hemorrhage
- A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B (PHASE1, PHASE2)
- Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B (PHASE1, PHASE2)
- A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
- Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B (PHASE2)
- Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs) (PHASE3)
- Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass (CPB) (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 |