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factor ix
REBINYN replaces missing coagulation Factor IX and slows its removal from circulation through PEGylation.
REBINYN, a PEGylated coagulation Factor IX replacement therapy, is currently marketed by the National Heart, Lung, and Blood Institute (NHLBI) for on-demand treatment and control of bleeding episodes in patients with hemophilia B. Its key strength lies in the PEGylation technology, which extends the half-life of Factor IX, thereby reducing the frequency of injections needed. The primary risk to consider is the key composition patent expiry in 2028, which could lead to increased competition from generic alternatives.
At a glance
| Generic name | factor ix |
|---|---|
| Sponsor | National Heart, Lung, and Blood Institute (NHLBI) |
| Target | coagulation Factor IX |
| Modality | Small molecule |
| Phase | FDA-approved |
Mechanism of action
Patients with hemophilia B lack coagulation Factor IX, essential for stopping bleeding. REBINYN provides this missing factor, helping to control bleeding. The drug's Factor IX is attached to a polyethylene glycol molecule, which extends its time in the bloodstream.
Approved indications
- On-demand treatment and control of bleeding episodes
- Perioperative management of bleeding
- Routine prophylaxis to reduce the frequency of bleeding episodes
Common side effects
- injection site reactions
- hypersensitivity
- itching
- factor IX inhibition
- anaphylactic reaction
- rash
- itching
Key clinical trials
- HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients (PHASE3)
- 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)
- 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)
- 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:
- factor ix CI brief — competitive landscape report
- factor ix updates RSS · CI watch RSS
- National Heart, Lung, and Blood Institute (NHLBI) portfolio CI