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Veopoz (POZELIMAB)
Veopoz works by blocking the activity of Complement C5, a protein that contributes to inflammation.
Veopoz (POZELIMAB) is a small molecule developed by Regeneron Pharmaceuticals that targets Complement C5. It was approved by the FDA in 2023 for the treatment of CHAPLE disease. Veopoz works by inhibiting the activity of Complement C5, a protein involved in the immune system's response to inflammation. As a patented product, Veopoz is currently only available from its manufacturer. Key safety considerations include the potential for increased risk of infections due to its immunomodulatory effects.
At a glance
| Generic name | POZELIMAB |
|---|---|
| Sponsor | Regeneron |
| Drug class | Complement Inhibitor [EPC] |
| Target | Complement C5 |
| Modality | Monoclonal antibody |
| Therapeutic area | Immunology |
| Phase | FDA-approved |
| First approval | 2023 |
Mechanism of action
Pozelimab-bbfg is human, monoclonal immunoglobulin G4P (IgG4P) antibody directed against the terminal complement protein C5 that inhibits terminal complement activation by blocking cleavage of C5 into C5a (anaphylatoxin) and C5b, thereby blocking the formation of the membrane-attack complex (C5b-C9, structure mediating cell lysis).
Approved indications
- CHAPLE disease
Boxed warnings
- WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update meningococcal vaccination (for serogroups A, C, W and Y, and serogroup B) at least 2 weeks prior to administering the first dose of VEOPOZ, unless the risks of delaying therapy outweigh the risk of developing a meningococcal infection. Follow the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients receiving a complement inhibitor. Patients receiving VEOPOZ are at increased risk for invasive disease caused by N. meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected [see Warnings and Precautions (5.1) ] . WARNING: SERIOUS MENINGOCOCCAL INFECTIONS See full prescribing information for complete boxed warning Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. ( 5.1 ) Complete or update meningococcal vaccination at least 2 weeks prior to administering the first dose of VEOPOZ, unless the risks of delaying therapy outweigh the risks of developing meningococcal infection. Follow the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccination in patients receiving a complement inhibitor. ( 5.1 ) Patients receiving VEOPOZ are at increased risk for invasive disease caused by N. meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs of meningococcal infections and evaluate immediately if infection is suspected. ( 5.1 )
Common side effects
- Upper respiratory tract infection
- Fracture
- Urticaria
- Alopecia
- Injection site reactions
- Metabolic acidosis
- Gingival bleeding
- Increased blood uric acid
- Increased liver enzymes
- Hematuria
- Proteinuria
Key clinical trials
- A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy (PHASE3)
- A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently (PHASE3)
- A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works (PHASE3)
- Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants (PHASE1)
- A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment (PHASE3)
- A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (PHASE3)
- A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease (PHASE4)
- Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis (EARLY_PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
| Biologic Exclusivity |
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 |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Veopoz CI brief — competitive landscape report
- Veopoz updates RSS · CI watch RSS
- Regeneron portfolio CI