Pozelimab

Pozelimab-bbfg is a human, monoclonal immunoglobulin G4 P (IgG4 P ) 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, a structure mediating cell lysis).

• VEOPOZ is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. VEOPOZ is a complement inhibitor indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.

• VEOPOZ is contraindicated in: Patients with unresolved Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . VEOPOZ is contraindicated in patients with unresolved Neisseria meningitidis infection.contraindicated

See the full prescribing information for meningococcal vaccine and prophylaxis recommendations prior to the first dose of VEOPOZ. ( 2.1 ) Recommended Dosage: Day 1 (loading dose): Administer a single 30 mg/kg dose by intravenous infusion after dilution. ( 2.2 ) Day 8 and thereafter (maintenance dosage): Inject 10 mg/kg as a subcutaneous injection once weekly starting on Day 8. The maintenance dosage may be increased to 12 mg/kg once weekly if there is inadequate clinical response after at least 3 weekly doses (i.e., starting from Week 4). The maximum maintenance dosage is 800 mg once weekly. ( 2.2 ) See full prescribing information for instructions on preparation and administration. ( 2.2 , 2.3 , 2.4 ) 2.1 Recommended Vaccination and Prophylaxis for Meningococcal Infection Prior to First Dose of VEOPOZ Vaccinate patients for meningococcal infection (serogroups A, C, W, and Y [MenACWY] and serogroup B [MenB]) according to current ACIP recommendations for patients receiving a complement inhibitor at least 2 weeks prior to administering the first dose of VEOPOZ [see Warnings and Precautions (5.1) ] . If urgent VEOPOZ therapy is indicated in a patient who is not up-to-date with vaccines for both MenACWY and MenB according to ACIP recommendations, administer meningococcal vaccine(s) as soon as possible and provide the patient with antibacterial drug prophylaxis. The efficacy, duration, and drug regimens for antibacterial drug prophylaxis have not been studied in patients receiving complement inhibitors, including VEOPOZ.

Other Bacterial Infections : Interrupt treatment with VEOPOZ in patients who are undergoing treatment for a serious encapsulated bacterial infection until the infection is resolved. ( 5.2 ) Systemic Hypersensitivity Reactions : Interrupt infusion and institute appropriate supportive measures if signs of cardiovascular instability or respiratory compromise occur. ( 5.3 ) Immune Complex Formation : Transition between other complement inhibitors has resulted in decreased drug concentrations and possible hypersensitivity reactions. Consider this potential if switching complement inhibitors to VEOPOZ. ( 5.4 ) 5.1 Serious Meningococcal Infections Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The use of VEOPOZ increases a patient's susceptibility to serious and life-threatening meningococcal infections (septicemia and/or meningitis) caused by any serogroup, including nongroupable strains. Complete or update meningococcal vaccination (for serogroups A, C, W, and Y [MenACWY] and serogroup B [MenB]) at least 2 weeks prior to administering the first dose of VEOPOZ, according to the most current Advisory Committee on Immunization Practices (ACIP) recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of VEOPOZ therapy.

The following clinically significant adverse reactions are described elsewhere in the labeling: Serious Meningococcal Infections [see Warnings and Precautions (5.1) ] Other Bacterial Infections [see Warnings and Precautions (5.2) ] Systemic Hypersensitivity Reactions [see Warnings and Precautions (5.3) ] Immune Complex Formation [see Warnings and Precautions (5.4) ] Most common adverse reactions (in two or more patients) are: upper respiratory tract infection, fracture, urticaria, and alopecia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Regeneron at 1-855-583-6769 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of VEOPOZ was evaluated in 10 patients with CD55-deficient PLE (ranging from 3 to 19 years of age) in a single-arm study [see Clinical Studies (14) ] . The median duration of exposure was 104 weeks (range: 75 to 140 weeks). Adverse reactions reported in two or more patients are summarized in Table 1.

8.1 Pregnancy Risk Summary Although there are no data on VEOPOZ use in pregnant women to inform a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes, monoclonal antibodies can be actively transported across the placenta . In an animal reproduction study in monkeys, pozelimab-bbfg did not adversely affect embryofetal or postnatal development when administered from pregnancy confirmation through parturition at doses that produced exposure up to 3.3 to 3.8 times the predicted clinical exposures (on an AUC basis; see Data ). The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other outcome. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Data Animal Data In an enhanced pre- and postnatal development study, pregnant female monkeys were subcutaneously administered pozelimab-bbfg at doses of 5 or 50 mg/kg once weekly from confirmation of pregnancy (gestation day 20) through parturition (approximately gestation day 160).

Metabolism

In healthy subjects, single intravenous infusions of VEOPOZ over approximately one hour resulted in dose proportional increases in mean C max , but greater than proportional increases in mean AUC inf (>16-fold) for total pozelimab concentrations in serum between 3 mg/kg and 30 mg/kg. The mean AUC inf increased by 3.5-fold between 10 mg/kg and 30 mg/kg.

• Aug 18, 2023BLABLA761339Regeneron Pharmaceuticals

1. 1Drug Effect Less Than Expected125%
2. 2Hepatitis125%
3. 3Injection Site Reaction125%
4. 4Meningococcal Infection125%
5. 5Seizure125%

The 10 most recently updated of 19 ClinicalTrials.gov registrations naming Pozelimab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants
  Recruiting · Phase 1 · Interventional · 54 enrolled · Regeneron Pharmaceuticals
  NCT07230834updated 2026-05-28
• A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
  Recruiting · Phase 3 · Interventional · 975 enrolled · Regeneron Pharmaceuticals
  NCT06541704updated 2026-05-27
• 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
  Recruiting · Phase 3 · Interventional · 35 enrolled · Regeneron Pharmaceuticals
  NCT07154745updated 2026-05-26
• 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
  Active not recruiting · Phase 3 · Interventional · 202 enrolled · Regeneron Pharmaceuticals
  NCT05133531updated 2026-04-30
• 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
  Recruiting · Phase 4 · Interventional · 5 enrolled · Regeneron Pharmaceuticals
  NCT07142343updated 2026-04-13
• 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
  Recruiting · Phase 3 · Interventional · 202 enrolled · Regeneron Pharmaceuticals
  NCT05744921updated 2026-03-18
• 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
  Active not recruiting · Phase 3 · Interventional · 288 enrolled · Regeneron Pharmaceuticals
  NCT05070858updated 2026-02-12
• Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis
  Recruiting · Early phase 1 · Interventional · 10 enrolled · Austin Neuromuscular Center
  NCT06479863updated 2026-01-16
• Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
  Approved for marketing · Expanded access · Regeneron Pharmaceuticals
  NCT06003881updated 2025-11-10
• Compassionate Use of Pozelimab
  Approved for marketing · Expanded access · Regeneron Pharmaceuticals
  NCT05232110updated 2025-11-10

Frequently asked questions

How does Pozelimab work?

Pozelimab-bbfg is a human, monoclonal immunoglobulin G4 P (IgG4 P ) 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, a structure mediating cell lysis).

What is Pozelimab used for?

According to FDA labeling, Pozelimab carries indications including: VEOPOZ is indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. VEOPOZ is a complement inhibitor indicated for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Pozelimab?

Pozelimab is classified as Complement inhibitors, Complement Inhibitor, Antibody Interactions, Complement Inhibitors.

What are the brand names for Pozelimab?

Pozelimab is marketed under brand names including Veopoz.

What are the contraindications for Pozelimab?

Pozelimab labeling lists contraindications including: VEOPOZ is contraindicated in: Patients with unresolved Neisseria meningitidis infection [see Warnings and Precautions (5.1) ] . VEOPOZ is contraindicated in patients with unresolved Neisseria meningitidis infection.. Always consult the full prescribing information and a clinician.