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General Information
Veopoz (pozelimab-bbfg) is a complement inhibitor.
Veopoz is specifically 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.
Dosing/Administration
Veopoz is supplied as an injection for intravenous or subcutaneous use.
Prior to the first dose:
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.
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.
The recommended dose of Veopoz is as follows:
Day 1 (Loading Dose): Administer a single 30 mg/kg dose by intravenous infusion after dilution.
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.
- Doses greater than 400 mg require 2 injections
Mechanism of Action
Pozelimab-bbfg is a fully human, monoclonal antibody designed to block the activity of complement factor C5 and prevent diseases mediated by the complement pathway.
Pozelimab-bbfg is a 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, a structure mediating cell lysis).
Side Effects
Adverse effects associated with the use of Vepoz may include, but are not limited to, the following:
- upper respiratory tract infection
- fracture
- urticaria
- alopecia
The Vepoz drug label comes with the following Black Box 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. Complete or update meningococcal vaccination at least 2 weeks prior to administering the first dose of Vepoz, 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. Patients receiving Vepoz 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.
Clinical Trial Results
The FDA approval of Vepoz was based on results from a Phase 2/3 open-label trial that investigated the efficacy and safety of pozelimab in 10 patients aged 3 to 19 (median of 8.5 years). Patients were given a single loading dose of pozelimab 30 mg/kg intravenously on day 1, followed by subcutaneous weekly weight-based doses of pozelimab.
All ten patients achieved normalization of serum albumin and serum IgG concentrations by week 12 and maintained these concentrations through at least 72 weeks of treatment. Five of the 10 patients received a total of 60 albumin transfusions in the 48 weeks prior to treatment. In the 48 weeks after starting treatment, one patient received one albumin transfusion. Nine of the 10 patients were hospitalized for a total of 268 days in the 48 weeks prior to treatment. In the 48 weeks after starting treatment, two patients were hospitalized for a total of 7 days.