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Pozelimab-bbfg

(Veopoz®)

Veopoz®

Drug updated on 12/11/2024

Dosage Forminjection (intravenous or subcutaneous; 400 mg/2 mL [200 mg/mL])
Drug ClassComplement inhibitors
Ongoing and
Completed Studies		ClinicalTrials.gov

Indication

  • 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.

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Summary
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  • This summary is based on the review of one randomized controlled trial. [1]
  • In the study, all ten patients treated with pozelimab achieved serum albumin normalization and improvement in clinical outcomes by week 24, with no worsening in inactive clinical outcomes. Complete inhibition of total complement activity was observed in all patients.
  • The study population predominantly consisted of pediatric patients with a median age of 8.5 years, and all patients, regardless of age or origin, experienced serum albumin normalization and clinical improvement.
  • Nine out of ten patients treated with pozelimab experienced adverse events, with two patients having severe adverse events. One patient had an adverse event considered related to pozelimab.
  • The abstract does not specify any differences in the occurrence of adverse events among different population types or subgroups, although the study primarily involved pediatric patients.
  • The study population primarily consisted of pediatric patients with a median age of 8.5 years, including patients from Turkiye, Syria, Thailand, and Bolivia. All patients, regardless of age or geographical origin, experienced serum albumin normalization and clinical improvement, although specific subgroup analyses were not detailed.