This Plain Language Summary of Publication article (PLSP) from Future Rare Diseases, evaluated pozelimab (VEOPOZ), the first FDA-approved treatment for CHAPLE disease, a rare inherited condition affecting children. CHAPLE disease impacts the immune system and causes severe digestive symptoms including abdominal pain, sickness, diarrhea, and loss of appetite. These symptoms can lead to serious complications like malnutrition, anemia, poor growth, intestinal blockages, and blood vessel clots, which can be life-threatening. While existing supportive treatments provide limited relief, pozelimab represents a breakthrough as the first FDA-approved therapy for both adults and children with this condition.

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This PLSP is based on an article called ‘Evaluating the efficacy and safety of pozelimab in patients with CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy disease: an open-label phase 2 and 3 study’ and was published in The Lancet.

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