The USFDA has recently approved pozelimab for treating CD55-deficient protein-losing enteropathy (CHAPLE disease) in adult and pediatric patients above one year of age. CHAPLE disease is a rare and life-threatening disease that affects fewer than 100 patients worldwide . It is an inherited immune disease caused by mutations in the CD55 gene . CHAPLE disease is characterized by complement hyperactivation, protein-losing enteropathy, and angiopathic thrombosis .
The symptoms of the disease include severe thrombotic vascular occlusions, loss of appetite, diarrhea, and abdominal pain. The approval was granted based on a clinical trial demonstrating the safety and efficacy of pozelimab. Clinical trial Ten patients aged three to 19 years were treated with 30 mg/kg of pozelimab intravenously on day one, followed by weekly subcutaneous injections. The pozelimab treatment achieved normalization…
