FDA approves Veopoz as the first treatment for children and adults with Chaple disease. Regeneron.

CHAPLE is an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. In healthy individuals, the complement system is a mechanism for destroying microbes. However, those living with CHAPLE are unable to regulate complement activity due to mutations in their CD55 gene. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of circulating proteins. There are fewer than 10 patients with CHAPLE disease identified in the U.S.

Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. Meningococcal infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update meningococcal vaccination at least two weeks prior to administering the first dose of Veopoz, unless the risks of delaying therapy outweigh the risks of developing meningococcal infection. If urgent therapy is indicated in a patient who is not up-to-date with both meningococcal vaccines according to Advisory Committee on Immunization Practices (ACIP) recommendations, administer meningococcal vaccines as soon as possible and provide the patient with antibacterial drug prophylaxis. Vaccination reduces, but does not eliminate, the risk of meningococcal infections.

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, M.D., Chief, Molecular Development of the Immune System Section; Co-Director, Clinical Genomics Program, National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH). “As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that pozelimab achieves in those suffering from CHAPLE. The approval of pozelimab is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”