FDA approves first ever treatment for rare CHAPLE disease

FDA approves first ever treatment for rare CHAPLE disease

The US Food and Drug Administration (FDA) has approved Regeneron’s Veopoz (pozelimab), a new treatment for CHAPLE disease for both adult and paediatric patients over one year of age. CHAPLE is a hereditary disease driven by the overactivation of a patient’s complement system; it has been known to cause life-threatening gastrointestinal and cardiovascular symptoms. The disease is ultra-rare, with fewer than ten patients in the US having been diagnosed.

Michael Lenardo, chief of molecular development of the immune system at the National Institutes of Health (NIH), commented: “Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy. 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.” […]

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