FDA approves first ever treatment for rare CHAPLE disease

Betsy Goodfellow | August 21, 2023 | News story | Research and Development CHAPLE disease, FDA, Rare Diseases, rare 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.”

The FDA approved this treatment based on successful results from a phase 2 and 3 open-label trial, where the efficacy and safety of pozelimab was tested in patients aged three to 19. While results were positive, it was confirmed that patients must receive meningococcal vaccines before taking the treatment as it heavily increases the chances of obtaining infections that could lead to death.

George D Yancopoulos, chief scientific officer and president at Regeneron, stated: “As the first-ever treatment for CHAPLE, Veopoz is a testament to our commitment to uncovering genetic insights and applying them to the development of effective treatments for patients in need – regardless of the prevalence of their disease. Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programmes to explore its broader potential.”

Rebecca Lee

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