Regeneron Pharmaceuticals has received FDA approval for Veopoz™ (pozelimab-bbfg) as a pioneering treatment for CHAPLE disease in both adults and pediatric patients aged one year and older. CHAPLE disease, also known as CD55-deficient protein-losing enteropathy, is an exceptionally rare and life-threatening hereditary immune disorder characterized by an overactive complement system. This system, typically responsible for fighting microbes in healthy individuals, attacks normal cells in CHAPLE patients due to mutations in their CD55 gene. This can result in damage to blood and lymph vessels in the upper digestive tract and the loss of circulating proteins.
Veopoz, a fully human monoclonal antibody, targets complement factor C5, a crucial protein in the complement system. As the first and only treatment indicated specifically for CHAPLE, Veopoz represents a significant advance in addressing the severe and often life-threatening symptoms experienced by affected children and adults.
It's crucial to note that patients treated with complement inhibitors like Veopoz may be at risk of life-threatening meningococcal infections. Proper meningococcal vaccination, administered at least two weeks before starting Veopoz treatment, is essential to mitigate this risk, unless delaying therapy poses a greater danger.
The FDA's approval is based on results from a Phase 2/3 open-label trial involving 10 patients aged 3 to 19, with a median age of 8.5 years. These patients received a single loading dose of pozelimab 30 mg/kg intravenously on day 1, followed by subcutaneous weekly weight-based doses of the medication.
Veopoz was developed using Regeneron's VelocImmune® technology and is a fully human monoclonal antibody designed to block complement factor C5's activity, preventing diseases mediated by the complement pathway. Additionally, it is currently being evaluated in combination with Alnylam's cemdisiran (siRNAi C5 inhibitor) as an investigational combination therapy for other complement-mediated disorders, such as paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis (MG). Please note that this combination therapy is still under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.