The regulator’s greenlight on Friday for Regeneron Pharmaceuticals’ monoclonal antibody Veopoz (pozelimab) makes it the first and only treatment indicated for children and adults with CHAPLE disease.
Pictured: Regeneron logo on a light brown building/Shutterstock, Lev Radin
The FDA on Friday approved Regeneron Pharmaceuticals’ Veopoz (pozelimab), the first and only treatment indicated specifically for CHAPLE disease, also known as CD55-deficient protein-losing enteropathy, according to the company.
A fully human monoclonal antibody, Veopoz is approved for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE, an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms.
In February 2023, the regulator gave Priority Review to Regeneron’s investigational antibody which works by binding to the C5 complement factor, thereby disrupting the complement cascade and preventing associated diseases.
CHAPLE causes a variety of life-threatening conditions, including recurrent infections, malnutrition, and bloody diarrhea. A mutated C55 gene results in an overactive complement system, which enhances the activity of antibodies and phagocytic cells. This in turn causes the hyperactive immune system to attack healthy cells, particularly blood and lymph vessels, as well as the digestive tract.
Michael Lenardo, co-director of the Clinical Genomics Program at the National Institute of Allergy and Infectious Disease, said in a statement that most patients with the disease “are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy” and that the FDA approval “is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”
Regeneron presented Phase II/III data in its application for regulatory approval showing “rapid and sustained normalization” of one of the key markers of CHAPLE disease, albumin, in all 10 patients at 24 weeks. The treatment also eased symptoms such as increased bowel movements and abdominal pain.
The drug is also being evaluated for use in combination with cemdisiran, an investigational NRAi therapeutic developed by Alnylam Pharmaceuticals, to treat other complement-mediated disorders, such as paroxysmal nocturnal hemoglobinuria and myasthenia gravis. Regeneron’s Chief Science Officer George Yancopoulos said in a statement that “Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential.”
The company noted in the release that patients treated with complementary inhibitors are prone to meningococcal infections, which can be life-threatening or fatal, and that patients receiving Veopoz should have their vaccinations completed or updated at least two weeks before receiving the medication.
Regeneron also said that the approval has resolved pre-approval inspection issues related to its aflibercept 8 mg Biologics License Application, and that the company is anticipating FDA action on the BLA in the coming weeks.
Connor Lynch is a freelance writer based in Ottawa, Canada. Reach him at lynchjourno@gmail.com.
