FDA Approves Veopoz for Hereditary Immune Disease

The FDA has approved Veopoz (pozelimab-bbfg), the first treatment for both adult and pediatric patients 1 year of age and older with CHAPLE disease, an ultra-rare and life-threatening hereditary immune disease. Those living with CHAPLE are unable to regulate complement activity due to mutations in the CD55 gene. This can lead the immune system to attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract. There are fewer than 100 patients worldwide who are known to have CHAPLE.

Developed by Regeneron, Veopoz is a fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation. The list price is $34,615.38 per single-use vial, but a company spokesperson the annual costs will vary based on weight. The spokesperson said that the company considered many factors including the new and significant advancement this medicine represents. Regeneron’s myRARE patient support program now includes assistance focused on the needs of eligible CHAPLE patients, including insurance coverage, financial support and information on treatment.

Michael Lenardo, M.D.

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” 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), said in a press release. “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 FDA approval is based on results from a phase 2/3 open-label trial that investigated the efficacy and safety of Veopoz in 10 patients aged 3 to 19. All patients achieved normalization of serum albumin and serum IgG concentrations by week 12 and maintained these concentrations through at least 72 weeks of treatment. The most common adverse reactions occurring in two or more patients included upper respiratory tract infection, fracture, urticaria, and alopecia.

Veopoz is also being evaluated in combination with Alnylam’s cemdisiran (siRNAi C5 inhibitor) as a combination therapy to treat other complement-mediated disorders, including paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis (MG).