FDA approves pozelimab-bbfg as first CHAPLE disease treatment for children, adults

FDA approves pozelimab-bbfg as first CHAPLE disease treatment for children, adults | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA has approved the first and only treatment specifically indicated to treat CHAPLE disease for pediatric patients 1 year or older and adults. Pozelimab-bbfg (Veopoz; Regeneron Pharmaceuticals, Inc.) has been approved to treat the ultra-rare, hereditary, and life-threatening immune disease, according to a press release from Regeneron.

CHAPLE disease, which is also known as CD55-deficient protein-losing enteropathy, is driven by an overactivation of the complement system, which serves as a mechanism for destroying microbes. Those living with CHAPLE cannot regulate complement activity because of mutations in their CD55 gene. A lack of this regulation can result in an attack from the complement system. This can cause damage to lymph and blood vessels on the upper digestive tract, which can lead to the loss of circulating proteins, according to the press release. The fully human monoclonal antibody was developed to target complement factor C5, a protein involved in complement system activation. In the United States, less than 10 patients have been identified with CHAPLE disease.

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, MD, 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), in the 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-bbfg achieves in those suffering from CHAPLE. The approval of pozelimab-bbfg is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”

The FDA approval is based on results demonstrated in a phase 2/3 open-label trial (NCT04209634) that determined the safety and efficacy of pozelimab-bbfg. Ten patients aged 3 to 19 years (median age: 8.5 years) were given a single loading dose of pozelimab-bbfg 30 mg/kg intravenously on day 1. Subcutaneous weekly, weight-based doses followed. Regeneron states all 10 patients achieved normalization of serum albumin and serum IgG concentrations by week 12. These concentrations were maintained through at least 72 weeks. Half of treated patients received a total of 60 albumin transfusions in the 48 weeks prior to treatment. One patient received 1 albumin transfusion in the 48 weeks after treatment initiation. Nine out of 10 patients were hospitalized for a total of 268 days in the 48 weeks before treatment. Two patients were hospitalized for a combined 7 days in the 48 weeks after treatment initiation.

Upper respiratory tract infection, urticaria, fracture, and alopecia were the most common adverse reactions occurring in 2 or more patients. It is unclear if pozelimab-bbfg is safe and effective in children under 1 year. Patients should not receive pozelimab-bbfg if they have a meningococcal infection. Click here for more safety information on pozelimab.

Reference:

Veopoz (pozelimab-bbfg) receives FDA approval as the first treatment for children and adults with CHAPLE disease. Regeneron Pharmaceuticals Inc. Press release. August 18, 2023. Accessed August 18, 2023. https://investor.regeneron.com/news-releases/news-release-details/veopoztm-pozelimab-bbfg-receives-fda-approval-first-treatment