FDA Approves Treatment for CD55-Deficient Protein-Losing Enteropathy

The FDA has approved pozelimab-bbfg (Veopoz; Regeneron) injection, which is a complement inhibitor, for the treatment of adult and pediatric patients 1 year of age and older with CD55-deficient protein-losing enteropathy (PLE), also known as complement hyperactivation, angiopathic thrombosis, and PLE (CHAPLE) disease, according to a statement released by the agency.

The first dose of pozelimab-bbfg is administered intravenously and is followed by weekly injections given below the skin by a health care provider. It is the first FDA-approved treatment for CHAPLE disease.

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Pozelimab-bbfg’s efficacy and safety were evaluated in a single-arm study in which patients’ outcomes were compared to pre-treatment data in those with active CD55-deficient PLE who had hypoalbuminemia. Diagnoses were based on a clinical history of PLE symptoms and with a confirmed genotype of biallelic CD55 loss-of-function mutation.

Patients were treated with a single 30 mg/kg loading dose of pozelimab-bbfg administered by intravenous infusion, followed by a weekly maintenance dosage administered as a subcutaneous injection starting 1 week after the loading dose. The 10 patients assessed for efficacy achieved a serum albumin concentration of at least 3.5 g/dL by week 12 and was maintained through at least 72 weeks.

Additionally, all 10 patients demonstrated a reduction in the number of hospitalizations and number of albumin transfusions over the first 48 weeks of treatment as compared to the 48 weeks prior to treatment.

CHAPLE is an inherited immune disease that causes the complement system—the part of the immune system that protects the body against injury and invaders such as bacteria and viruses—to become overactive. The disease is caused by mutations of the complement regulator CD55 gene, which can lead to the system attacking the body’s own cells.

Fewer than 100 patients have diagnosed CHAPLE disease worldwide. Symptoms can include nausea, abdominal pain, vomiting, loss of appetite, diarrhea, weight loss, impaired growth, and edema. Additionally, severe thrombotic vascular occlusions can occur among patients with CHAPLE disease, which can be life-threatening.

The most common adverse effects with pozelimab-bbfg include upper respiratory tract infections, fractures, hives, and alopecia. It also has a Boxed Warning for serious meningococcal infections, and life-threatening and fatal meningococcal infections have occurred in patients with complement inhibitors. The statement advises patients receiving the drug to complete or update meningococcal vaccination at least 2 weeks prior to treatment.

Reference

U.S. Food & Drug Administration. FDA approves first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease). News release. Accessed on August 21, 2023. Updated on August 18, 2023. Accessible at https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-cd55-deficient-protein-losing-enteropathy-chaple-disease.