INDIANA – The FDA has approved Veopoz (pozelimab-bbfg) injection, 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 CHAPLE disease.
Veopoz is the first FDA-approved treatment for CHAPLE disease. An initial dose of Veopoz is administered intravenously, followed by weekly injections given subcutaneously (below the skin) by a healthcare provider. See prescribing information for detailed dosing information.
CHAPLE stands for complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy. It is an inherited immune disease that causes the complement system (the part of your immune system that defends the body against injury and foreign invaders like bacteria and viruses) to become overactive. It is caused by mutations of the complement regulator CD55 gene, which can lead to the complement system attacking the body’s cells.
CHAPLE disease is rare, with fewer than 100 patients diagnosed worldwide. Symptoms can include abdominal pain, nausea, vomiting, diarrhea, loss of appetite, weight loss, impaired growth, and edema (swelling). Severe thrombotic vascular occlusions (blockage of blood vessels) can also occur among patients with CHAPLE disease, which can be life-threatening.
The most common adverse reactions to Veopoz are upper respiratory tract infections, fractures, hives, and alopecia. Veopoz has a Boxed Warning for serious meningococcal infections. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors and may become rapidly life-threatening or fatal if not recognized and treated early. Patients receiving Veopoz should complete or update meningococcal vaccination at least two weeks before treatment. Patients receiving Veopoz are at increased risk for invasive disease caused by N. meningitidis, even if they develop antibodies following vaccination. Patients are also at increased risk of other bacterial infections, particularly those caused by encapsulated bacteria (such as Streptococcus pneumoniae, Haemophilus influenzae type b (Hib), and Neisseria gonorrhoeae). Patients should receive vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib), per the Advisory Committee on Immunization Practice guidelines. See full prescribing information for additional information on risks associated with Veopoz.
