Development
Veopoz (pozelimab-bbfg) injection, a complement inhibitor, has received FDA approval for managing CD55-deficient protein-losing enteropathy (PLE), or CHAPLE disease, in patients aged one year and above. This marks the pioneering FDA-approved therapy for CHAPLE disease. Initially, Veopoz is administered intravenously, followed by weekly subcutaneous injections supervised by a healthcare professional. For comprehensive dosing instructions, refer to the prescribing information
Disease Overview
CHAPLE disease, or complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy, is a hereditary immune disorder causing excessive activation of the complement system. This system usually defends against invaders like bacteria and viruses. CHAPLE disease arises from mutations in the CD55 gene, a complement regulator. These mutations lead the complement system to mistakenly target the body’s own cells.
CHAPLE disease is exceedingly rare, with fewer than 100 documented cases worldwide. Its symptoms encompass abdominal discomfort, vomiting, nausea, diarrhea, weight loss, loss of appetite, edema (swelling), and compromised growth. In severe instances, life-threatening thrombotic vascular occlusions (blood vessel blockages) can transpire.
Veopoz Efficacy Assessment
The efficacy and safety of Veopoz were assessed in a single-arm study (NCT04209634), where patient outcomes were compared to their pre-treatment data. The study involved patients with active CD55-deficient protein-losing enteropathy (PLE) exhibiting hypoalbuminemia. Diagnosis criteria included a clinical history of PLE symptoms and confirmed biallelic CD55 loss-of-function mutation. Active CD55-deficient PLE was defined as hypoalbuminemia (serum albumin concentration of ≤3.2 g/dL) accompanied by specific signs or symptoms within the past six months.
Patients received a single 30 mg/kg loading dose of Veopoz intravenously, succeeded by a weekly maintenance dose via subcutaneous injection, adjusting based on body weight, commencing one week post the loading dose. A group of ten patients, aged 3 to 19 years (median 8.5 years), were evaluated for efficacy. All ten patients exhibited a serum albumin concentration of at least 3.5 g/dL by week 12, which persisted for at least 72 weeks. Additionally, all ten displayed reduced hospitalizations and albumin transfusions in the first 48 weeks of treatment compared to the prior 48 weeks.
Safety Considerations
Common adverse reactions to Veopoz encompass upper respiratory tract infections, fractures, hives, and alopecia. Veopoz carries a Boxed Warning for severe meningococcal infections. Fatal meningococcal infections have arisen in patients treated with complement inhibitors. Swift identification and early intervention are crucial in such cases. Patients on Veopoz should complete meningococcal vaccination at least two weeks before treatment. They face an elevated risk of invasive disease caused by N. meningitidis, even if they develop antibodies following vaccination. An increased risk of bacterial infections, notably by encapsulated bacteria, is also observed. Patients should adhere to vaccination guidelines outlined by the Advisory Committee on Immunization Practice. Comprehensive risk details are available in the full prescribing information.
Designations
Veopoz secured fast track, orphan drugs, and rare pediatric disease designations.
Sources: FDA