Regeneron received approval from the U.S. Food and Drug Administration (FDA) on Aug. 18, 2023, for Veopoz™ (pozelimab-bbfg). The immunoglobulin G (IgG) 4 monoclonal antibody interferes with the activity of complement 5 to treat CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE disease) for patients as young as one year old. It will be given by healthcare providers as one intravenous (IV) infusion of 30mg/kg, which is followed once each week at 10mg/kg administered subcutaneously (SC) with an upper limit of 800mg per dose. Veopoz will be launched before the end of September 2023, at a wholesale acquisition cost (WAC) of $34,615 per 400mg vial. It has a boxed warning because some patients developed severe meningitis while using it. Full prescribing information is here.
At a Glance
- Brand (Generic) Name: Veopoz (pozelimab-bbfg)
- Manufacturer: Regeneron
- Date Approved: Aug. 18, 2023
- Indication: to treat patients who are one year old and older for CHAPLE disease
- Dosage Forms Available: single-dose vials of 400mg/2mL
- Launch Date: Third quarter 2023
- Estimated Annual Cost: At one vial per week for a 35kg (77 pounds) patient, WAC for one year is slightly less than $1.8 million
- At an estimated incidence of fewer than one in one million, CHAPLE disease is an ultra-rare hereditary condition that affects 10 or fewer American patients. It generally is diagnosed in early childhood,
- Patients have cardiovascular (CV) and gastrointestinal (GI) symptoms that contribute to anemia, blood clots, malabsorption, and stunted growth.
- One biomarker of the condition is extremely low blood levels of albumin, which is caused by the complement system attacking normal cells, especially along the digestive tract, blood, or lymph vessels.
- Veopoz blocks over activity of the immune system protein, complement C5, to interfere with the progression of CHAPLE disease.
- Veopoz is the first drug specifically indicated to treat CHAPLE disease. Previously, treatment consisted only of symptom control.
- In a phase II/III open-label clinical trial, 10 participants were treated with Veopoz for 12 weeks and their albumin and IgG levels remained within normal ranges for at least 72 weeks. In the 48 weeks before the trial started, five out of the 10 study patients needed 60 transfusions of albumin and nine out of the 10 patients were hospitalized for 268 days. Only one patient needed a transfusion and two patients needed hospital treatment for a total of seven days within the 48 weeks after treatment was initiated.
- Common side effects included fractures, itching, hair loss, and upper respiratory infections.
- Patients should have meningitis vaccines two weeks or earlier before starting Veopoz.
- Veopoz is an Orphan Drug approved through Fast Track and Rare Pediatric Disease pathways.