Veopoz (pozelimab) is a monoclonal antibody that's the first and only treatment for a rare genetic problem called CD55-deficient protein-losing enteropathy (CHAPLE disease). It can be used in people over the age of 1 with this genetic condition. Veopoz (pozelimab) is given as an injection under the skin once per week. Possible side effects include upper respiratory infections and hair loss.
CHAPLE disease is an autoimmune condition, which means that the body's immune system becomes overactive and starts attacking itself. This condition causes many problems in the body, including a loss of proteins through the gut and life-threatening complications like blood clots, infections, and malnutrition.
Veopoz (pozelimab) is a monoclonal antibody that works as a complement inhibitor. It blocks proteins that are part of the body's immune system called complement proteins from turning on. Veopoz (pozelimab) stops your immune system from being overactive and attacking your own body.
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Contact your healthcare provider immediately if you experience any of the following.
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Results from a small clinical study of 10 children (ages 3 to 19 years) with CHAPLE disease showed that protein levels improved after 11 months of treatment with Veopoz (pozelimab). They also had a lower number of hospitalizations and protein transfusions than when they weren't taking the medication. These results suggest that Veopoz (pozelimab) can potentially be helpful for people with CHAPLE disease. People with CHAPLE disease typically need protein transfusions since the genetic condition causes them to lose protein from the body through the gut. In addition, complications of CHAPLE disease include serious and possibly life-threatening blood clots, infections, and malnutrition, which all can lead to hospitalizations.
Yes, Veopoz (pozelimab) is a type of biologic medication. Biologics are medications that are made from living sources (e.g., cells) in the lab. Veopoz (pozelimab) is a monoclonal antibody, which is a lab-made protein that's made from cells. The medication helps block the immune system from being overactive from CHAPLE disease.
Yes, the FDA approved Veopoz (pozelimab) in August 2023. The approval was based on results from a small clinical study of 10 children (ages 3 to 19 years) with CHAPLE disease who took Veopoz (pozelimab) for about 11 months. The clinical evidence showed that the medication can help people with CHAPLE disease and that it's safe to use.
Possibly. In a small clinical study, 2 out of the 10 people with CHAPLE disease who took Veopoz (pozelimab) reported hair loss as a side effect. Contact your provider if you notice hair loss while you're getting treated with Veopoz (pozelimab). Your provider can examine you and recommend treatment options that might be right for you.
Veopoz (pozelimab) doesn't appear to cause weight changes. In a small clinical study, people who took Veopoz (pozelimab) didn't report weight gain or weight loss. Keep in mind that people with CHAPLE disease can have symptoms like weight loss, low appetite, vomiting, and diarrhea. Speak with your provider if you have unusual weight changes while you're taking Veopoz (pozelimab).
No, you'll need to go to your provider's office, a clinic, or an infusion center to get your Veopoz (pozelimab) injections. Veopoz (pozelimab) is prepared and injected by a healthcare provider. The medication is given as an infusion through the vein (IV) for the first dose, then given as an injection under the skin for the rest of your doses. Ask your healthcare provider whether it's possible at some point to arrange for a provider to come to your house to administer the medication (also called home health).
It's not completely clear whether Veopoz (pozelimab) is safe to take if you're pregnant or breastfeeding because it hasn't been well-studied. But it's known that antibody medications like Veopoz (pozelimab) can get passed to babies through the placenta during pregnancy and through breast milk when nursing. It's not known what effects the medication will have on your pregnancy, your unborn baby, your milk supply, or your breastfed baby. Talk to your provider if you're pregnant, thinking of getting pregnant, or breastfeeding while you're getting treatment with Veopoz (pozelimab). Your provider can discuss the risks and benefits of continuing the medication.
CHAPLE disease is a very rare genetic condition. It's an autoimmune disease, meaning that the body's immune system becomes overactive and starts attacking itself. People with CHAPLE disease lose proteins, including antibodies that help fight infection, through the gut. Symptoms can include stomach pain, stomach upset, diarrhea, loss of appetite, and abnormal growth. Additionally, complications of CHAPLE disease include serious and possibly life-threatening blood clots, infections, and malnutrition, which can lead to hospitalizations. Veopoz (pozelimab) is the first FDA-approved treatment for CHAPLE disease. It works by lowering the activity of the immune system.
Veopoz (pozelimab) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Veopoz (pozelimab) can affect the body's ability to fight off infections. This can raise the risk for serious infections, especially meningitis but also pneumonia and Hib disease. Before you start Veopoz (pozelimab), you must be up-to-date on your meningitis vaccinations to help lower your risk for serious infection. Your provider will check whether you need any vaccinations before and during treatment with Veopoz (pozelimab). Your provider will also routinely examine you for infections. Let your provider know right away if you notice any signs of infection like fever, chills, body aches, cough, neck pain or stiffness, or severe headache.
Serious allergic reactions to medications like Veopoz (pozelimab) are possible, including life-threatening reactions like anaphylaxis (e.g., closing of the throat). Get medical attention right away if you notice dizziness, nausea, a choking sensation, trouble breathing, swelling around your eyes, or swelling of the lips or tongue after you get your Veopoz (pozelimab) injection.
Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Veopoz (pozelimab) will not be safe for you to take.