Veopoz (pozelimab-bbfg) is used to treat CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. CHAPLE disease is a rare and life-threatening hereditary immune disease caused by an overactivation of the complement system. Veopoz is a fully human, monoclonal antibody G4 (IgG4) that blocks the activity of complement factor C5 to regulate the complement pathway and relieve the symptoms of CHAPLE disease.

CHAPLE (complement hyperactivation, angiopathic thrombosis, and protein-losing enteropathy) disease is an immune disorder caused by an overactive complement system, which is part of the immune system. Usually, the complement system helps fight microbes and is kept under control by the CD55 gene. In CHAPLE disease, there are mutations in the CD55 gene which means complement activity is unregulated and attacks normal cells. This causes damage to blood and lymph vessels along the upper digestive tract and leads to the loss of circulating proteins. Veopoz blocks complement factor C5 to regulate the complement system.

Veopoz became the first FDA-approved medicine for CD55-deficient protein-losing enteropathy on August 18, 2023.  Approval was based on positive results from the Phase 2/3 open-label trial (NCT04209634), showing improvement in serum albumin concentrations. All 10 patients using Veopoz achieved normalization by Week 12 and maintained serum albumin concentrations within the normal range through at least 72 weeks of treatment. Veopoz is approved for use for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease. 

• Veopoz must be prepared and administered by a healthcare provider. 
• The first dose of Veopoz is given through a vein (I.V. or intravenous infusion), usually over 1 hour or more, depending on your weight. If you have an allergic reaction during your infusion, your healthcare provider may decide to give your infusion more slowly or stop your infusion.
• Your next doses will be given an injection under the skin (subcutaneous injection) in your abdomen, thigh, or upper arm. 
• You will be given your first dose (infusion), the second dose subcutaneously on day 8, then you will have Veopoz subcutaneously weekly.
• After the infusion and your first subcutaneous injection, you will be monitored for 30 minutes for allergic reactions.
• If you miss receiving a Veopoz subcutaneous dose, call your healthcare provider right away to find out when you will receive your next injection.

Veopoz Recommended Dosage:

Day 1 (loading dose): Administer a single 30 mg/kg dose by intravenous infusion after dilution. (2.2)

Day 8 and thereafter (maintenance dosage): Inject 10 mg/kg as a subcutaneous injection once weekly starting on Day 8.

Comments:

• The maintenance dosage may be increased to 12 mg/kg once weekly if there is inadequate clinical response after at least 3 weekly doses (i.e., starting from Week 4).
• The maximum maintenance dosage is 800 mg once weekly. 
• See full prescribing information for meningococcal vaccine and prophylaxis recommendations prior to the first dose of Veopoz and for instructions on preparation and administration.

Dosage forms available: Veopoz is available Injection: 400 mg/2 mL (200 mg/mL) in a single-dose vial.

Do not receive this medicine if you have a meningococcal infection. 

What should I tell my doctor before receiving Veopoz?

Before you receive Veopoz, tell your healthcare provider about all of your medical conditions, including if you have an infection or fever.

It is important that you:

• Have all recommended vaccinations before you start treatment.
• Receive antibiotics if you start Veopoz within 2 weeks of receiving meningococcal vaccination.
• Stay up to date with all recommended vaccinations during treatment with Veopoz.
• Tell your healthcare provider if you take Intravenous Immunoglobulin (IVIg).

Pregnancy

Tell your healthcare provider if you are pregnant, become pregnant, or plan to become pregnant. It is not known if Veopoz will harm your unborn baby.

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if Veopoz passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with Veopoz.

Common Veopoz side effects

• upper respiratory tract infection
• hair loss (alopecia)
• fracture
• raised, red patches of skin that are often very itchy (hives)

Serious Veopoz side effects

Also see Warnings section below.

Allergic (hypersensitivity) reactions. Allergic reactions, including infusion-related reactions, may happen during your intravenous or subcutaneous treatment. Tell your healthcare provider right away if you develop the following symptoms or any other symptoms during your treatment that may mean you are having a serious allergic reaction:

• chest pain
• trouble breathing or shortness of breath
• swelling of your face, tongue, or throat
• feel faint or pass out

Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all of the possible side effects of this medicine Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088

Warnings

Veopoz is a medicine that affects your immune system and so can lower the ability of your immune system to fight infections.

This medicine increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.

You must receive meningococcal vaccines at least 2 weeks before your first dose of this medicine if you have not already had these vaccines.

If you had a meningococcal vaccine in the past, you might need additional vaccination before starting this medicine. Your healthcare provider will decide if you need additional meningococcal vaccination.

If your healthcare provider decided that urgent treatment with Veopoz is needed, and your meningococcal vaccines are not up-to-date, you should receive meningococcal vaccination as soon as possible. You should also receive antibiotics.

Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection:

• headache with nausea or vomiting
• headache and fever
• headache with a stiff neck or stiff back
• fever
• fever and a rash
• confusion
• muscle aches with flu-like symptoms
• eyes sensitive to light

Your healthcare provider will give you a Patient Safety Card about the symptoms of meningococcal or other infections. Carry it with you at all times during treatment and for 3 months after your last dose. Your risk of meningococcal infection may continue for several weeks after your last dose of this medicine. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

Veopoz may also increase the risk of other types of serious bacterial infections.

• People who take Veopoz may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae.
• Certain people may also have an increased risk of bacterial infections, including gonorrhea infection. Talk to your healthcare provider to find out if you are at risk of gonorrhea infection, about gonorrhea prevention, and regular testing.

Call your healthcare provider right away if you have any new signs or symptoms of infection.

Who should not receive Veopoz?

Do not receive this medicine if you have a meningococcal infection. 

What should I tell my doctor before receiving Veopoz?

Before you receive Veopoz, tell your healthcare provider about all of your medical conditions, including if you have an infection or fever.

It is important that you:

• Have all recommended vaccinations before you start treatment.
• Receive antibiotics if you start Veopoz within 2 weeks of receiving meningococcal vaccination.
• Stay up to date with all recommended vaccinations during treatment with Veopoz.
• Tell your healthcare provider if you take Intravenous Immunoglobulin (IVIg).

Pregnancy

Tell your healthcare provider if you are pregnant, become pregnant, or plan to become pregnant. It is not known if Veopoz will harm your unborn baby.

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if Veopoz passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with Veopoz.

How should I receive Veopoz?

• Veopoz must be prepared and administered by a healthcare provider. 
• The first dose of Veopoz is given through a vein (I.V. or intravenous infusion), usually over 1 hour or more, depending on your weight. If you have an allergic reaction during your infusion, your healthcare provider may decide to give your infusion more slowly or stop your infusion.
• Your next doses will be given an injection under the skin (subcutaneous injection) in your abdomen, thigh, or upper arm. 
• You will be given your first dose (infusion), the second dose subcutaneously on day 8, then you will have Veopoz subcutaneously weekly.
• After the infusion and your first subcutaneous injection, you will be monitored for 30 minutes for allergic reactions.
• If you miss receiving a Veopoz subcutaneous dose, call your healthcare provider right away to find out when you will receive your next injection.

Dosing information

Veopoz Recommended Dosage:

Day 1 (loading dose): Administer a single 30 mg/kg dose by intravenous infusion after dilution. (2.2)

Day 8 and thereafter (maintenance dosage): Inject 10 mg/kg as a subcutaneous injection once weekly starting on Day 8.

Comments:

• The maintenance dosage may be increased to 12 mg/kg once weekly if there is inadequate clinical response after at least 3 weekly doses (i.e., starting from Week 4).
• The maximum maintenance dosage is 800 mg once weekly. 
• See full prescribing information for meningococcal vaccine and prophylaxis recommendations prior to the first dose of Veopoz and for instructions on preparation and administration.

Dosage forms available: Veopoz is available Injection: 400 mg/2 mL (200 mg/mL) in a single-dose vial.