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Pegcetacoplan (Empaveli)
EMPAVELI was approved by the FDA in July 2025 to treat adults and children (12 years and older) with two rare kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). It works by reducing protein in the urine (proteinuria).
Medically reviewed by Dr. Kenar D. Jhaveri, MD
How does EMPAVELI work?
EMPAVELI is also the only therapy for these conditions that targets complement protein C3 and its fragment C3b. In C3G and IC-MPGN, the complement pathway can become overactive, and instead of attacking only infection, the complement pathway also attacks and damages kidney cells. EMPAVELI works by blocking the complement pathway and stopping the disease and damage to the kidney.
Cómo es EMPAVELI administered?
It is given under the skin twice a week using either the EMPAVELI injector or an infusion pump(subcutaneous). The device to do this is provided by the company. Most patients take it twice a week (about every 3–4 days). It can be given using either:
- El EMPAVELI Injector (a handheld device that delivers the dose), or
- An infusion pump (a small machine that slowly pushes the medicine under the skin).
The injection is usually given in areas like the stomach (abdomen), thighs, or upper arms. Your healthcare provider will train you (or a caregiver) on how to prepare and give the injections at home.
Is EMPAVELI right for me?
You and your doctor can decide together whether this medication is right for you. If you have a kidney condition called C3G or IC-MPGN and you have protein leaking in the urine, this medication may be ideal for you. In people with C3G and IC-MPGN who have significant protein in their urine, this medication can help to further reduce or lower proteinuria. Lower proteinuria is associated with preserving kidney function over time.
*Nota: La decisión de recetar un medicamento es responsabilidad de su médico/proveedor de atención primaria según la evaluación de su afección. Lo anterior está destinado únicamente a fines informativos. Discuta esta información y toda la información sobre drogas/medicamentos con su médico antes de comenzar o suspender cualquier medicamento.
Preguntas frecuentes
EMPAVELI is approved for two rare kidney diseases: C3GN and IC-MPGN. EMPAVELI works by blocking a part of the immune system called complement protein C3, which plays a role in kidney injury. It is approved for adults and children 12 years and older to help lower protein in the urine. The approval was based on the VALIANT clinical trial, the largest study ever done in these conditions. It included 124 patients (69 adults and 55 children over 12), some with their own kidneys and others with disease that returned after a kidney transplant. Patients were randomly assigned to receive EMPAVELI twice a week or a placebo (inactive treatment), while continuing standard kidney medicines such as ACE inhibitors, ARBs, SGLT2 inhibitors, or steroids. After 26 weeks, patients taking EMPAVELI had about a 68% drop in proteinuria, while those on placebo had no improvement. The benefit was seen across age, sex, race, transplant status, and whether or not patients used immunosuppressants. It is not yet known if EMPAVELI is safe or effective in children younger than 12 or in patients with recurrent IC-MPGN after transplant.
Overall, EMPAVELI is well tolerated. EMPAVELI works on your immune system, which may make it harder for your body to fight certain infections. Because of this, you have a higher chance of getting serious infections caused by bacteria such as pneumococcus, meningococcus, and Haemophilus influenzae type B. These infections can become life-threatening very quickly if not treated right away. It is recommended that all patients receive vaccines against these bacteria before starting this drug. Vaccines should be given at least 2 weeks before your first dose. If EMPAVELI must be started right away, you may need to get vaccinated as soon as possible and also take antibiotics for a period of time. In adults and children (12 years and older) with C3G or IC-MPGN, the most common side effects are: Reactions where the medicine is injected (such as redness, pain, or swelling), fever, cold-like symptoms (stuffy or runny nose, sore throat), Flu-like illness, cough, and nausea (feeling sick to your stomach)
The two other agents used and available to treat C3G are iptacopan( FABHALTA) and mycophenolate mofetil(MMF or CELLCEPT).
EMPAVELI (pegcetacoplan) and FABHALTA (iptacopan) are both medicines that target the immune system’s complement pathway, which plays a role in kidney and blood diseases They both are FDA approved for C3G and IC-MPGN treatment. EMPAVELI is an injection given under the skin, usually twice a week. It is approved for adults and children 12 years and older with C3G or primary IC-MPGN. It works by blocking complement protein C3. FABHALTA is taken by mouth as a pill twice a day. It is approved only for adults with C3G, IgA nephropathy (IgAN), or paroxysmal nocturnal hemoglobinuria (PNH). It works by blocking complement factor B, which affects the pathway in a slightly different way. Both medicines increase the risk of serious infections caused by certain bacteria. Patients must be up to date on vaccines before starting treatment, and in some cases may need preventive antibiotics. FABHALTA also requires participation in a safety program (REMS). On the other hand, CELLCEPT is an oral pill that has been used for many years. It works by suppressing the overall immune system, preventing immune cells from multiplying. MMF is not FDA-approved specifically for C3G or IC-MPGN, but doctors often use it “off-label” because it can reduce kidney inflammation. MMF lowers general immunity and can increase risks for viral, bacterial, or fungal infections.
EMPAVELI is FDA-approved for the treatment of C3G and IC-MPGN for ages 12 and above and therefore should be covered by most insurance plans. There are other financial support options that also may be available through the drug company that can help with medication costs.
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