What Is Empaveli, and Why Does It Matter?
First approved in 2021 for a rare blood disorder, Empaveli (pegcetacoplan) is now approved as the first targeted medicine for adults and children aged 12 years or older with two rare and serious kidney diseases: complement component 3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC‑MPGN). These diseases can cause serious damage to your kidneys and may lead to kidney failure over time. Although rare, these conditions affect about 5,000 people in the U.S. Without effective treatment, many people may eventually need dialysis or a kidney transplant.
In both conditions, a part of your immune system called the complement system becomes overactive. This leads to kidney inflammation and damage. Empaveli helps by blocking this part of the immune system, which may slow down or prevent further kidney damage.
Why Was It Approved?
The FDA approved Empaveli based on positive results in studies for people with C3G and IC-MPGN. It helped lower the amount of protein in the urine, which is a sign of kidney damage. People who took Empaveli had a 68% decrease in protein levels compared to those who received a placebo.
The medicine also helped stabilize kidney function and reduced harmful C3 deposits in the kidneys. Many people had no visible C3 deposits after treatment. These benefits were seen in both teens and adults, including those who had received a kidney transplant.
Like all medicines, Empaveli can cause side effects. The most common ones were reactions at the infusion site, fever, sore throat, flu-like symptoms, cough, and nausea. A few people developed serious infections, such as pneumonia or viral illnesses, especially if they were also taking medicines that weaken the immune system.
What Do I Need to Know?
Empaveli is given as an injection under the skin using either a special infusion pump or a single-use disposable injector. After proper training, you or a caregiver can give the medicine at home.
You can only get Empaveli through a special safety program called Risk Evaluation and Mitigation Strategy (REMS). This is because there is an increased risk of serious infections, especially meningitis and pneumonia. Before starting treatment, you need to be vaccinated against certain bacteria that can cause serious infections, like meningitis and pneumonia. If you haven’t had these vaccines yet, your health care provider may give you antibiotics to help protect you until you’re fully vaccinated.
Even with vaccines, a small risk for infection remains. You’ll need to carry a safety card while using this medicine and for two months after your last dose. Call your health care provider right away if you have symptoms like fever, stiff neck, or confusion, which could be signs of a serious infection.
Empaveli may also be harmful during pregnancy. If you can become pregnant, you should use birth control while taking Empaveli and for 40 days after your last dose.