Waltham, Massachusetts, July 28, 2025 — The United States Food and Drug Administration has officially approved Empaveli, also known as pegcetacoplan. This innovative treatment was developed by Apellis Pharmaceuticals. It is now the first approved therapy for two rare and serious kidney diseases. These are C3 glomerulopathy, also called C3G, and primary immune complex membranoproliferative glomerulonephritis, also known as IC-MPGN. The approval applies to patients who are 12 years of age and older.
What Is the Significance?
C3G and IC-MPGN are life-threatening kidney conditions that cause severe inflammation and damage due to the overactivation of the immune system’s complement pathway. These diseases can lead to kidney failure within five to ten years of diagnosis. An estimated 5,000 people in the United States and up to 8,000 in Europe are affected. Until now, there were no FDA-approved treatments.
With Empaveli’s approval, patients now have a new option to delay disease progression and improve their quality of life.
What Does Empaveli Do?
Empaveli works by targeting and regulating the C3 protein, which plays a key role in the immune system. In people with C3G and IC-MPGN, this protein becomes overactive, leading to kidney inflammation and damage. By controlling this process, Empaveli helps protect the kidneys.
Evidence From the VALIANT Study
The approval is based on results from the Phase 3 VALIANT trial, which included 124 patients aged 12 or older. This was the largest clinical trial conducted for these diseases. It was also the only one to include both children and adults, with or without kidney transplants.
Key outcomes at the end of 26 weeks included:
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A 68% average reduction in proteinuria, the main sign of kidney damage.
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Stabilized kidney function, measured by eGFR levels.
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Clearance of harmful C3 deposits from kidney tissue. In fact, 71% of treated patients showed complete removal of these deposits.
The trial also showed that Empaveli worked consistently well across different age groups and disease types.
Safety and Side Effects
The safety profile of Empaveli is well documented, with over 2,200 patient years of data. Common side effects include:
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Infusion site reactions
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Fever
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Sore throat
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Cough
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Flu-like symptoms
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Nausea
Three serious infections were reported in the treatment group, but none were linked to encapsulated bacteria. One death due to COVID-19 pneumonia occurred but was unrelated to the drug.
Because Empaveli increases the risk of infections caused by encapsulated bacteria, all patients must receive required vaccinations before starting the treatment. The drug is only available under a restricted FDA program called EMPAVELI REMS to ensure patient safety.
Statements From Experts
Dr. Carla Nester, the lead investigator for the VALIANT study and a pediatric nephrologist at the University of Iowa, called the approval “a pivotal moment in the treatment of rare kidney diseases.” She emphasized the urgent need for options, especially in children, who often face long-term dialysis or kidney transplants.
Cedric Francois, CEO of Apellis, stated, “This approval highlights our commitment to finding solutions for patients with limited treatment options. Empaveli shows what is possible when we target the complement system in a precise and effective way.”
Josh Tarnoff, CEO of NephCure, an organization focused on rare kidney conditions, praised Apellis for bringing hope to young patients and their families.
Access and Support
Apellis has launched the ApellisAssist program to help patients get started with Empaveli. This service provides:
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Help with insurance and reimbursement
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Financial aid for eligible patients
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Disease education
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Ongoing support throughout the treatment
Patients and healthcare providers can call 1-888-273-5547 for assistance.
How Empaveli Will Impact the Pharma Industry and Companies Going Forward
Empaveli’s approval is expected to drive new interest and investments in rare disease research. The success of this C3-targeted therapy shows that it is possible to treat complex and previously untreatable conditions with precision-based approaches. As a result:
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More companies may prioritize complement system research, opening doors to treatments for other rare and chronic conditions
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Biopharma firms could seek accelerated approval pathways for other therapies in small patient populations
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Larger pharmaceutical companies may consider acquisitions or partnerships with smaller biotech firms focused on rare diseases
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Drug developers might invest more in combination trials, targeting the immune system in new ways across nephrology, hematology, and neurology
Overall, Empaveli’s entry into the market sets a new standard for how the pharma industry addresses rare kidney diseases. It also reinforces the value of investing in targeted therapies with strong trial data and real patient outcomes.
What’s Next?
Apellis plans to continue researching Empaveli for other rare kidney diseases. The company already has approvals for its use in paroxysmal nocturnal hemoglobinuria (PNH) in the U.S. and other countries.
It also remains committed to unlocking the broader potential of targeting the C3 protein in other conditions.
Conclusion
The FDA’s approval of Empaveli is a major step forward in the treatment of rare kidney diseases. For the first time, patients with C3G or IC-MPGN have a therapy that addresses the root cause of their disease and offers real hope for better long-term outcomes. This decision also marks a strong example of how targeted science can bring new solutions to patients in need.
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