Apellis Receives Approval for EMPAVELI® (C3 Inhibitor) to Treat Rare Kidney Diseases
Tuesday, July 29, 2025
Apellis Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 years and older.
C3G and primary IC-MPGN are progressive and often result in kidney failure, typically within five to ten years of diagnosis. Many patients eventually require lifelong dialysis or kidney transplantation. Disease recurrence is also common following transplantation.
EMPAVELI is the first therapy approved to target excessive C3 activation, a key contributor to the inflammation and damage associated with these diseases. It is administered twice weekly and is designed to regulate the complement cascade, part of the body’s immune system.
The safety profile of the treatment is supported by over 2,200 patient-years of data. The most common side effects reported in the VALIANT study were infusion site reactions, fever, nasopharyngitis, flu, cough, and nausea.
EMPAVELI is already approved in several markets for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and is being further explored for other rare conditions.
These rare kidney diseases are estimated to affect around 5,000 people in the United States.
The approval is based on positive results from the Phase 3 VALIANT study, which demonstrated significant improvements across key disease markers. EMPAVELI treatment led to a 68% reduction in proteinuria compared with placebo.
In addition, patients showed stabilised kidney function and a marked reduction in C3 deposits. These outcomes were observed across both adolescent and adult patients, including those with post-transplant recurrence.
The VALIANT study involved 124 patients aged 12 years and older and is the largest clinical trial to date in these populations. Participants were randomised to receive either EMPAVELI or a placebo over 26 weeks.
A further open-label phase followed, allowing all patients access to EMPAVELI.
Source: apellis.com
