FDA

Empaveli OK’d for rare kidney disorders

August 4, 2025

FDA approved Empaveli (pegcetacoplan), a complement inhibitor, for the treatment of adult and pediatric patients ≥12 years of age with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria.

Empevali is also indicated for the treatment of paroxysmal nocturnal hemoglobinuria in adults.

Efficacy

Approval of the new indications was based on positive six-month results from the VALIANT study (NCT05067127) involving 124 adult and pediatric patients ≥12 of age and weighing ≥30 kg with biopsy-proven, native kidney or, post-transplant recurrent C3G, or native kidney primary IC-MPGN, eGFR ≥30 mL/min/1.73 m², proteinuria ≥1 g/day, and urine protein-to-creatinine ratio (UPCR) ≥1 g/g. For at least 12 weeks before randomization and throughout the 26-week placebo-controlled period, patients were required to be on stable and optimized doses of ACE inhibitors, ARBs and/or SGLT2 inhibitors. Immunosuppressant medication doses (e.g., steroids no higher than 20 mg daily, mycophenolate mofetil, tacrolimus) had to be stable for at least 12 weeks before randomization and throughout the 26-week placebo-controlled period.

Study results demonstrated benefits across key disease markers:

• Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in Empaveli-treated patients vs. placebo.
• Stabilization of kidney function: Empaveli-treated patients achieved stabilization of kidney function compared with placebo (nominal p=0.03) as measured by eGFR.
• Reduction of C3 staining: A majority of Empaveli-treated patients achieved a reduction in C3 staining intensity (nominal p<0.0001) compared with placebo. 71% of Empaveli-treated patients achieved zero C3 staining intensity, indicating complete clearance of C3 deposits.

Safety

The most common adverse reactions in patients with C3G or primary IC-MPGN (≥10%) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea.

Sources:

Empevali (pegcetacoplan) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/215014s011lbl.pdf Revised July 2025. Accessed August 1, 2025.

FDA approves Apellis’ Empaveli® (pegcetacoplan) as the first C3G and primary IC-MPGN treatment for patients 12 and older. [News release]. 2025. https://investors.apellis.com/news-releases/news-release-details/fda-approves-apellis-empavelir-pegcetacoplan-first-c3g-and