FDA Grants Priority Review for EMPAVELI® in Treating Rare Kidney Diseases
Waltham, MA – April 1, 2025 – Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review status to the supplemental New Drug Application (sNDA) for EMPAVELI® (pegcetacoplan). This application covers the treatment of two rare, severe kidney diseases: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The FDA’s Prescription Drug User Fee Act (PDUFA) target action date is set for July 28, 2025.
EMPAVELI® Shows Promising Results in VALIANT Phase 3 Study
The Priority Review was granted based on positive data from the VALIANT Phase 3 clinical trial, which evaluated pegcetacoplan’s safety and efficacy in 124 patients aged 12 and older with C3G or primary IC-MPGN. This randomized, placebo-controlled, double-blind, multicenter trial is the largest of its kind in this patient population and included both adolescent and adult participants, as well as patients with native and transplanted kidneys.
Key Efficacy Outcomes
- Proteinuria Reduction: EMPAVELI® met the trial’s primary endpoint, showing a statistically significant 68% reduction in proteinuria compared to placebo (p < 0.0001).
- Kidney Function Stabilization: Patients receiving EMPAVELI® demonstrated stabilization of kidney function as measured by estimated glomerular filtration rate (eGFR) with a nominal p-value of 0.03.
- Reduction in C3c Deposits: There was a substantial decrease in C3c staining intensity—a key disease activity marker—with 71% of EMPAVELI®-treated patients achieving complete clearance compared to placebo (nominal p < 0.0001).
Additionally, EMPAVELI® proved to have a favorable safety and tolerability profile consistent with previous findings.
Clinical Significance of the VALIANT Study Findings
Dr. Bradley P. Dixon, a VALIANT study co-investigator and professor at the University of Colorado School of Medicine, highlighted the importance of these results: “EMPAVELI® demonstrated clinically meaningful benefits by significantly reducing proteinuria, stabilizing kidney function, and clearing C3c deposits. Given the high risk of kidney failure progression in C3G and IC-MPGN, this disease-modifying therapy could be life-changing for patients if approved.”
Understanding C3 Glomerulopathy and Primary IC-MPGN
C3G and primary IC-MPGN are rare, progressive kidney diseases marked by abnormal deposits of complement protein C3c in the kidneys. These deposits lead to inflammation and damage, often resulting in kidney failure. Nearly 50% of affected patients require kidney transplantation or dialysis within five to ten years following diagnosis. Unfortunately, disease recurrence is common, with about 90% of kidney transplant recipients experiencing relapse.
In the U.S., these disorders affect an estimated 5,000 people, with prevalence reaching approximately 8,000 in Europe.
About Pegcetacoplan: Targeted Complement Inhibition
Pegcetacoplan is a targeted complement component 3 (C3) inhibitor designed to regulate excessive activation of the complement cascade—a part of the immune system implicated in several serious diseases. By modulating this pathway, pegcetacoplan addresses the underlying cause of diseases such as C3G and IC-MPGN.
The drug is already approved under the brand names EMPAVELI® and Aspaveli® for treating paroxysmal nocturnal hemoglobinuria (PNH) in the United States, Europe, and other countries. Beyond nephrology, pegcetacoplan is being studied for multiple rare disorders in hematology and nephrology.
Apellis and Sobi Partnership
Apellis holds exclusive commercialization rights for systemic pegcetacoplan in the U.S. and worldwide rights for ophthalmological uses, including geographic atrophy. Meanwhile, Sobi has exclusive rights to commercialize systemic pegcetacoplan outside the United States, with both companies jointly responsible for global development.
Apellis Pharmaceuticals: Company Overview
Apellis Pharmaceuticals is a global biopharmaceutical company committed to pioneering novel complement-targeted therapies for serious diseases. The company introduced the first new class of complement medicines in 15 years, including FDA-approved treatments targeting C3 for conditions such as geographic atrophy, a major cause of blindness worldwide. Apellis continues to explore the potential of complement inhibition to address multiple life-threatening conditions.
Forward-Looking Statements
This announcement contains forward-looking statements regarding the anticipated regulatory timelines, clinical development plans, and potential benefits of EMPAVELI® for treating C3G and primary IC-MPGN. Actual outcomes may differ due to risks and uncertainties, including FDA review decisions and clinical trial results. Apellis disclaims any obligation to update these statements in light of new information or future events.
Media Contact:
Lissa Pavluk
[email protected] | 617-977-6764
Investor Relations:
Meredith Kaya
[email protected] | 617-599-8178
References:
- National Institute of Health – Genetics Home Reference
- Tarragón B, et al. Clinical Journal of the American Society of Nephrology, 2024
- Apellis data on file based on literature consensus