Apellis Pharmaceuticals has secured the US Food and Drug Administration (FDA) approval for Empaveli (pegcetacoplan) to treat two rare kidney diseases.
Empaveli is a targeted C3 therapy designed to regulate the excessive activation of the complement cascade, which is linked to the progression of several serious diseases.
The drug is approved as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged twelve years and above.
It is indicated for reducing proteinuria, a key concern in C3G and IC-MPGN, which are rare kidney diseases that can lead to kidney failure.
In addition to C3G and IC-MPGN, Empaveli is sanctioned for treating paroxysmal nocturnal haemoglobinuria (PNH) in the US, European Union (EU), and other countries.
Apellis co-founder and CEO Cedric Francois said: “Empaveli has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options.
“As Apellis’ third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients’ lives.
“We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.”
The FDA’s approval of Empaveli is based on positive results from Phase 3 VALIANT study, a randomised, placebo-controlled, double-blinded, multi-centre clinical trial.
The Phase 3 study evaluated the efficacy and safety of Empaveli in 124 patients aged twelve and older with C3G or primary IC-MPGN.
Participants received either Empaveli or a placebo twice weekly for twenty-six weeks, followed by an open-label phase where all patients received Empaveli.
The primary endpoint of the study was the log-transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.
Empaveli showed a 68% reduction in proteinuria, stabilisation of kidney function, and significant clearance of C3 deposits as measured by C3 staining, compared to placebo.
The study results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, including those with post-transplant disease recurrence.
Empaveli-treated patients showed stabilisation of kidney function compared to placebo, as measured by estimated glomerular filtration rate (eGFR).
Also, 71% of Empaveli-treated patients achieved zero C3 staining intensity, indicating complete clearance of C3 deposits.
The safety profile of Empaveli was well-established across all approved indications.
Common adverse reactions in the VALIANT study included infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea.
Apellis is launching the ApellisAssist programme to support patients by offering a comprehensive support system.
The initiative includes assistance with insurance coverage, financial aid for eligible patients, disease education, and ongoing product support.
VALIANT study lead principal investigator Carla Nester said: “I’m excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN.
“With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant.
“Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.”
