Apellis Pharmaceuticals has announced US FDA acceptance of the supplemental new drug application (sNDA) and the granting of priority review status to Empaveli (pegcetacoplan) to treat the rare and serious kidney conditions 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 28 July 2025.
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The application is based on the positive outcomes of the Phase III VALIANT trial at week 26. The trial assessed the safety and efficacy of the therapy in 124 subjects aged 12 and above with C3G or primary IC-MPGN.
Findings from the trial were consistent across adolescents and adult subjects, and those with native and post-transplant kidney conditions.
The primary endpoint was met, with therapy-treated subjects showing a statistically significant 68% decrease in proteinuria against placebo.
The therapy also demonstrated a favourable tolerability and safety profile, in line with its established safety profile.
C3G and primary IC-MPGN both cause kidney failure.
Apellis Pharmaceuticals co-founder and CEO Cedric Francois stated: “This milestone represents a significant step toward our goal of bringing Empaveli to people living with C3G and primary IC-MPGN, regardless of their disease type, age or transplant status.
“We look forward to working with the FDA to make this treatment available to patients in need as quickly as possible.”
The therapy is currently being investigated for rare diseases in haematology and nephrology, and has already gained approval for treating paroxysmal nocturnal haemoglobinuria (PNH) as Empaveli/Aspaveli in the US, the European Union and other nations.
Along with Swedish biopharma company Sobi, Apellis has worldwide co-development rights for systemic pegcetacoplan.
While Sobi holds exclusive commercialisation rights excluding the US, Apellis retains exclusive commercialisation rights in the US for systemic pegcetacoplan and global rights for ophthalmological pegcetacoplan, inclusive of geographic atrophy.
In April 2023, Apellis obtained validation for its four marketing applications for intravitreal pegcetacoplan from regulatory authorities in the UK, Canada, Australia and Switzerland.
