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Apellis Pharmaceuticals APLS announced that the FDA has accepted its supplemental new drug application (sNDA) seeking the label expansion of Empaveli (pegcetacoplan) for two rare and severe kidney disease indications for review under the Priority Review pathway. The indications are C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
A filing accepted under the regulatory body’s Priority Review pathway reduces the review period to six months from the standard 10 months. This status is awarded to marketing applications for medicines intended to treat serious conditions and that, if approved, would offer a substantial improvement in safety, effectiveness, prevention, or diagnosis of such conditions.
A final decision from the FDA is expected on July 28, 2025.
Please note that Empaveli is approved in the United States for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The drug is also approved in Europe under the brand name Aspaveli for the same indication.
Year to date, shares of Apellis have plunged 28.9% against the industry’s 2.4% growth.
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APLS’ Empaveli sNDA Based on VALIANT Study Data
Apellis’ sNDA submission was based on positive results from the phase III VALIANT study evaluating Empaveli in C3G and IC-MPGN patients.
Per the data readout, the study achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful 68% proteinuria reduction in C3G and IC-MPGN patients treated with Empaveli compared to placebo, both in addition to background therapy, at week 26. The reported results were consistent across all subgroups, including C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys.
Additionally, it was observed that patients treated with the drug experienced stabilized kidney function, as indicated by estimated glomerular filtration rate measurements. A significant number of Empaveli-treated patients demonstrated a decrease in C3c staining intensity, with 71% achieving complete clearance compared to the placebo group.
In the VALIANT study, Empaveli was overall well-tolerated with a consistent safety profile. Adverse events were mostly mild to moderate in severity.
A similar filing seeking the label expansion of the drug to treat C3G and IC-MPGN patients is also currently under review in the EU.
APLS’ Competition in Target Market
Swiss pharma giant, Novartis NVS, is also a formidable player in the market for kidney disease treatments.
NVS’ Fabhalta (iptacopan) obtained approval in the United States and EU in December 2023 and May 2024, respectively, for the treatment of adults with PNH. The drug also received accelerated approval in the United States in August 2024 for reducing the risk of rapid disease progression in proteinuria adults with primary IgA nephropathy.