APLS' Empaveli sNDA for 2 Rare Kidney Diseases Gets FDA Priority Tag

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.Image Source: Zacks Investment ResearchAPLS’ Empaveli sNDA Based on VALIANT Study DataApellis’ 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 MarketSwiss 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.Novartis recently announced that the FDA has approved Fabhalta for a third indication, C3G, to reduce proteinuria. Per NVS, this is the first and only treatment approved for this condition. A similar filing by Novartis, seeking label expansion of Fabhalta in the EU to treat C3G, is also currently under review in the EU.Apellis' Empaveli currently competes with NVS’ Fabhalta for the PNH indication, and this competition is expected to intensify if Empaveli receives approval for the C3G indication.Novartis is evaluating Fabhalta in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis and lupus nephritis.Apellis Pharmaceuticals, Inc. Price and Consensus Apellis Pharmaceuticals, Inc. price-consensus-chart | Apellis Pharmaceuticals, Inc. QuoteAPLS’ Zacks Rank & Stocks to ConsiderApellis currently carries a Zacks Rank #3 (Hold).Some better-ranked stocks from the sector are Bayer BAYRY and Corcept Therapeutics CORT, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.In the past 30 days, estimates for Bayer’s earnings per share have increased from $1.14 to $1.19 for 2025. During the same time, earnings per share have increased from $1.23 to $1.28 for 2026. Year to date, shares of Bayer have gained 24%.BAYRY’s earnings matched estimates in two of the trailing three quarters while missing the same on the remaining occasion, the average negative surprise being 19.61%.In the past 30 days, the estimate for Corcept Therapeutics’ 2025 earnings per share has increased from $1.84 to $1.87. The estimate for 2026 earnings per share has increased from $3.05 to $3.11. Year to date, shares of Corcept Therapeutics have gained 82.4%.CORT’s earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 20.08%.5 Stocks Set to DoubleEach was handpicked by a Zacks expert as the #1 favorite stock to gain +100% or more in 2024. While not all picks can be winners, previous recommendations have soared +143.0%, +175.9%, +498.3% and +673.0%.Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.Today, See These 5 Potential Home Runs >>Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Novartis AG (NVS): Free Stock Analysis Report Bayer Aktiengesellschaft (BAYRY): Free Stock Analysis Report Corcept Therapeutics Incorporated (CORT): Free Stock Analysis Report Apellis Pharmaceuticals, Inc. (APLS): Free Stock Analysis ReportThis article originally published on Zacks Investment Research (zacks.com).Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks