FDA Approves Novartis Drug for Rare Kidney Disease Treatment

Novartis NVS announced that the FDA has approved Fabhalta (iptacopan) for a third indication.The regulatory body approved the drug for the treatment of adults with C3 glomerulopathy (C3G) to reduce proteinuria.C3G is a progressive and ultra-rare kidney disease that is typically diagnosed in young adults and often progresses to kidney failure.Per NVS, this is the first and only treatment approved for this condition.Data from the phase III APPEAR-C3G study supported the label expansion of the drug for this C3G indication.Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway. Patients have been relying on supportive care, broad immunosuppression and symptom management so far for C3G.Shares of Novartis have risen 20.3% in the past year against the large cap pharmaceutical industry’s decline of 2%.Image Source: Zacks Investment ResearchMore on NVS’ FabhaltaThe APPEAR-C3G study evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G. Treatment with Fabhalta resulted in clinically meaningful proteinuria reduction, which was seen as early as 14 days and sustained at 12 months. In the open-label period, proteinuria reduction was seen in participants who switched to Fabhalta.Fabhalta obtained FDA and European Commission (EC) approval in December 2023 and May 2024, respectively, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (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 (IgAN).Sales in the fourth quarter totaled $57 million, driven by a modest rise in PNH globally and in IgAN in the United States.Last month, Fabhalta received a positive Committee for Medicinal Products for Human Use (CHMP) in C3G by the European Medicines Agency (EMA). Regulatory reviews for this indication are ongoing in China and Japan.A final decision from the EC is expected in two months.Novartis is evaluating Fabhalta in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis and lupus nephritis.Novartis is also advancing the late-stage development of two additional IgAN therapies with highly differentiated mechanisms of action: atrasentan, an investigational oral endothelin A receptor antagonist that received FDA filing acceptance in the second quarter of 2024 and a decision is anticipated in the first half of 2025, and zigakibart, an investigational subcutaneously administered anti-APRIL monoclonal antibody that is currently in phase III development.  NVS Looks to Strengthen PipelineIn January, the company reported better-than-expected fourth-quarter 2024 results, wherein both earnings and revenues beat estimates.Investors were impressed by NVS’ strong quarterly performance and impressive outlook for 2025.Net sales are expected to grow in the mid to high single digits in 2025. Core operating income is expected to grow in high single to low double digits.Label expansion of existing drugs should further fuel sales in the wake of generic competition for Tasigna, Promacta and Entresto. Novartis has a deep pipeline with a number of promising candidates (remibrutinib, ianalumab, pelacarsen, atrasentan and zigakibart).While organic growth continues to drive business, NVS is also focused on strategic bolt-in acquisitions to strengthen its pipeline.Novartis recently announced that it will acquire Anthos Therapeutics, Inc., a Boston-based, privately held, clinical-stage biopharmaceutical company. The acquisition will add abelacimab, a late-stage candidate in development for the prevention of stroke and systemic embolism in patients with atrial fibrillation.Novartis will make an upfront payment of $925 million upon closing of the transaction and potential additional payments of up to $2.15 billion upon achievement of specified regulatory and sales milestones.NVS Rank and Other Stocks to ConsiderNovartis currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the pharma/biotech sector are Bayer BAYRY and Gilead Sciences GILD, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.BayerIn the past 30 days, Bayer’s earnings per share (EPS) estimate for 2025 has increased to $1.20 from $1.14. During the same timeframe, the figure for 2026 has increased to $1.28 from $1.23. Shares of BAYRY have surged 33.8% in the past three months.Gilead SciencesIn the past 30 days, Gilead Sciences’ EPS estimate for 2025 has increased from $7.80 to $7.87. During the same timeframe, the figure for 2026 has increased to $8.27 from $8.12.GILD’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 19.47%. Only $1 to See All Zacks' Buys and SellsWe're not kidding.Several years ago, we shocked our members by offering them 30-day access to all our picks for the total sum of only $1. No obligation to spend another cent.Thousands have taken advantage of this opportunity. Thousands did not - they thought there must be a catch. Yes, we do have a reason. We want you to get acquainted with our portfolio services like Surprise Trader, Stocks Under $10, Technology Innovators,and more, that closed 256 positions with double- and triple-digit gains in 2024 alone.See Stocks Now >>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 Gilead Sciences, Inc. (GILD): Free Stock Analysis Report Bayer Aktiengesellschaft (BAYRY): 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