Press Release: Novartis Fabhalta(R) (iptacopan) meets Phase III primary endpoint, slows kidney function decline in patients with IgA nephropathy (IgAN)

Novartis

103.77 CHF -0.10%

• Charts
• News
• Analysen

Kaufen Verkaufen

-- In APPLAUSE-IgAN final analysis, Fabhalta demonstrated statistically

significant, clinically meaningful improvement in estimated glomerular

filtration rate (eGFR) slope vs. placebo over two years1

-- eGFR is key marker of kidney function; IgAN is progressive autoimmune

kidney disease that leads to kidney failure in many patients1-3

-- Fabhalta is first and only approved complement inhibitor for adults with

IgAN and has potential to delay disease progression4,5

-- Fabhalta received accelerated approval for reduction of proteinuria in

adults with IgAN in US in 2024; data support 2026 submission for

traditional FDA approval4,5

Basel, October 16, 2025 -- Novartis today announced positive final results from APPLAUSE-IgAN, a Phase III study evaluating Fabhalta(R) (iptacopan) in adults living with IgA nephropathy (IgAN). Fabhalta, an oral alternative complement pathway inhibitor, demonstrated statistically significant, clinically meaningful superiority compared to placebo in slowing IgAN progression measured by annualized total slope of estimated glomerular filtration rate (eGFR) decline over two years(1).

"Progressive diseases such as IgAN present an urgent need for interventions that can ultimately improve kidney health. Many people with IgAN commonly experience fear and anxiety of disease progression," said Ruchira Glaser, Development Unit Head, Cardiovascular, Renal & Metabolic, Novartis. "We are excited about today's positive Phase III APPLAUSE-IgAN results showing slowed eGFR decline, which add to the growing evidence of Fabhalta as a targeted therapy to preserve long-term kidney function, giving hope to people living with this condition."

Novartis intends to use these data to support Fabhalta submissions in 2026. Alongside Fabhalta, Novartis continues to advance its multi-asset IgAN portfolio, which also includes Vanrafia(R) (atrasentan) and investigational compound zigakibart.

IgAN is a progressive autoimmune kidney disease with approximately 25 per million people newly diagnosed worldwide each year(3). IgAN is highly debilitating as it leads to glomerular inflammation, proteinuria, and a gradual decline in eGFR(2). Up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring dialysis or kidney transplantation as part of long-term disease management(2,6,7). Furthermore, people living with IgAN often face mental, social, and economic challenges(2,8). Supportive care does not address the underlying causes of the disease and often fails to slow disease progression, reinforcing the need for more targeted therapies for IgAN(3,9).

In APPLAUSE-IgAN, Fabhalta was well tolerated with a favorable safety profile in line with previously reported data(10). Full data from the APPLAUSE-IgAN final analysis will be presented at future medical meetings.

About Fabhalta(R) (iptacopan)

Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway(10).

Discovered at Novartis, Fabhalta received US Food and Drug Administration (FDA) and European Commission (EC) approval in December 2023 and May 2024, respectively, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta also received accelerated approval in the US in August 2024, and in China in September 2025, for the reduction of proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression (generally UPCR >=1.5 g/g(4,5,11,12). In 2025, Fabhalta received FDA and EC approval as well as approvals in China and Japan for the treatment of adults with C3 glomerulopathy (C3G), making it the first treatment approved for this condition(13-15).

Fabhalta is being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN)(16-18). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions(16-18).

About APPLAUSE-IgAN

APPLAUSE-IgAN (NCT04578834) is a Phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of twice-daily oral Fabhalta (200 mg) in 477 adult primary IgAN patients (main study population). Patients were randomized to Fabhalta or placebo, on top of supportive care (a stable dose of maximally-tolerated renin-angiotensin system (RAS) inhibitor therapy with or without a stable dose of SGLT2i)(1).

The two primary endpoints of the study for the interim and final analysis, respectively, are proteinuria reduction at 9 months as measured by UPCR, and the annualized total eGFR slope over 24 months(10,18). During the final analysis, the following secondary endpoints were assessed: proportion of participants reaching UPCR <1 g/g without receiving corticosteroids/immunosuppressants or other newly approved drugs or initiating new background therapy for treatment of IgAN or initiating kidney replacement therapy (KRT), time from randomization to first occurrence of composite kidney failure endpoint event (reaching either sustained >=30% decline in eGFR relative to baseline or sustained eGFR <15 mL/min/1.73 m(2) or maintenance dialysis or receipt of kidney transplant or death from kidney failure), and change from baseline to 9 months in the fatigue scale measured by the Functional Assessment Of Chronic Illness Therapy-Fatigue questionnaire(19).

The main study population enrolled patients with an eGFR >=30 mL/min/1.73 m(2) and UPCR >=1 g/g at baseline(10,18). In addition, a smaller cohort of patients with severe renal impairment (eGFR 20--30 mL/min/1.73 m(2) at baseline) was also enrolled to provide additional information but not contributing to the main efficacy analyses(10,19).

Novartis commitment to kidney diseases

Building on a 40-year legacy that began in transplant, Novartis is on a mission to empower breakthroughs and transform care in kidney health, starting with kidney conditions that have significant unmet need.

Historically, these conditions have had considerably less funding and research, leading to a treatment landscape largely focused on reactive or end-stage disease management, often with significant physical, emotional, and financial burdens. Our portfolio targets the underlying causes of disease, with an aim to protect kidney health and delay or prevent dialysis and/or transplantation. Our goal is to help patients get back to living life on their terms - whether at work, in school, or with loved ones, and by partnering with patients, advocates, clinicians and policymakers, we aim to raise awareness, accelerate diagnosis and get patients the right care, sooner.

Disclaimer

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "potential," "can," "will," "plan," "may," "could," "would," "expect," "anticipate," "look forward," "believe," "committed," "investigational," "pipeline," "launch," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis

Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.

(MORE TO FOLLOW) Dow Jones Newswires

October 16, 2025 01:15 ET (05:15 GMT)