FDA Grants Traditional Approval to Novartis’ Fabhalta for IgAN
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Novartis has earned full traditional FDA approval for Fabhalta (iptacopan) to slow loss of kidney function in adults with primary IgA nephropathy, backed by successful Phase III APPLAUSE-IgAN trial results demonstrating significant disease modification.
The US Food and Drug Administration (FDA) has officially granted traditional regulatory approval to Novartis for its targeted oral therapy Fabhalta (iptacopan), indicated specifically to slow the progressive decline of kidney function in adult patients diagnosed with primary immunoglobulin A nephropathy (IgAN) who are at elevated risk of disease progression.
This first-in-class complement inhibitor achieved full approval following a priority review evaluation by the regulatory agency, converting its initial accelerated approval granted in August 2024, which was based on preliminary data demonstrating significant reductions in proteinuria in primary IgAN patients.
Phase III APPLAUSE-IgAN Clinical Trial Results
The FDA's traditional approval decision was supported by comprehensive clinical data from the pivotal Phase III APPLAUSE-IgAN clinical trial. Throughout the study, Fabhalta demonstrated statistically significant and sustained clinical benefits over placebo across key primary and secondary measures of renal function and overall kidney health.
The safety and tolerability profile documented during the APPLAUSE-IgAN trial remained consistent with previously reported clinical data. The most frequently observed adverse events among treated patients included mild-to-moderate abdominal pain, dizziness, and nausea.
Because Fabhalta's mechanism targets complement pathways, it can increase patient susceptibility to serious, potentially life-threatening infections caused by encapsulated bacteria. Consequently, prescribing access in the United States requires mandatory enrolment in a dedicated Risk Evaluation and Mitigation Strategy (REMS) program, requiring patients to receive recommended vaccinations prior to initiating therapy.
Disease Impact and Executive Commentary
Novartis highlighted that IgA nephropathy is recognized globally as one of the most prevalent autoimmune kidney disorders, with an estimated global incidence of approximately 25 newly diagnosed cases per million individuals each year.
Victor Bultó, President of Novartis US, commented on the regulatory decision: “Today’s approval reinforces Fabhalta’s role in preserving long-term kidney function by significantly slowing disease progression — a clinical outcome that matters deeply to patients facing the risk of irreversible kidney damage. This regulatory milestone underscores the necessity of continuous therapeutic innovation for individuals living with IgAN and highlights our commitment to targeting the root pathological drivers of autoimmune renal disease.”
Mechanistically, Fabhalta is a targeted, orally administered Factor B inhibitor that selectively acts on the alternative complement pathway, preventing immune-mediated glomerular damage.
In related corporate news, Novartis recently received marketing approval from the European Commission for Itvisma (onasemnogene abeparvovec) as a gene therapy treatment for pediatric patients aged two years and older, adolescents, and adults suffering from 5q spinal muscular atrophy (SMA) carrying a bi-allelic mutation in the SMN1 gene.
