The US Food and Drug Administration (FDA) has issued traditional approval to Novartis for Fabhalta (iptacopan), to slow the decline of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression.
This first-in-class complement inhibitor received the approval through a priority review process after its initial accelerated approval in August 2024 for reducing proteinuria in primary IgAN.
Results from the Phase III APPLAUSE-IgAN study supported the traditional FDA approval.
The data indicated that patients receiving Fabhalta experienced a smaller annualised mean decrease in estimated glomerular filtration rate (eGFR) of -3.0 mL/min/1.73m²/yr compared to -5.7 mL/min/1.73m²/yr in the placebo group over two years.
Fabhalta showed consistent benefit over placebo across important kidney health measures.
The safety profile observed in the APPLAUSE-IgAN study aligned with prior findings. The most frequently reported adverse events in treated patients included abdominal pain, dizziness and nausea.
Fabhalta may elevate the risk of serious infections from encapsulated bacteria, so its access in the US requires enrolment in a Risk Evaluation and Mitigation Strategy (REMS) programme with recommended vaccinations before treatment.
Novartis stated that IgAN is recognised as one of the most common autoimmune kidney diseases, with roughly 25 newly diagnosed cases per million people annually worldwide.
Novartis US president Victor Bultó said: “Today’s approval reinforces Fabhalta’s role in preserving kidney function by significantly slowing disease progression, an outcome that matters deeply to patients at risk of long-term kidney damage.
“This milestone underscores the importance of continued innovation for people living with IgAN and our commitment to addressing the underlying drivers of disease.”
Fabhalta is an oral Factor B inhibitor that targets the alternative complement pathway.
Earlier this month, the European Commission granted approval for Novartis’ Itvisma (onasemnogene abeparvovec) as a treatment for children two years and older, teenagers and adults who have 5q spinal muscular atrophy with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.
