Novartis has received the US Food and Drug Administration (FDA) accelerated approval for Fabhalta (iptacopan) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN), a rare, progressive kidney disease.
Fabhalta targets the alternative complement pathway in the immune system, which, when overactivated in the kidneys, is believed to support IgAN.
The approval is based on an interim analysis from the Phase 3 APPLAUSE-IgAN study.
APPLAUSE-IgAN is an ongoing trial investigating the efficacy and safety of twice-daily oral Fabhalta compared to placebo in adult IgAN patients.
The primary endpoint of the interim analysis was defined as the percent reduction in proteinuria, measured by comparing the urine protein-to-creatinine ratio (UPCR) at nine months to baseline.
According to the results, iptacopan achieved a 44% reduction in proteinuria from baseline, whereas the placebo group saw only a 9% reduction, at nine months.
It resulted in a statistically significant and clinically meaningful 38% reduction compared to placebo.
Additionally, Fabhalta demonstrated a favourable safety profile, aligning with previously reported data.
Continued approval of Fabhalta may depend on confirming and detailing its clinical benefits through the ongoing Phase 3 APPLAUSE-IgAN study.
Results from this estimated glomerular filtration rate (eGFR) evaluation are anticipated upon study completion in 2025 and will be used to support traditional FDA approval.
Novartis US president Victor Bultó said: “Today’s approval of Fabhalta as a first-in-class medicine for IgA nephropathy is an important milestone in our journey to evolve rare renal disease care by bringing new treatments to people in urgent need of options.
“We are deeply committed to those living with rare renal diseases and look forward to continued partnership with this community as we further advance our broad portfolio.”
Novartis is also developing two additional IgAN therapies including atrasentan and zigakibart, in late-stage trials.
In addition to IgAN, iptacopan is being tested for several other rare diseases, including C3 glomerulopathy (C3G), atypical haemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN).
The Swiss drugmaker is planning to submit Fabhalta for FDA and European Medicines Agency (EMA) approval for C3G by the end of the year.
In May, Novartis reported positive results from the Phase 3 APPEAR-C3G trial of Fabhalta in adult patients with C3G.
