Novartis’ Fabhalta (Iptacopan) Receives FDA Priority Review for IgA Nephropathy (IgAN) Treatment

1. The FDA has granted priority review for Fabhalta (iptacopan), a Novartis drug, to treat IgA nephropathy (IgAN), a rare kidney condition.
2. The Phase 3 study, APPLAUSE-IgAN (NCT04578834), demonstrated clinically meaningful and statistically significant proteinuria reduction in patients with IgAN.
3. The primary endpoints of the study include proteinuria reduction at 9 months and the annualized total estimated glomerular filtration rate (eGFR) slope over 24 months.
4. Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway, currently in development for various rare diseases, including IgAN, C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN).
5. Fabhalta was approved by the FDA in December 2023 for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).
6. IgAN is a progressive, rare kidney disease, with approximately 25 new cases per million people worldwide each year.
7. Up to 30% of people with persistent higher levels of proteinuria (≥1 g/day) may progress to kidney failure.
8. Novartis is committed to developing innovative treatment options for rare kidney diseases, including atrasentan and zigakibart, which are both in Phase III development for IgAN.

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