Swiss pharmaceutical giant Novartis (NYSE: NVS) has secured regulatory approval from the US Food and Drug Administration (FDA) for its first-in-class orphan drug Fabhalta (iptacopan) as an oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH). The approval was granted based on Phase III clinical data that demonstrated a significant improvement in healthy hemoglobin levels without transfusions, increasing from 0% to 67.7% for unresponsive patients switched from anti-C5 treatment to Fabhalta. The transfusion avoidance rate also improved significantly, from 45.7% to 95.2%. A sustained increase in hemoglobin without transfusions was observed in 82.3% and 77.5% of Fabhalta users with and without preceding anti-C5 therapy, respectively.
Novartis plans to launch Fabhalta in the US market this month, while the drug is under review in other regions. The Factor B inhibitor is also in clinical development for other complement-mediated diseases, including immunoglobulin A nephropathy (IgA nephropathy), C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and atypical hemolytic uremic syndrome (aHUS).- Flcube.com
