Ad hoc announcement pursuant to Art. 53 LR Phase III APPEAR-C3G study met its primary endpoint, demonstrating superiority of iptacopan vs. placebo in proteinuria reduction at six-month analysis1; the safety profile of iptacopan was consistent with previously reported data1-3 C3 glomerulopathy (C3G) is an ultra-rare complement-mediated kidney disease, with approximately 50% of patients progressing to kidney failure within 10 years of diagnosis4-7; currently no treatments address the underlying ca
Novartis today announced results from the extension period of the pivotal Phase III APPLY-PNH trial of oral monotherapy Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy1,2. Continuous Fabhalta treatment (200 mg twice daily) for 48 weeks enabled sustained hemoglobin-level increases to near-normal (12 g/dL or more), blood transfusion avoidance, and reduced patient-reported fatigue in the ma
On Nov. 28, the Food and Drug Administration (FDA) announced that it was opening an investigation into the possibility of previously unknown serious safety risks associated with the hottest and most technological group of cancer medicines produced to date. Now, drugmakers including Novartis (NYSE: NVS), Gilead Sciences (NASDAQ: GILD), and Bristol Myers Squibb (NYSE: BMY) are on the hot seat, with their patients' well-being and billions of dollars in revenue on the line. The FDA is concerned about chimeric antigen receptor T-cell (CAR T-cell) therapies.