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Novartis Reports Positive 48-Week Results for Fabhalta in PNH Trial

Novartis Pharmaceuticals Corporation has released new 48-week results from the Phase III APPLY-PNH trial, showcasing sustained efficacy and long-term safety of Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH). The trial focused on patients with residual anemia (hemoglobin <10 g/dL) despite prior anti-C5 therapy.

Continuous treatment with Fabhalta (200 mg twice daily) for 48 weeks demonstrated sustained increases in hemoglobin levels to near-normal (≥12 g/dL), avoidance of blood transfusions, and reduced patient-reported fatigue in the majority of patients. Comparable benefits were observed in patients switching from anti-C5 therapy to Fabhalta during the extension period.

Principal co-investigator Antonio Risitano emphasized that the results indicate meaningful hemoglobin improvement over nearly a year, and the benefits can occur within weeks after switching from anti-C5 therapies. These findings reinforce Fabhalta as a promising therapeutic option for individuals with PNH.

In the continuous Fabhalta group, outcomes from the randomized period were maintained at 48 weeks, with a mean hemoglobin level of 12.2 g/dL, 91.9% of patients avoiding transfusions, and improvements in patient-reported fatigue. Similar benefits were observed in the anti-C5-to-Fabhalta group after the switch.

Fabhalta’s safety profile at 48 weeks remained consistent with the 24-week data. Adverse events included three major adverse vascular events (MAVEs), one non-serious transient ischemic attack (TIA), and one serious portal vein thrombosis (PVT). Clinical breakthrough hemolysis (BTH) occurred in six patients receiving continuous Fabhalta and one patient in the anti-C5-to-Fabhalta group. All cases of clinical BTH resolved without changing Fabhalta dosing. COVID-19, headache, and diarrhea were the most frequently reported treatment-emergent adverse events.

Fabhalta was recently FDA-approved for adults with PNH, including both previously treated and treatment-naive patients. The drug’s safety information highlights its impact on the immune system and the increased risk of serious infections caused by encapsulated bacteria.

Novartis plans to present full 48-week results from the Phase III APPOINT-PNH trial in treatment-naïve PNH patients at a congress in 2024. Fabhalta is also being investigated for additional indications in complement-mediated diseases, including immunoglobulin A nephropathy (IgA nephropathy), C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and atypical hemolytic uremic syndrome (aHUS).


Joan David-Leonhard

Joan David Leonhard is a recent Pharm.D graduate with a strong passion for the pharmaceutical industry and a particular interest in pharmaceutical media and communication. Her brief internship experience includes roles in pharmacy where she built strong patient-pharmacist relationships and a pharmaceutical media internship where she actively contributed to drug information articles, blog posts, social media engagement, and various media projects.
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