Novartis Reveals Sustained Efficacy and Safety of Fabhalta in Patients Switching from Anti-C5 Therapies for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Efficacy and Safety:
Fabhalta demonstrated sustained efficacy and long-term safety in adults with PNH, particularly in those switching from anti-C5 therapies24.
Clinical Trials:
The Phase III APPLY-PNH trial showed that patients who switched to Fabhalta from anti-C5 therapies achieved outcomes comparable to those who were on Fabhalta from the start, including near-normal hemoglobin levels and transfusion avoidance24.
Hemoglobin Improvement:
At 24 weeks, 82.3% of anti-C5-experienced patients on Fabhalta achieved a sustained increase in hemoglobin levels ≥2 g/dL without transfusions, compared to 2.0% for those on anti-C5 therapies1.
Transfusion Avoidance:
Fabhalta patients had a transfusion avoidance rate of 94.8% compared to 25.9% for those on anti-C5 therapies1.
Long-term Safety:
The safety profile of Fabhalta at 48 weeks was consistent with the 24-week data, indicating long-term safety24.
Switching Therapies:
Patients switching from eculizumab should start Fabhalta within 1 week, and those switching from ravulizumab should start within 6 weeks to minimize the risk of hemolysis35.
Discontinuation Risks:
Abrupt discontinuation of Fabhalta increases the risk of hemolysis, and patients should be closely monitored for at least 2 weeks after stopping treatment35.
Sources:
1. https://www.novartis.com/news/media-releases/novartis-fabhalta-iptacopan-receives-positive-chmp-opinion-first-oral-monotherapy-adult-patients-paroxysmal-nocturnal-hemoglobinuria-pnh
2. https://www.novartis.com/us-en/news/media-releases/novartis-presents-new-48-week-results-from-phase-iii-apply-pnh-trial-showing-sustained-efficacy-and-long-term-safety-fabhalta-iptacopan-adults-paroxysmal-nocturnal-hemoglobinuria-pnh
3. https://www.rarediseaseadvisor.com/cch/fabhalta-for-the-treatment-of-paroxysmal-nocturnal-hemoglobinuria/
4. https://www.novartis.com/news/media-releases/novartis-presents-new-48-week-results-from-phase-iii-apply-pnh-trial-showing-sustained-efficacy-and-long-term-safety-fabhalta-iptacopan-adults-paroxysmal-nocturnal-hemoglobinuria-pnh