Swiss pharm giant Novartis (NOVN: VX) has announced positive results from the extension period of the pivotal Phase III APPLY-PNH trial of oral monotherapy Fabhalta (iptacopan), which last week won approval as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) from the US Food and Drug Administration.
The Phase III study evaluated Fabhalta in adults with PNH who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy. The data were released at the 65th American Society of Hematology Annual Meeting & Exposition (ASH).
Continuous Fabhalta treatment (200mg 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 majority of patients; comparable benefits emerged in those patients switching from anti-C5 therapy to Fabhalta in the extension.
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