FDA Gives First Approval for Novartis’ Iptacopan, a Revolutionary Oral Treatment for Rare Blood Disorder PNH

December 6, 2023

Novartis has received FDA approval for iptacopan, an innovative oral treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Marketed as Fabhalta, it’s the first oral monotherapy for PNH, a disease affecting 10-20 per million globally. Iptacopan, lauded for its potential as a “pipeline in pill,” is also positioned to treat other complement-mediated diseases like primary immunoglobulin A nephropathy (IgAN). Analysts estimate its peak sales potential at $3.6 billion. Fabhalta, which works in the immune system’s alternate complement pathway, offers advantages over existing treatments, such as ease of oral administration and effectiveness in new and previously treated patients. Its approval was based on phase 3 trials showing significant improvements in hemoglobin levels. However, it comes with a boxed warning for a heightened risk of severe infections and will be distributed through a particular program. Novartis plans to pursue fast-track FDA approval for iptacopan in IgAN in 2024, following promising phase 3 study results.

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[Source: Fierce Pharma, December 6th, 2023]

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