Novartis Challenger To Alexion Advances In Rare Kidney Disease
Ultomiris Head-to-Head Planned
First-in-class iptacopan could help Novartis expand into kidney disease, and take on Alexion’s blockbuster rare disease franchise.
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The Phase III APPOINT-PNH study, testing iptacopan in treatment-naïve PNH patients, met its primary endpoint, positioning the oral drug as a potential new competitor to Soliris and Ultomiris.
Having finally confirmed the Sandoz spin-off, Novartis will become a 'pure play' pharma company and focus on picking bigger blockbuster winners from both internal and external pipelines. CEO Vas Narasimhan talked details at an investor day in Basel.
Phase II data position cemdisiran well for Phase III, but several agents are already well ahead. However, Alnylam foresees a polypharmacy approach.