Novartis AG has two successful Phase III clinical trials in hand for its targeted factor B inhibitor iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH), positioning the drug for a regulatory filing in 2023 and eventually a commercial face off with AstraZeneca PLC's C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab).
The company announced on 8 December that the Phase III APPOINT-PNH study, testing iptacopan in treatment-naïve patients with PNH, was a success
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