Novartis: positive phase III data in PNH

( – Novartis reports that in the phase III APPLY-PNH study, its investigational oral monotherapy iptacopan demonstrated clinically significant superiority over anti-C5 treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH ).

‘PNH has a significant unmet need that is not being addressed by anti-C5 therapies; despite treatment with anti-C5, a large proportion of those affected remain anaemic, tired and dependent on blood transfusions’, he underlines.

Detailed results will be presented at an upcoming medical meeting and included as part of global regulatory submissions in 2023. Iptacopan is also being studied in phase II or III trials in other indications.

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