Novartis' iptacopan meets PhIII test in fight against rare blood disease
Novartis declared a Phase III success for its drug iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria, a rare disorder in which flawed red blood cells are attacked by the immune system, forcing patients to get blood transfusions.
The study, called APPOINT-PNH, showed that patients who got 200 mg of iptacopan twice daily achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks, the company announced in a press release Thursday.
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