Novartis (NVS) announced Friday positive topline results from a phase 3 study evaluating the efficacy and safety of Fabhalta in adult patients with paroxysmal nocturnal hemoglobinuria who were switched from other therapies.
The company said that after 24 weeks of treatment with twice-daily oral monotherapy of the drug, also called iptacopan, average hemoglobin levels improved compared with the baseline.
Paroxysmal nocturnal hemoglobinuria is a rare, chronic blood disorder that causes the production of red blood cells susceptible to premature destruction, leading to anemia, blood clots, and fatigue among other symptoms.
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