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GEORGIOS KEFALAS
The Basel pharmaceutical company announced this on Wednesday. The state of the art therapy is designed to accelerate drug development and testing for serious disease when initial clinical evidence suggests that a drug could represent a significant improvement over an available therapy.
The FDA granted Iptacopan this status based on positive interim results from two ongoing phase II studies. In these, iptacopan showed considerable advantages both in patients who remained anemic and transfusion dependent despite standard therapy for anemia, and as monotherapy in patients with PNH not previously treated with C5.
PNH is a rare and life-threatening blood disorder in which an acquired genetic defect leads to the destruction of red blood cells, particularly by the immune system, the complement system.
