Manufactures report positive outcomes for iptacopan for paroxysmal nocturnal haemoglobinuria

This low-molecular weight complement factor B inhibitor was found to achieve a 60% reduction in lactate dehydrogenase levels (a biomarker of intravascular haemolysis) after 12 weeks treatment. Manufacturers also report a transfusion-free improvement of haemoglobin levels.

PharmaTimes