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The oral drug is iptacopan (trade name Fabhalta). It was found to improve hemoglobin levels and reduce the need for transfusions in adults with PNH.
Iptacopan is a complement factor B inhibitor, which means that it binds to factor B of the alternative complement pathway, preventing hemolysis. In trials it was found to be a better treatment than anti-C5 therapy.
Side effects are mostly minor, such as cold symptoms, diarrhea, or rashes, but there's some risk of infections.
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