FDA approves new drug for PNH
 

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.