FDA approves new drug for PNH

[Marlene]

https://ashpublications.org/ashclinicalnews/news/7755/pnh-treatment-receives-fda-approval

[Neil Cuadra]

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.