Whether another PNH treatment option will be born is gaining attention in Korea.

According to industry sources, Novartis Korea has submitted a reimbursement application for its oral paroxysmal nocturnal hemoglobinuria (PNH) treatment Fabhalta (iptacopan).

PNH is a rare disease with an estimated incidence of 1.5 per million people worldwide. 

The treatment of this disease has historically relied on C5 inhibitors. Soliris (eculizumab) was first approved in Korea in 2010 and Ultomiris (ravulizumab) was approved in 2022 for PNH. Both are C5 inhibitors that inhibit the terminal C5 in the alternative pathway of the complement system involved in the body's immunity and are