FDA approves first oral monotherapy for paroxysmal nocturnal hemoglobinuria

Iptacopan (Fabhalta) is a complement factor B inhibitor indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). According to a manufacturer press release, approval was based on data from the phase 3 APPLY-PNH trial in adults with PNH and anemia despite prior anti-C5 treatment, and supported by data from the phase 3 APPOINT-PNH study in complement inhibitor-naïve patients.