Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria

In 2 phase 3 trials (n=137), iptacopan, a 1st-in-class oral factor B inhibitor, improved haemoglobin levels (HLs) in patients treated with anti-C5 with persistent anaemia & in patients not previously on complement inhibitor therapy, with ~2/3 attaining normal/near-normal HLs.

SPS commentary:

In the first trial, anti-C5–treated patients were randomised to switch to iptacopan or to continue anti-C5 therapy and 51 of the 60 patients on iptacopan had an increase in haemoglobin of ≥2 g/dl from baseline, and 42 had level of ≥12 g/dl, each without transfusion, whilst none of the 35 anti-C5–treated patients attained the end-point levels. Overall, 59 of the 62 patients who received iptacopan, and 14 of the 35 anti-C5–treated patients, did not require or receive transfusion;

In the second, single-group trial, patients who had not received complement inhibitors and who had LDH levels > 1.5 times upper limit of normal received iptacopan. A total of 31 of 33 patients had an increase in hemoglobin of ≥2 g/dl from baseline without red-cell transfusion.

New England Journal of Medicine