Phase III study of Ultomiris shows efficacy and safety in paroxysmal nocturnal hemoglobinuria

Alexion Pharmaceuticals announced the presentation of new data demonstrating that Ultomiris (ravulizumab) provided consistent efficacy and safety through 52 weeks in the extension of the Phase III study of Ultomiris and Soliris (eculizumab) in complement inhibitor-naïve, adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Sustained efficacy of Ultomiris was observed on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase (LDH) levels and the secondary endpoints of LDH level reduction and breakthrough hemolysis (BTH). All patients in the initial Ultomiris group of the extension study maintained complete C5 inhibition through 52 weeks, and no patient experienced BTH associated with incomplete C5 inhibition. All patients who had experienced incomplete C5 inhibition while receiving Soliris in the first 26 weeks achieved complete C5 inhibition after the switch to Ultomiris in the extension phase. No patient experienced BTH associated with incomplete C5 inhibition between weeks 27 and 52 after switching to Ultomiris compared to six percent while receiving Soliris in the first 26 weeks.

In an additional sub-study, nearly all patients preferred Ultomiris over Soliris. The most common adverse events occurred less frequently in the extension phase than during the initial treatment phase where the safety profile of Ultomiris was consistent with that of Soliris. The data will be presented at the Annual Congress of the European Hematology Association.