Novartis has announced positive results from its phase 3 APPOINT-PNH trial of oral monotherapy with iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

The results, announced via news release, were presented at the 49th Annual Meeting of the European Society for Blood and Marrow Transplantation in Paris.

“The APPOINT-PNH results are consistent with the tolerability and safety profiles seen in APPLY-PNH and show oral iptacopan controls hemolysis while nearly eliminating the need for blood transfusions,” noted study principal coinvestigator Régis Peffault de Latour, MD, PhD, of Saint-Louis Hospital, Greater Paris University Hospital.

PNH is a rare, chronic, complement-mediated condition characterized by anemia, fatigue, and a need for regular red blood cell transfusions.

Read more about PNH therapies

The APPOINT-PNH trial enrolled 40 adults with PNH who were complement-inhibitor naive (including anti-C5 treatments). They were given 200 mg of oral iptacopan monotherapy twice daily. The results met the primary endpoint of a 2 g/dL or greater increase from baseline in hemoglobin with no need for transfusions of red blood cells following the 24-week core study period.

In terms of secondary outcomes, significant benefits of iptacopan were also observed, including achievement of 12 g/dL or greater hemoglobin levels without the need for transfusions in 62.8% of patients.

Overall, 97.6% of the enrolled patients achieved independence from red blood cell transfusions at 24 weeks of treatment. Furthermore, no breakthrough hemolysis or major adverse vascular events occurred during the study period, and no patient discontinued the therapy.

The patients reported less fatigue, which was reflected in the change from baseline in scores on the Functional Assessment of Chronic Illness Therapy–Fatigue scale. Safety and tolerability were similar to that reported in previous studies.

Novartis will submit the results to regulatory authorities in the first half of 2023 and will have results of their phase III studies on C3 glomerulopathy and immunoglobulin A nephropathy later in the year.


Novartis phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients. News release. Novartis, Inc.; April 26, 2023.