GlobalResearch and clinical trials
Oral iptacopan in PNH: final 48-week results of two phase 3 studies published
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
In two phase 3 studies, adults with PNH took oral iptacopan for 48 weeks. The first study had 97 patients and the second had 40. At week 48, an increase in hemoglobin was seen in 86% of patients in the first study and 97% in the second. No one stopped treatment because of side effects or died. The study was supported by the drug's manufacturer.
What does it mean for patients and families?
The effect of this oral drug lasted for a full year. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes. Treatment decisions are made with a doctor.
Source
Scientific paper: Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors. The Lancet. Haematology, 2025. DOI: 10.1016/S2352-3026(25)00081-X · PubMed 40447351
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Paroxysmal nocturnal hemoglobinuria (PNH)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.