Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.
Régis Peffault de LatourAlexander RöthAustin G KulasekararajBing HanPhillip ScheinbergJaroslaw P MaciejewskiYasutaka UedaCarlos M de CastroEros Di BonaRong FuLi ZhangMorag GriffinSaskia M C LangemeijerJens PanseHubert SchrezenmeierWilma BarcelliniVitor A Q MauadPhilippe SchafhausenSuzanne TavitianEloise BeggiatoLee Ping ChewAnna GayaWei-Han HuangJun Ho JangToshio KitawakiAbdullah KutlarRosario NotaroVinod PullarkatJörg SchubertLouis TerriouMichihiro UchiyamaLily Wong Lee LeeEng-Soo YapFlore Sicre de FontbruneLuana MaranoFerras AlashkarShreyans GandhiRoochi TrikhaChen YangHui LiuRichard J KellyBritta HöchsmannCécile KerloeguenPartha BanerjeeRafael LevitchRakesh KumarZhixin WangChristine ThorburnSamopriyo MaitraShujie LiAurelie VerlesMarion DahlkeAntonio M RisitanoPublished in: The New England journal of medicine (2024)
Iptacopan treatment improved hematologic and clinical outcomes in anti-C5-treated patients with persistent anemia - in whom iptacopan showed superiority to anti-C5 therapy - and in patients who had not received complement inhibitors. (Funded by Novartis; APPLY-PNH ClinicalTrials.gov number, NCT04558918; APPOINT-PNH ClinicalTrials.gov number, NCT04820530.).