Fabhalta for PNH – pro
Fabhalta is a complement inhibitor that works by treating hemolytic anemia and Reducing the risk of thrombosis (blood clots), which was treated with we\afarfine and then Eliquis in the past. It is FDA indicated for PNH.
The alternatives are reasonable, but older than the newest drug, that has the above-described advantages and is now FDA approved.
Peffault de Latour R, Röth A, Kulasekararaj AG, Han B, Scheinberg P, Maciejewski JP, Ueda Y, de Castro CM, Di Bona E, Fu R, Zhang L, Griffin M, Langemeijer SMC, Panse J, Schrezenmeier H, Barcellini W, Mauad VAQ, Schafhausen P, Tavitian S, Beggiato E, Chew LP, Gaya A, Huang WH, Jang JH, Kitawaki T, Kutlar A, Notaro R, Pullarkat V, Schubert J, Terriou L, Uchiyama M, Wong Lee Lee L, Yap ES, Sicre de Fontbrune F, Marano L, Alashkar F, Gandhi S, Trikha R, Yang C, Liu H, Kelly RJ, Höchsmann B, Kerloeguen C, Banerjee P, Levitch R, Kumar R, Wang Z, Thorburn C, Maitra S, Li S, Verles A, Dahlke M, Risitano AM. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2024 Mar 14;390(11):994-1008.
Régis Peffault de Latour, M.D., Alexander Röth, M.D., Austin G. Kulasekararaj, M.D., Bing Han, M.D., Phillip Scheinberg, M.D., Jaroslaw P. Maciejewski, M.D., Yasutaka Ueda, M.D., +45, and Antonio M. Risitano, M.D.Author Info & Affiliations, Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med 2024;390:994-1008
Fabhalta Prescribing Information 2024