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FABHALTA is the first and only oral monotherapy that can help deliver comprehensive hemolysis control (IVH and EVH)

FABHALTA is a Factor B inhibitor that acts proximally in the alternative pathway of the complement system1

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TWO DRIVERS OF HEMOLYSIS:

 

  1. Proximal (upstream)
    C3b opsonization drives EVH in the liver and spleen, which can emerge in a proportion of patients treated with a C5i2-9,*

  2. Terminal (downstream)
    MAC formation drives IVH, the primary type of hemolysis seen in treatment-naive patients3,5,10

*Based on preliminary studies in patients treated with eculizumab suggesting terminal complement inhibition induces C3 fragment deposition on PNH RBCs. The C3 fragments trigger destruction of the RBCs by macrophages, making EVH a potential consequence of terminal complement inhibition. Additional studies are needed to further understand the impact of terminal complement inhibition on EVH.2-9

 

“I was most excited about what Fabhalta could mean for controlling intravascular and extravascular hemolysis.” – Patient taking Fabhalta. Compensated for their time by Novartis. Individual results may vary.

FABHALTA (Fab-HALT-ah) describes a Factor B inhibitor (FAB) of the alternative pathway (ALTA) of the complement system1

See how FABHALTA controls both IVH and EVH

See the efficacy results for FABHALTA

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Explore the safety profile for FABHALTA

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See how adult patients take FABHALTA
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Definitions
C5i, C5 inhibitor; EVH, extravascular hemolysis; IVH, intravascular hemolysis; MAC, membrane attack complex; PNH, paroxysmal nocturnal hemoglobinuria; RBC, red blood cell.

References
1. Fabhalta. Prescribing information. Novartis Pharmaceuticals Corp.
2. Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101:251-263. doi:10.1007/s00277-021-04715-5
3. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811. doi:10.1182/blood-2014-02-522128
4. Hill A, Rother RP, Arnold L, et al. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica. 2010;95(4):567-573. doi:10.3324/haematol.2009.007229
5. Notaro R, Luzzatto L. Breakthrough hemolysis in PNH with proximal or terminal complement inhibition. N Engl J Med. 2022;387(2):160-166. doi:10.1056/NEJMra2201664
6. Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10(1157):1-24. doi:10.3389/fimmu.2019.01157
7. Risitano AM, Notaro R, Marando L, et al. Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood. 2009;113(17):4094-4100. doi:10.1182/blood-2008-11-189944
8. Shammo J, Kim J, Georget M, Pattipaka T, Fermont JM. P796: Hospitalization in patients with paroxysmal nocturnal hemoglobinuria: a retrospective analysis of observational study data from the United States. Hemasphere. 2023;7(suppl):e22585a2. doi:10.1097/01.HS9.0000970088.22585.a2
9. Versmold K, Alashkar F, Raiser C, et al. Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting. Eur J Haematol. 2023;111(1):84-95. doi:10.1111/ejh.1397010
10. Risitano AM, Frieri C, Urciuoli E, Marano L. The complement alternative pathway in paroxysmal nocturnal hemoglobinuria: from a pathogenic mechanism to a therapeutic target. Immunol Rev. 2023;313(1):262-278. doi:10.1111/imr.1313710.1111/imr.13137