Discover FABHALTA® (iptacopan) | PNH

Who is FABHALTA for?

How does FABHALTA work?

C5i-experienced (eculizumab and ravulizumab) patients and complement inhibitor–naive patients can share common challenges of PNH

_^.

Image of a man, portrayal of a patient currently on a C5i.

Patients currently on C5is

These patients may^1-5:

Have below-normal Hb: Hb level <12 for women or <13 for men*
Be looking for an option other than infusions
Require ≥1 RBC transfusion per year
Have elevated ARC
Experience fatigue

*Normal Hb levels vary, but generally are between 12-16 g/dL for women and 13-18 g/dL for men.⁵

Image of a woman, newly diagnosed patient portrayal.

Newly diagnosed patients or those not on a complement inhibitor

These patients may^3,4,6:

Have an Hb level <10 g/dL
Require ≥1 RBC transfusion per year
Have elevated ARC and/or LDH levels
Experience fatigue

Oral FABHALTA represents a novel approach to complement inhibition 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 system⁷

Graph illustrating the FABHALTA mechanism of action. — Click on image to enlarge.

TWO DRIVERS OF HEMOLYSIS⁷:

Proximal (upstream)
C3b opsonization drives EVH in the liver and spleen, which can emerge in a proportion of patients treated with a C5i^1,8-14,†
Terminal (downstream)
MAC formation drives IVH, the primary type of hemolysis seen in treatment-naive patients^8,9,15

ONE TREATMENT CONTROLS BOTH DRIVERS⁷

FABHALTA acts proximally in the alternative complement pathway to control both C3b-mediated extravascular hemolysis and terminal complement–mediated intravascular hemolysis.

^†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.^1,8-14

FABHALTA (Fab-HALT-ah) describes a Factor B inhibitor (FAB) of the alternative pathway (ALTA) of the complement system⁷

See how FABHALTA controls both IVH and EVH⁷

Definitions
ARC, absolute reticulocyte count; C5i, C5 inhibitor; EVH, extravascular hemolysis; Hb, hemoglobin; IVH, intravascular hemolysis; LDH, lactate dehydrogenase; MAC, membrane attack complex; PNH, paroxysmal nocturnal hemoglobinuria; RBC, red blood cell.

References
1. 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
2. Peipert JD, Kulasekararaj AG, Gaya A, et al. Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. PLoS One. 2020;15(9):e0237497. doi:10.1371/journal.pone.0237497
3. Shammo J, Gajra A, Patel Y, et al. Low rate of clinically evident extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria treated with a complement C5i: results from a large, multicenter, US real-world study. J Blood Med. 2022;13:425-437. doi:10.2147/JBM.S361863
4. Kulasekararaj AG, Griffin M, Langemeijer S, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022;109:205-214. doi:10.1111/ejh.13783
5. Cappellini MD, Motta I. Anemia in clinical practice—definition and classification: does hemoglobin change with aging? Semin Hematol. 2015;52(4):261-269. doi:10.1053/j.seminhematol.2015.07.006
6. Schrezenmeier H, Roth A, Araten DJ, et al. Baseline characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505-1514. doi:10.1007/s00277-020-04052-z
7. Fabhalta. Prescribing information. Novartis Pharmaceuticals Corp.
8. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811. doi:10.1182/blood-2014-02-522128
9. 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
10. 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
11. 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
12. 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
13. 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.1397010.1111/ejh.13970
14. 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
15. 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

C5i-experienced (eculizumab and ravulizumab) patients and complement inhibitor–naive patients can share common challenges of PNH

Patients currently on C5is

Newly diagnosed patients or those not on a complement inhibitor

Oral FABHALTA represents a novel approach to complement inhibition 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 system⁷

Click on image to enlarge.

FABHALTA (Fab-HALT-ah) describes a Factor B inhibitor (FAB) of the alternative pathway (ALTA) of the complement system⁷

See the efficacy results for FABHALTA

Explore the safety profile for FABHALTA

See how adult patients take FABHALTA

IMPORTANT SAFETY INFORMATION

Definitions and References

C5i-experienced (eculizumab and ravulizumab) patients and complement inhibitor–naive patients can share common challenges of PNH

Patients currently on C5is

Newly diagnosed patients or those not on a complement inhibitor

Oral FABHALTA represents a novel approach to complement inhibition 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 system7

Click on image to enlarge.

FABHALTA (Fab-HALT-ah) describes a Factor B inhibitor (FAB) of the alternative pathway (ALTA) of the complement system7

See the efficacy results for FABHALTA

Explore the safety profile for FABHALTA

See how adult patients take FABHALTA

Definitions and References

FABHALTA is a Factor B inhibitor that acts proximally in the alternative pathway of the complement system⁷

FABHALTA (Fab-HALT-ah) describes a Factor B inhibitor (FAB) of the alternative pathway (ALTA) of the complement system⁷