PNH is a rare, life-threatening hematologic disease caused by an acquired mutation in the PIG-A gene. This mutation results in red blood cells lacking GPI-anchored proteins (CD55 and CD59), which leads to complement-mediated hemolysis. C5, a key protein in the complement system, drives intravascular hemolysis (IVH), resulting in the hallmark symptoms and effects of PNH, such as1:
Difficulty breathing
Hemoglobinuria
Thrombosis
Chronic Kidney Disease
C5 Inhibitors as the Established Standard of Care in PNH2
The Role of C5 Inhibitors in Treating PNH Today
C5 inhibitors have been the standard of care for >15 years, with an established and tolerable treatment profile.²˒³ By targeting C5, and blocking a critical step in the complement cascade,³ these drugs have shown a proven reduction of thrombotic risk and improved survival.⁴⁻⁶
Why are people with PNH unsatisfied with their current treatments?
Current C5 inhibitor infusions are required every 2 or 8 weeks.⁸
Some of these infusions may last hours including monitoring time.⁸˒⁹
Patient Feedback on Subcutaneous Administration of PiaSky
C5 = complement protein 5; GPI = glycosylphosphatidylinositol; PIG-A = phosphatidylinositol glycan anchor biosynthesis class A; PNH = paroxysmal nocturnal hemoglobinuria.
Additional Resources
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PiaSky® Efficacy
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References
- Hill A, et al. Nat Rev Dis Primers. 2017;3:17028
- Bektas M, et al. J Manag Care Spec Pharm. 2020;26:S14–S20
- Waheed A, et al. Blood Reviews. 2024;64:101158
- Kelly RJ, et al. Blood. 2011;117:6786–6792.
- Kulasekararaj A, et al. Ann Hematol. 2025;104:81–94
- Terriou L, et al. Eur J Haematol. 2023;111:796–804
- Kaiser K, et al. Patient Prefer Adherence. 2020;14:705–715
- UltomirisTM (ravulizumab-cvwz) Prescribing Information. Boston MA: Alexion Pharmaceuticals, Inc; 2024.