Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, rare, life-threatening hematopoietic stem cell disorder that causes stem cell-derived cells to be vulnerable to complement-mediated lysis and manifests as hemolytic anemia, thrombosis, and peripheral blood cytopenias. C5 inhibitors, eculizumab, and ravulizumab, are recognized as the current standard of care for PNH treatment in countries where they are available. Crovalimab (PiaSky®), which is approved for the treatment of PNH, is a novel anti-C5 inhibitor with an every-4-weeks, low-volume, subcutaneous maintenance dosing regimen with the possibility for self-administration. Data from three phase III studies highlight the overall favorable benefit-risk profile of crovalimab, showing that crovalimab has promising potential to address the unmet medical and socioeconomic challenges in the PNH treatment landscape.
Keywords: Crovalimab; PNH; anti-C5 recycling monoclonal antibody; complement inhibitor; every-4-weeks dosing; paroxysmal nocturnal hemoglobinuria; subcutaneous self-administration.
Paroxysmal nocturnal hemoglobinuria or PNH is a rare, life-threatening blood disorder that leads to the breakdown of red blood cells. People with PNH can have symptoms including tiredness, headaches, less appetite, and difficulty concentrating. Where available, the most common treatment for PNH is a type of medicine called a “C5 inhibitor.” C5 inhibitors block (inhibit) C5, one of the proteins that allow red blood cells to be destroyed. Crovalimab (Piasky®) is a new C5 inhibitor taken once every four weeks, mostly as an injection under the skin (subcutaneous injection) at home or in a healthcare setting. Data from three large studies showed that crovalimab had an overall benefit and might help with some of the challenges that still exist for people with PNH.