pozelimab + cemdisiran

Pozelimab + cemdisiran is an investigational combination therapy for complement-mediated diseases, notably paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG). Pozelimab is a fully human monoclonal antibody that binds to complement component C5, preventing its cleavage and subsequent activation of the terminal complement pathway. Cemdisiran is an N-acetylgalactosamine-conjugated small interfering RNA (siRNA) that targets liver cells to suppress the production of C5 at the mRNA level. Together, these agents provide dual inhibition of C5—pozelimab blocks circulating C5 protein while cemdisiran reduces its synthesis—resulting in more complete and durable suppression of complement activity than either agent alone. The combination can be administered subcutaneously every four weeks and has shown promising efficacy and safety in clinical trials for PNH, with ongoing studies in gMG and other complement-mediated disorders[2][3][4][5][6].

Other names

poze-cemdi