Pharmacological and clinical profile of ravulizumab 100 mg/mL formulation for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

INTRODUCTION: Paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are two rare and severe conditions caused by chronic complement (C') system dysregulation. Treatment with eculizumab, a recombinant, humanized monoclonal antibody against complement C5, changed the natural history of both diseases inducing remission and improving patient outcome. Ravulizumab, a new long-acting next-generation C5 inhibitor has been recently approved for treatment of PNH and aHUS.

AREAS COVERED: Main characteristics of ravulizumab are described: composition, dosing, efficacy and safety profile. Further, an overview of seminal studies and clinical trials using ravulizumab to treat PNH and aHUS in children and adults is detailed. Literature review was performed using the following key words: paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and ravulizumab.

EXPERT OPINION: Ravulizumab profile to treat PNH and aHUS is equivalent to eculizumab in efficacy and safety but allows extended dosing interval to every 4-8 weeks based on patient weight, and requires reduced infusion time. Less travels to infusion centers and medical visits and decreasing job and school absences, significantly increases patient and families' QoL, while reducing cost. Further infusion time is reduced Ravulizumab will possibly become the treatment of choice for patients with PNH and aHUS on chronic C5 inhibition.