Ravulizumab is a suitable long-term treatment option for patients with paroxysmal nocturnal hemoglobinuria

Abstract

Eculizumab and ravulizumab are approved treatments for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease which can cause potentially fatal complications if left untreated. Long-term ravulizumab treatment in patients with PNH is under investigation in two ongoing studies (‘301’ and ‘302’). This article describes the results at 2 years for both studies. Ravulizumab continued to manage most patients' symptoms and less than 3% of patients experienced serious side effects related to treatment during this time. This article highlights why eculizumab and ravulizumab are the usual treatments for PNH, where available. Long-term, ravulizumab controlled patients' PNH disease activity with few side-effects related to treatment. Clinical Trial Registration: Study 301: NCT02946463 Study 302: NCT03056040