The US Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.
‘The approval of Ultomiris will change the way that patients with PNH are treated,’ explained Richard Pazdur, MD, Director of the FDA’s Oncology Centre of Excellence, and Acting Director of the Office of Haematology and Oncology Products in the FDA’s Centre for Drug Evaluation and Research.
‘Prior to this approval, the only approved therapy for PNH required treatment every two weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every eight weeks, without compromising efficacy.’
PNH is a rare acquired disorder that leads to the rupture or destruction of red blood cells (hemolysis). Patients with PNH are missing a certain protein that normally protects red blood cells from being destroyed by the patient’s immune system. Patients with PNH have sudden, recurring episodes where red blood cells are prematurely destroyed which may be triggered by stresses on the body, such as infections or physical exertion. During these episodes, the following symptoms may occur: severe anaemia, profound fatigue, shortness of breath, intermittent episodes of dark coloured urine, kidney disease, or recurrent pain. PNH can occur at any age, although it is most often diagnosed in young adulthood.