Alexion Pharmaceuticals reports that the European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion, recommending marketing authorization for ULTOMIRIS® (ravulizumab), the first and only long-acting C5 complement inhibitor administered every eight weeks.
The recommended indication is for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) with haemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with SOLIRIS® (eculizumab) for at least the past six months.
PNH is a severe, complement-mediated ultra-rare disease that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body and result in organ damage and premature death.