21 December 2018 - The U.S. FDA today approved Ultomiris (ravulizumab) injection for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria, a rare and life-threatening blood disease.

Ultomiris is a long-acting complement inhibitor that prevents haemolysis. The efficacy of Ultomiris was studied in a clinical trial of 246 patients who previously had not been treated for paroxysmal nocturnal haemoglobinuria (PHN) (treatment naïve), who were randomised to be treated with Ultomiris or eculizumab, the current standard of care for PNH. The results of the trial demonstrated that Ultomiris had similar results to eculizumab (non-inferior) – patients did not receive a transfusion and had similar incidence of haemolysis measured by the normalisation of LDH levels in patients’ blood (lactate dehydrogenase, or LDH, is an enzyme required during the process of turning sugar into energy in the body’s cells).

The FDA granted this application priority review designation. Ultomiris also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Read FDA press release