FDA approves Ultomiris for paroxysmal nocturnal hemoglobinuria.- Alexion Pharma.

The FDA has approved Ultomiris (ravulizumab) injection, from Alexion Pharmaceuticals, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease. Ultomiris uses a novel formulation so patients only need treatment every eight weeks, without compromising efficacy.

Ultomiris is a long-acting complement inhibitor that prevents hemolysis. The efficacy of Ultomiris was studied in a clinical trial of 246 patients who previously had not been treated for PNH (treatment na�ve), who were randomized 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 hemolysis measured by the normalization 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). In addition, Ultomiris was studied in a second clinical trial of 195 patients with PNH who were clinically stable after having been treated with eculizumab for at least the past six months. These patients were randomly selected to be treated with Ultomiris or to continue eculizumab. Ultomiris again demonstrated similar effects to eculizumab (non-inferior) based on several clinical measures including hemolysis and avoiding transfusion. Common side effects reported by patients in clinical trials were headache and upper respiratory infection. Health care providers are advised to use caution when administering Ultomiris to patients with any other systemic infection.