ALXN - New formulation of Alexion's Ultomiris Ok'd in Europe for rare blood disease and kidney disorder

The European Commission has granted marketing authorization to Alexion Pharmaceuticals' (ALXN) Ultomiris (ravulizumab-cwvz) 100 mg/mL intravenous formulation for the treatment of adults with paroxysmal nocturnal hemoglobinuria ((PNH)), a rare blood disease in which the immune system destroys red blood cells and atypical hemolytic uremic syndrome ((aHUS)), is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots to inhibit complement-mediated thrombotic microangiopathy, for adult and pediatric patients.The European Commission approval is based on a comprehensive chemistry, manufacturing and control submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of ULTOMIRIS 10 mg/mL and ULTOMIRIS 100 mg/mL. The data showed no relevant changes in the efficacy measure of mean lactate dehydrogenase ((LDH)) levels across the two formulations.Ultomiris is a long-acting C5 inhibitor for aHUS and is administered every other month for adults and children (20 kg or more) and monthly for children

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New formulation of Alexion's Ultomiris Ok'd in Europe for rare blood disease and kidney disorder