EU approves new IV formulation of Ultomiris for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.- Alexion Pharma

Alexion Pharmaceuticals announced that the European Commission (EC) has approved the new 100 mg/mL intravenous (IV) formulation of Ultomiris (ravulizumab) for the treatment of two ultra-rare diseases – paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Ultomiris is the first and only long-acting C5 inhibitor administered to patients every eight weeks or every four weeks for pediatric patients less than 20 kg. Ultomiris 100 mg/mL is an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by approximately 60 percent compared to Ultomiris 10 mg/mL, while delivering comparable safety and efficacy. With Ultomiris 100 mg/mL, most patients will spend six hours or less a year receiving treatment. 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 were comparable. Similarly, the data set showed no relevant changes in the efficacy measure of mean lactate dehydrogenase (LDH) levels across the two formulations. The new proposed formulation requires an infusion time of 0.4 to 1.3 hours (25 to 75 minutes) depending on body weight, reducing the infusion time by approximately 60 percent compared with the currently available 10 mg/mL IV formulation, which ranges from 1.3 to 3.3 hours (77 to 194 minutes) depending on body weight.