CHMP recommends a new 100 mg/mL intravenous (IV) advanced formulation of Ultomiris.- Alexion Pharma

Alexion Pharmaceuticals, Inc.announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorization in the European Union for a new 100 mg/mL intravenous (IV) advanced formulation of Ultomiris (ravulizumab). Ultomiris is the first and only long-acting C5 inhibitor administered every eight weeks for the treatment of two ultra-rare diseases—paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Ultomiris 100 mg/mL would constitute 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 CHMP positive opinion is based on a comprehensive chemistry, manufacturing and control (CMC) submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of Ultomiris 10 mg/mL and Ultomiris 100mg/mL were comparable. Similarly, the data set showed no notable effect 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 more than half compared with the currently available 10mg/mL IV formulation, which ranges from 1.3 to 3.3 hours (77 to 194 minutes) depending on body weight..