Research & Development
Alexion Pharmaceuticals' supplemental biologics licence application for Ultomiris priority review approved by US FDA
24 June 2019 -

US-based Alexion Pharmaceuticals' supplemental biologics licence application (sBLA) for priority review of Ultomiris has been approved by the US Food and Drug Administration (FDA), it was reported on Friday.

The product is intended to treat people with aHUS, a disease of the red blood cells, and inhibition of complement-mediated thrombotic microangiopathy. It is said to be the first and only long-acting C5 inhibitor administered every eight weeks and it works by inhibiting the C5 protein in the terminal complement cascade.

Alexion Pharmaceuticals has submitted the sBLA based on results of the phase three study of Ultomiris that achieved its primary objective in complement inhibitor-naive patients with Atypical HUS. The global, multicentre, single arm and phase three study evaluated the safety and efficacy of Ultomiris administered by intravenous infusion in 56 adults who had not been treated with a complement inhibitor before. The trial included up to seven-day screening period, a 26-week initial evaluation period and an extension period of up to two years, which is still ongoing.



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