AstraZeneca's Ultomiris Scores European Nod For Rare Blood Disorder In Kids, Adolescents

The European Commission has approved Alexion's Ultomiris (ravulizumab) paroxysmal nocturnal hemoglobinuria (PNH) in children (with a bodyweight of 10 kg or above) and adolescents. Alexion became a part of AstraZeneca Plc AZN in July.
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PNH is an ultra-rare and severe blood disorder characterized by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage.
The approval was based on interim results from the Phase 3 trial that demonstrated Ultomiris effectively achieved complete C5 complement inhibition through 26 weeks for the treatment.
Ultomiris was first approved in the EU in 2019 to treat adults with PNH and in the EU to treat adults and children with the atypical hemolytic uraemic syndrome (aHUS).
In June, the FDA expanded the use of Ultomiris to include children (one month of age and older) and adolescents with PNH.
Also Read: AstraZeneca's Alexion Stops Ultomiris Trial In Amyotrophic Lateral Sclerosis Patients.
Price Action: AZN stock closed 1.48% higher at $59.59 on Thursday.