Benzinga | Sep 3, 2021 08:57AM EDT

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 (NASDAQ:AZN) in July.

* Read Next: Alexion's Ultomiris Shows Compelling, Durable Efficacy In Rare Muscle Weakness Disorder.

* 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.