AstraZeneca Ultomiris receives EU approval for the treatment of PNH in children and adolescents

CompilationFan Dongdong

A few days ago, the European Commission (EC) has approved an extended indication application for AstraZeneca’s Alexion rare disease drug Ultomiris (ravulizumab), which is approved for the treatment of children (weight 10 kg or more) and adolescents with paroxysmal nocturnal hemoglobinuria Syndrome (PNH)

PNH is an extremely rare serious blood disease.

This time, the European Commission’s decision to approve Ultomiris was mainly based on the interim results of a phase III clinical trial conducted on children and adolescents with PNH, which proved the safety and effectiveness of Ultomiris in these patients

In another previous trial, compared with the infusion of Soliris every 2 weeks, the infusion of Ultomiris every 8 weeks achieved non-inferiority in all 11 primary endpoints and all key secondary endpoints

It is worth noting that Soliris is also a star product under Alexion.

Ultomiris was first approved in the EU in 2019 for the treatment of adult PNH and for the treatment of atypical hemolytic uremic syndrome (aHUS) in adults and children

In recent years, the drug has achieved regulatory breakthroughs in multiple indications, but just in August this year, the Phase III CHAMPION-ALS trial of the drug for the treatment of adult amyotrophic lateral sclerosis (ALS) failed to reach the pre-specified interim analysis Clinical endpoint

Reference source: Approval based on interim results from Phase III trial demonstrating complete terminal complement inhibition with reduced dosing frequency compared to Soliris