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 Disease (PNH).

PNH is an extremely rare serious blood disease. Patients may develop blood clots (blood clots), causing organ damage and even premature death. In 2018, Ultomiris was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with PNH. In April 2019, Ultomiris once again obtained the positive review opinions of the European Medicines Agency (EMA) Committee on Medicinal Products for Human Use (CHMP) on the treatment of PNH for the reference of the European Commission.

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. The test results show that Ultomiris can effectively and completely inhibit C5 complement in PNH patients under 18 years of age within 26 weeks. In addition, patients in the Ultomiris trial group did not report serious adverse events related to treatment, and no patients stopped treatment or experienced breakthrough hemolysis during the initial evaluation period (which may cause disabling or fatal thrombosis). The effectiveness and safety characteristics of Ultomiris in children and adolescents are consistent with the performance of the drug in adult PNH clinical trials.

It is worth noting that Soliris is also a star product under Alexion. The drug is one of the world's best-selling rare disease drugs. Its sales in 2020 reached US$4.065 billion. As a long-acting version of the drug, Ultomiris will be released in 2020. Sales have reached 1.077 billion U.S. dollars. In December 2020, AstraZeneca announced the acquisition of rare disease giant Alexion in the form of $39 billion in cash and stock. The transaction was officially completed in July 2021.

Ultomiris was first approved in the European Union in 2019 for the treatment of adult PNH and for the treatment of atypical hemolytic uremic syndrome (aHUS) in adults and children. In June 2021, the US Food and Drug Administration approved the expansion of the use of the drug to cover children (1 month and older) and adolescent patients with PNH. Ultomiris has also become the first treatment for this age group in the United States.

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. At the recommendation of the Independent Data Monitoring Committee (IDMC), Alexion decided to terminate the trial early.