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AstraZeneca’s Ultomiris approved in the EU

Marc Dunoyer

Ultomiris (ravulizumab) has been approved in the European Union for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH), 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 and potentially premature death.

The approval by the European Commission was based on interim results from the Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients.

“This approval of Ultomiris reflects our ongoing commitment to delivering new treatments that can make a meaningful difference in patients’ lives. Ultomiris has become the standard of care for the treatment of adults with PNH and we will make it available to this younger patient population as soon as possible,” says Marc Dunoyer, Chief Executive Officer, Alexion.

Ultomiris

Ultomiris has an established safety and efficacy profile, offering reduced dosing frequency compared to Soliris (eculizumab), which is the current standard of care in the EU for the treatment of children and adolescents with PNH. Ultomiris is administered every four or eight weeks (depending on body weight), following a loading dose. The approval follows the recommendation of the Committee for Medicinal Products for Human Use of the European Medicines Agency in July 2021.

Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the US Food and Drug Administration approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.

Photo of Marc Dunoyer: AstraZeneca