AstraZeneca’s Ultomiris approved for use on children in EU

pharmafile | September 3, 2021 | News story | Medical Communications, Research and Development  

AstraZeneca has gained EU approval for its rare blood disorder drug, Ultomiris, for use in children.

The drug was developed as a treatment for paroxysmal nocturnal haemoglobinuria, a severe blood disorder that destroys red blood cells, leading to blood clots, organ damage and sometimes premature death.

The recent EU approval means the drug can be used to treat children with a body weight of 10kg or above and adolescents.

The EC approval was based on positive interim results from AstraZeneca’s Phase III clinical trial in children and adolescents with paroxysmal nocturnal haemoglobinuria.

In a statement AstraZeneca said: “Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period.”

Data from the Phase III trial show the drug was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of patients up to 18 years of age who have the ultra-rare blood disorder.

Ultomiris was recommended for child approval by The Committee for Medicinal Products for Human Use of the European Medicines Agency back in July.

Ultomiris was previously approved in the EU for the treatment of adults with paroxysmal nocturnal haemoglobinuria and is also approved for the treatment of adults and children with atypical haemolytic uraemic syndrome.

Marc Dunoyer, CEO of Alexion, said: “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.”

Kat Jenkins

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