AstraZeneca's 'Ultomiris' gets EU approval for use in children

AstraZeneca said on Monday that ‘Ultomiris’, or ravulizumab, has been recommended for marketing authorisation in the European Union for expanded use, to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).

The FTSE 100 pharmaceuticals giant said the European Medicines Agency based its positive opinion on interim results from the phase 3 clinical trial in children and adolescents with PNH, which were recently presented during the European Hematology Association 2021 Virtual Congress.

It said the trial demonstrated that Ultomiris - developed by its rare diseases division Alexion - was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of children and adolescents up to 18 years of age with PNH.

Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.

The efficacy and safety of Ultomiris in children and adolescents was observed as consistent with the established profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.

AstraZeneca described PNH as an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis or blood clots, which can occur throughout the body, and result in organ damage and potentially premature death.

“This recommendation shows that Ultomiris - which has become the standard of care for the treatment of adults with PNH - has the potential to transform the lives of children and adolescents in Europe suffering from this devastating rare disease,” said Alexion chief executive officer Marc Dunoyer.

“As we listen to the patient community and understand the challenges of living with a rare disease, we recognize the importance of continuing to deliver options and formulations that enhance patient care and disease management.”

At 0812 BST, shares in AstraZeneca were down 0.24% at 8,460p.