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(Alliance News) - AstraZeneca PLC on Monday announced its Ultomiris treatment has been recommended for approval in the EU for use on children and adolescents suffering from a rare blood disorder.
The Committee for Medicinal Products for Human Use of the European Medicines Agency based its positive opinion on interim results from a phase three clinical trial on children and adolescents with Paroxysmal Nocturnal Haemoglobinuria.
The trial showed "an established efficacy and safety profile with reduced treatment burden for children with PNH and their families".
Ultomiris had no reported treatment-related severe adverse events and was "effective in achieving complete C5 complement inhibition through 26 weeks" for the treatment of children and adolescents up to 18 years of age, Astra noted.
Paroxysmal nocturnal hemoglobinuria is an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause a wide range of symptoms, including 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," commented Alexion incoming Chief Executive Marc Dunoyer.
"As we listen to the patient community and understand the challenges of living with a rare disease, we recognise the importance of continuing to deliver options and formulations that enhance patient care and disease management."
On Wednesday last week, AstraZeneca sealed its acquisition of biotech firm Alexion Pharmaceuticals Inc in a cash-and-stock deal worth about USD39 billion.
Shares in AstraZeneca were trading down 0.5% at 8,434.00 pence each early Monday morning in London.
By Will Paige; willpaige@alliancenews.com
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