EU approves AstraZeneca blood disorder treatment for children
AstraZeneca
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AstraZeneca said its Ultomiris blood disorder treatment has been approved in the European Union for expanded use to include children and adolescents.
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Pharmaceuticals & Biotechnology
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The drug treats paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage and potentially premature death, AstraZeneca said.
Approval by the European Commission was based on interim results from a Phase III clinical trial in children and adolescents.