Ultomiris approved in the EU for children and adolescents with paroxysmal nocturnal haemoglobinuria

AstraZeneca

3 September 2021 - Approval based on interim results from Phase 3 trial demonstrating complete terminal complement inhibition with reduced dosing frequency compared to Soliris.

Ultomiris (ravulizumab) has been approved in the European Union for expanded use to include children (with a body weight of 10 kg or above) and adolescents with paroxysmal nocturnal haemoglobinuria, an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause thrombosis and result in organ damage and potentially premature death.

Read AstraZeneca press release

Michael Wonder

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Michael Wonder

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Outcome , Medicine , Europe , Paediatrics