AstraZeneca: approval for a treatment for children

(CercleFinance.com) - The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a favourable opinion for the use of Ultomiris in children suffering from paroxysmal nocturnal haemoglobinuria, AstraZeneca announced Monday.


The decision paves the way for the product, developed by AstraZeneca's new rare disease business Alexon, to be marketed, having already received marketing authorisation in adult patients.

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood cell disease that mainly affects young adults and is characterised by fatigue, difficulty swallowing, dark urine and anaemia, amongst other symptoms.

It could also cause severe thrombosis, which may even result in death.


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