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Paroxysmal nocturnal haemoglobinuria
The European Commission (EC) approved Ultomiris for children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
In a Phase III study pegcetacoplan resulted in 86 percent of treatment-naïve paroxysmal nocturnal haemoglobinuria (PNH) patients achieving haemoglobin stabilisation.
NICE has recommended that Ultomiris (ravulizumab) be available on the NHS for patients with paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome.
On the NICE’s recommendation, ravulizumab will be made available to hundreds of patients with paroxysmal nocturnal haemoglobinuria on the UK’s NHS.
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