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EC approves Ultomiris for children and adolescents with PNH
The European Commission (EC) approved Ultomiris for children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
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Paroxysmal nocturnal haemoglobinuria
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EMA’s human medicine committee (CHMP) meeting highlights, July 2021
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
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Pegcetacoplan safe and effective in paroxysmal nocturnal haemoglobinuria trial
In a Phase III study pegcetacoplan resulted in 86 percent of treatment-naïve paroxysmal nocturnal haemoglobinuria (PNH) patients achieving haemoglobin stabilisation.
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UK patients with rare blood disorders receive new treatment option
NICE has recommended that Ultomiris (ravulizumab) be available on the NHS for patients with paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome.
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NICE recommends ravulizumab for treatment of a rare blood disorder
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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Boom times for pharma M&A
Global mergers and acquisitions (M&A) activity last year was expected to decline from 2019 levels, mainly due to the COVID-19 pandemic and resulting uncertainty around the global economy, trade tensions between the US and China, and the presidential election in the US. However, James Baillieu from Bird & Bird reveals…