UK – NICE recommends Ultomiris for NHS use in patients with rare blood disorders

Final guidance published yesterday has recommended Ultomiris (ravulizumab) as a treatment for paroxysmal nocturnal haemoglobinuria (PNH), while draft guidance also published this week has accepted the therapy for use in atypical haemolytic uraemic syndrome (aHUS).

PNH is a rare blood condition, wherein red blood cells are destroyed which can result in blood clots and impaired bone marrow function.

According to Johns Hopkins Medicine, the condition affects between 1-1.5 million people per million of the population, with the median age of diagnosis being 34-50 years old.

aHUS is an ultra-rare and life-threatening disease that causes progressive injury to vital organs – primarily the kidneys – via damage to the walls of blood vessels and blood clots…