UK – NICE recommends Alexion’s PNH treatment Ultomiris

The National Institute for Health and Care Excellence (NICE) has recommended Alexion’s C5 complement inhibitor Ultomiris for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

Specifically, NICE has backed the use of Ultomiris (ravulizumab) in the NHS for the treatment of PNH in adult patients with haemolysis and clinical symptoms suggesting high disease activity, or for those whose disease is clinically stable after taking Soliris (eculizumab) for six months.

Data from a pivotal Phase III study showed that Ultomiris was non-inferior to Soliris in complement inhibitor treatment-naïve patients with PNH, based on the co-primary endpoints of transfusion avoidance and normalisation of lactate dehydrogenase (LDH) levels…