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.

“We are delighted at the recommendation from NICE, meaning we will be able to offer ravulizumab as a new treatment option to adult patients with PNH across the UK, helping to improve their overall quality of life,” said Sean Richardson, Alexion general manager, UK & Ireland.

“We believe ravulizumab will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the eight-week dosing interval,” he added.

PNH is an ultra-rare blood disorder that can affect men and women with no prior warning and has an average age of onset in the early 30s.

Patients living with PNH experience complement-mediated destruction of red blood cells – haemolysis – and also face risks of blood clots, which can result in organ damage and premature death.

Often, PNH is not immediately recognised, with delays in the diagnosis of this condition ranging from one to over five years.

“Ravulizumab is as effective as the previous standard of care, eculizumab, with a similar safety profile, but with a longer-acting formulation,” said Peter Hillmen, professor of experimental haematology at the University of Leeds and honorary consultant in clinical haematology at Leeds Teaching Hospitals NHS Trust.

“Patients with PNH will now only require infusions every 8 weeks rather than every 2 weeks which will have a major impact on their quality of life,” he added.