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

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Alexion’s long-acting C5 complement inhibitor 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yesterday has recommended Ultomiris (ravulizumab) as a treatment for paroxysmal nocturnal haemoglobinuria (PNH), while draft guidance also published this week has accepted the therapy for use in atypical haemolytic uraemic syndrome (aHUS).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.

In many cases, the prognosis for people living with aHUS is poor, reflecting the importance for timely and accurate diagnoses as well as treatment.

NICE has approved Ultomiris for the treatment of aHUS and PNH patients with a body weight of 10kg or more who are complement inhibitor treatment-naive or have received Alexion’s Soliris (eculizumab) for at least three months and have evidence of response to this therapy.

Clinical trial evidence has shown that treatment with Ultomiris in people with PNH is at least as clinically effective as Soliris and it is administered less frequently – every eight weeks as opposed to every two weeks.

Ultomiris has also been found to cause fewer episodes of breakthrough haemolysis – a symptom of PNH – thereby reducing the number of hospital admissions and need for blood transfusions.

“[Ultomiris] is also cost saving for the NHS in both patient populations and is therefore considered an effective use of NHS resources, and is recommended for routine use on the NHS,” NICE said in a statement.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, commented: “Living with a rare blood condition can be both physically and mentally challenging, especially when frequent treatment is required."

“We are pleased to be able to recommend [Ultomiris] for people who have paroxysmal nocturnal haemoglobinuria or atypical haemolytic uraemic syndrome. We are hopeful that the increased time between doses with this new treatment will lead to a better quality of life for these individuals and their loved ones,” he added.

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