New treatment available to patients with rare blood disorders
pharmafile | May 20, 2021 | News story | |
Hundreds of people with rare blood disorders will have access to a new treatment following guidance from NICE today.
The treatment, ravulizumab (also branded as Ultomiris by Alexion Pharmaceuticals), has been recommended as a treatment for paroxysmal nocturnal haemoglobinuria (PNH), a rare blood disorder characterised by an excessive breakdown in red blood cells, which can lead to anaemia, kidney problems, and clots in the blood vessels.
Draft guidance published yesterday also recommends the treatment for atypical haemolytic uraemic syndrome (aHUS), a disorder which causes clots in small blood vessels, which can lead to organ damage and significant kidney impairment, thrombosis, heart failure, and brain injury.
The current treatment option available for people with symptomatic PNH or aHUS on the NHS, also recommended by NICE, is eculizumab, which is delivered intravenously every two weeks.
Clinical trial evidence has shown that in people with PNH, treatment with ravulizumab is at least as clinically effective as eculizumab, and was found to cause fewer episodes of breakthrough haemolysis, a symptom of PNH, reducing the number of hospital admissions and the need for blood transfusions.
Indirect comparisons of ravulizumab with eculizumab for people with aHUS similarly suggest that ravulizumab is as effective for treating aHUS.
The new guidance means that patients with either PNH or aHUS will now be eligible for treatment with ravulizumab, which is delivered every eight weeks. Patient experts stated that the increased between treatments may improve the quality of life for people with the rare blood disorders, increasing their independence and mental health.
Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “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 ravulizumab 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.”
Ravulizumab 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.
Around 270 people with PNH and 160 people with aHUS will be eligible for the new treatment.