NICE Recommends Ravulizumab for treating PNH in Adults
20th April 2021
We are delighted that NICE recommended Ravulizumab “as an option for treating paroxysmal nocturnal haemoglobinuria in adults:
• with haemolysis with clinical symptoms suggesting high disease activity, or
• whose disease is clinically stable after having eculizumab for at least 6 months”.
If you would like to read the Final Appraisal document please see here.
Like Soliris, Ravulizumab is also a monoclonal antibody which blocks the complement part of the immune system (also by attaching to C5). It is delivered by 8 weekly infusion into the vein on an ongoing basis.
PNH Support collected survey data from 54 patients and 20 carers on unmet need, quality of life and the advantages and disadvantages of ravlizumab for the purpose of this appraisal process. The Aplastic Anaemia Trust also relied on this data for their submission to NICE.
“NICE’s decision to recommend ravulizumab is a game changer for PNH patients, their families and carers. Our community will immediately feel and see the benefits of having the option of an eight-weekly infusion. This level of independence from our lifelong chronic disease will enable us to live a life less impacted by the physiological and psychological burdens we all have to endure.“
Alex Naylor, Patient Expert and Trustee, PNH Support.
The PNH National Service is currently making arrangements for all eligible patients to be moved from eculizumab to ravulizumab.