Liver UK is delighted to hear Friday’s announcement from the National Institute for Health and Care Excellence (NICE) that they recommend maralixibat for use within the NHS for treating cholestatic pruritus (severe itch) due to Alagille syndrome in patients two months of age and older.
The announcement follows over five years of campaigning by our Children and Families team. Head of the service, Michelle Wilkins said: “Families living with Alagille Syndrome know how devastating the itch can be – children scratch until they bleed, struggle to sleep, and their daily lives are dominated by discomfort. NICE’s recommendation of maralixibat is an important moment for the UK patient community, offering a potential option for a debilitating condition and recognising the urgent need for better treatment options for families affected.
“I’d like to thank those young people and families who played a part in our campaign especially our patient advocates who took the time and trouble to provide moving testimonies of the realities of living with Alagille’s.
This is a bittersweet day for us because Katie, one of our truly amazing young people who had been part of the campaign from the start and was so eloquent in her description of what it meant to have this condition, passed away in August without knowing that her efforts had been successful. We know her family will be incredibly proud of what she has achieved.”
Patient advocate, Mel, who has also played a key role in the campaign added:
“I’m so, so thrilled that finally — finally! — we have a licensed medication to treat the awful, relentless itch that Alagille syndrome brings to our children. As Otto’s mum, watching him scratch until he’s sore and wake night after night because of the itching is heartbreaking. It isn’t ‘just an itch’ — it can completely take over their lives. After such a long wait, knowing that children like Otto finally have another chance of finding some relief means more than I can put into words. This approval will bring so much hope to Alagille families.”
Full details on NICE guidelines are here .
