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Draft guidance consultation

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1 Recommendations

1.1 Sebelipase alfa is not recommended, within its marketing authorisation, for treating Wolman disease (rapidly progressive lysosomal acid lipase deficiency [LAL-D]) in people who are 2 years or younger when treatment starts.

1.2 This recommendation is not intended to affect treatment with sebelipase alfa that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. This decision should be made jointly by the clinician, the child or young person, and their parents or carers.

Why the committee made these recommendations

Wolman disease is a rare genetic condition that presents in babies and children under 2 years old. It causes a build-up of fat in cells in the liver, heart, blood vessels, and digestive system. Without treatment, the baby or child will not survive. There are no treatments for Wolman disease available in the NHS. Standard care without sebelipase alfa is palliative. Sebelipase alfa is used as an enzyme replacement therapy alongside a restricted diet, and can allow a haematopoietic stem cell transplant to be done, if needed.

Clinical trial evidence suggests that sebelipase alfa increases how long people live. But it is not clear how much longer people will live or how their quality of life compares with people without the condition.

Haematopoietic stem cell transplant is an option for people having sebelipase alfa, those who can no longer have sebelipase alfa, or when sebelipase alfa stops working. But it is not clear when a transplant is done and what proportion of people have this. After a transplant, it is not clear how much the dose of sebelipase alfa can be reduced. It is also not known what dose of sebelipase alfa a person is likely to have over their lifetime.

Because of the clinical uncertainties and uncertainties around the likely treatment pathway when sebelipase alfa is used, the cost-effectiveness estimates are highly uncertain. Even when considering the condition's severity, and the effect of sebelipase alfa on quality and length of life, the most likely cost-effectiveness estimates are much higher than what NICE considers an acceptable use of NHS resources. So, sebelipase alfa is not recommended.