NICE (UK): Xenpozyme (olipudase alfa) for treating acid sphingomyelinase deficiency type AB and type B is not recommended – Sanofi

NICE (UK): Olipudase alfa is not recommended, within its marketing authorisation, for treating acid sphingomyelinase deficiency (ASMD; Niemann–Pick disease) in people with type AB or type B.

This recommendation is not intended to affect treatment with olipudase 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. For children or young people, 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 – ASMD (type AB and type B) is a genetic disorder that severely affects the quality of life of people with the condition, and their families and carers. It also increases the risk of death. There is no licensed treatment for the underlying causes of ASMD. Best supportive care, such as improving nutrition and breathing, and treating infection, aims to manage the symptoms. Clinical trial evidence shows that, 1 year after starting treatment with olipudase alfa, lung function is improved and the size of the spleen is reduced in adults and children with ASMD. The improvements may continue in the longer term, but become more gradual as the condition stabilises. There are uncertainties in the economic model. And even when taking into account the substantial effect of olipudase alfa on quality and length of life, the cost-effectiveness estimates are higher than what NICE usually considers an acceptable use of NHS resources for highly specialised technologies. So, olipudase alfa is not recommended.