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Lumasiran for treating primary hyperoxaluria type 1

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1 Recommendations

1.1 Lumasiran is not recommended, within its marketing authorisation, for treating primary hyperoxaluria type 1 (PH1).

1.2 This recommendation is not intended to affect treatment with lumasiran 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/young people, this decision should be made jointly by the clinician, the child/young person and/or their parents or carers.

Why the committee made these recommendations

PH1 is a rare, inherited condition that can significantly affect the quality of life of people with the condition, and their families and carers. In PH1, the liver produces excess oxalate which combines with calcium in the tissues to form toxic crystals. These crystals can cause recurrent kidney stones, kidney damage and in severe cases kidney failure and multiorgan damage. Standard care includes supportive measures, dialysis and a liver–kidney transplant depending on a person's kidney function.

Clinical trial evidence suggests that, after 6 months of treatment, lumasiran plus standard care reduces a person's oxalate levels compared with standard care alone. The economic model assumes that the probability of having a transplant is higher if a person's plasma oxalate levels are controlled than if they are uncontrolled. Clinical opinion suggests that this does not reflect clinical practice. Therefore, the cost-effectiveness estimates from the model are not appropriate for decision-making. So, lumasiran is not recommended for use.