1.1
Lumasiran is recommended, within its marketing authorisation, as an option for treating primary hyperoxaluria type 1 (PH1) in people of all ages. It is recommended only if the company provides lumasiran according to the commercial arrangement.
1 Recommendations
Why the committee made this recommendation
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 impact of PH1 and advanced kidney disease on people's quality of life in the economic model is uncertain. This makes the cost-effectiveness estimates uncertain. Despite this, lumasiran is likely to provide important clinical benefits for people with PH1 and is considered an appropriate use of NHS resources within the context of a highly specialised service. So, lumasiran is recommended for use in the NHS.