Evinacumab alongside diet and other low-density lipoprotein-cholesterol (LDL‑C) lowering therapies is recommended, within its marketing authorisation, as an option for treating homozygous familial hypercholesterolaemia (HoFH) in people 12 years and over. It is only recommended if the company provides it according to the commercial arrangement.
Why the committee made this recommendation
When LDL‑C is not lowered enough by diet and LDL‑C lowering therapies in people with HoFH, lipoprotein apheresis may be added. In adults, lomitapide may also be used but is not licensed for people aged 12 to 17 years.
Clinical trial evidence shows that evinacumab can lower LDL‑C levels when statins and other lipid-lowering therapies have not reduced them enough. There is no data directly comparing evinacumab with lomitapide, and the results of an indirect treatment comparison are uncertain. There is also no long-term evidence on whether evinacumab reduces the risk of cardiovascular death or events such as heart attacks.
There are some uncertainties in the cost-effectiveness evidence comparing evinacumab with lomitapide in adults with HoFH. But, overall, there are cost savings with evinacumab compared with lomitapide. This means that, despite the uncertainties in the evidence, evinacumab is a cost-effective use of NHS resources when compared with lomitapide in adults. The cost-effectiveness evidence in people aged 12 to 17 years is uncertain. Evinacumab costs more than LDL‑C lowering therapies alone. But, because LDL-C lowering therapies have limited effectiveness in people with HoFH, there is a high unmet need in young people for effective treatments. So, evinacumab is recommended for the whole population for which it is licensed.