NHS England okays funding for cholesterol-lowering drug

NHS England has okayed funding for Lojuxta, Amryt Pharma’s treatment for adults with an ultra-rare inherited condition causing extreme levels of cholesterol.

Lojuxta (lomitapide) is approved to reduce extreme levels of cholesterol in adults with homozygous familial hypercholesterolaemia (HofH), used as an adjunctive therapy to other lipid lowering medications, and where available apheresis.

HoFH is a genetic, life limiting disease in which the liver is unable to remove cholesterol in the bloodstream, causing excess levels of “bad” low density lipoprotein (LDL) cholesterol to accumulate in the body from before birth.

NHS England assessed Lojuxta alongside around 15 other treatments for its clinical benefit, based on data from clinical trials and in the real world, and its per patient cost.

The magnitude of LDL cholesterol lowering has been observed to be an average of 40%-50%, and can exceed 70%.

Current standard of care consists of a combination of drugs such as statins, ezetimibe, PCSK9 inhibitors and LDL apheresis.

But this means that people with HoFH often still have very high cholesterol levels, and are at continued elevated risk of life-threatening cardiac events such as heart attack, stroke and premature cardiac death.

In the UK, 64 people are currently diagnosed and receiving treatment for the condition, and around half of these are likely to eligible for treatment with lomitapide.

Dr Handrean Soran, consultant physician and endocrinologist, Manchester University Hospital NHS Foundation Trust, said: “Unlike currently available treatments, lomitapide lowers cholesterol in adults with HoFH by switching off the release of ‘bad cholesterol’ from the liver and reduces the uptake from the gut.”

“The treatment has been shown it can enable more than half of adults with this life-threatening condition to finally be able to reach target cholesterol levels, previously thought not to be possible with current available therapies, and to substantially reduce their need for lipoprotein apheresis, a lengthy and cumbersome ‘dialysis for cholesterol’, available only in six specialist centres in the UK. This is very encouraging news for patients, their families and clinicians.”