Patients with ultra-rare, extreme cholesterol condition given lifeline as breakthrough treatment receives NHS funding

Amryt Pharma announce today that patients in England will be able to access Lojuxta® Black triangle A5 (lomitapide), a clinically effective medicine that has been shown to significantly reduce extreme levels of cholesterol in adults with homozygous familial hypercholesterolaemia (HoFH), when used as an adjunctive therapy to other lipid lowering medications and where available, apheresis.

The decision means that, from this year, patients with HoFH being treated on the NHS in England can be prescribed this ‘first in class’ medicine.

HoFH is a genetic, life limiting disease in which the liver is unable to remove cholesterol in the bloodstream, causing excessive levels of ‘bad cholesterol’ (low density lipoprotein (LDL) cholesterol), to accumulate in the body from before birth.[i]

According to Dr Handrean Soran, Consultant Physician & Endocrinologist, Manchester University Hospital NHS Foundation Trust, “NHS England’s decision is a vital step in ensuring patients with HoFH have access to a new effective treatment option. Lomitapide works differently to other available medicines for this condition. 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.”

  • Current standard of care, which consists of a combination of medicines such as statins, ezetimibe, PCSK9 inhibitors and LDL apheresis has been somewhat effective, but this means that people with HoFH, often still have very high cholesterol levels, and are at a continued elevated risk of life-threatening cardiac events, such as, heart attack, stroke and premature cardiac death.[ii]
  • Lomitapide was assessed alongside approximately 15 other treatments for its clinical benefit, based on published data from clinical trials and in the real world, and its per patient cost. Clinical trials have consistently shown that lomitapide is a clinically effective cholesterol-lowering agent in this tough to treat population. The magnitude of bad cholesterol lowering has been observed to be an average of 40%-50% and in some patients >70% which has not typically be seen with other available modalities for this condition.
  • In the UK, 64 people are currently diagnosed and receiving treatment. It is estimated that around half of those patients will be eligible for treatment with lomitapide on the NHS.

“We are delighted that NHS England has recognised the significant unmet need in the current treatment of HoFH in England and the potential lomitapide has to significantly improve the lives of HoFH patients,” said Joe Wiley, CEO of Amryt Pharma.

Lomitapide has been shown to be an effective adjunctive treatment for adult HoFH patients and has enabled many patients to achieve the recommended target levels of cholesterol for the first time, even stopping apheresis in some cases.”


Lojuxta is indicated as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolaemia (HoFH). Genetic confirmation of HoFH should be obtained whenever possible. Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, hypothyroidism) must be excluded.

This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. for how to report side effects.

Further information can be found on the European Medicines Agency website.[iii]


Amryt Pharma is a specialty biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases. The Company is building a diversified portfolio of commercially attractive, best‐in‐class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available treatments. The Company holds an exclusive licence to sell Lojuxta (lomitapide), across the European Economic Area, Middle East and North Africa, Switzerland, Turkey and Israel.

[i] Horton JD et al. PCSK9: a convertase that coordinates LDL catabolism. Journal of Lipid Research (2009) 50: S172–S177.

[ii] HEART UK (2018), Homozygous Familial Hypercholesterolemia​. Available at Last access: 14th June 2018.

[iii] Lojuxta (lomitapide) capsules European Assessment Report. Available at Last access 1st July 2018.

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