Amryt Pharma Plc – NHS England Approves Funding for Lojuxta

9 July 2018

Amryt, a biopharmaceutical company focused on rare and orphan diseases, is pleased to announce that Lojuxta® (lomitapide) has been approved for funding as an NHS treatment for adult patients with Homozygous Familial Hypercholesterolaemia (“HoFH”) in England.


The decision means that, from this year, patients with this ultra-rare, life-threatening genetic condition being treated on the NHS in England can be prescribed a ‘first in class’ medicine able to reduce the production and uptake of low density lipoprotein (LDL) cholesterol, often referred to as ‘bad cholesterol’, when used as an adjunctive therapy to other lipid lowering medications and where available, apheresis.


The clinical value of Lojuxta in managing adult HoFH has been demonstrated in clinical trials and in the real world (see previously announced study results on Amryt’s website, here). Lojuxta delivers significant reductions in LDL cholesterol, enabling patients to reach target levels of cholesterol that they have not otherwise been able to reach. The Company believes that this outcome is a vital step in helping adults with HoFH across England, who are in urgent need of alternative treatment options, receive a better standard of care and has the potential to transform their lives with one simple capsule a day.


The Company expects Lojuxta to be available to HoFH patients treated by NHS England during the fourth quarter of 2018.


Joe Wiley, CEO of Amryt Pharma, commented:

“We are delighted that NHS England has recognised the significant unmet need in the current treatment of HoFH in England and the potential Lojuxta has to significantly improve the lives of HoFH patients. Lojuxta 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.


“This decision today is in line with our strategy to make Lojuxta available to more patients across Amryt’s territories and it is estimated that funding approval will have a positive impact on revenue from 2019 and beyond. We look forward to working with the NHS to make this treatment available to patients in need of this life changing therapy as soon as possible.”

Dr Handrean Soran, Consultant Physician & Endocrinologist at Manchester University Hospital NHS Foundation Trust, added:

“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.”



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About Amryt


Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases.


Lojuxta is an approved treatment for adult patients with the rare cholesterol disorder – Homozygous Familial Hypercholesterolaemia (“HoFH”). This disorder impairs the body’s ability to remove low density lipoprotein (“LDL”) cholesterol (“bad” cholesterol) from the blood, typically leading to abnormally high blood LDL cholesterol levels in the body from before birth – often ten times more than people without HoFH – and subsequent aggressive and premature narrowing and blocking of blood vessels, heart attacks and strokes, even at a very young age if not properly diagnosed or receiving adequate treatment.  Lojuxta is indicated as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without LDL apheresis in adult patients with HoFH.


Amryt holds an exclusive licence to sell Lojuxta (lomitapide) across the European Economic Area, Middle East and North Africa, Switzerland, Turkey, Israel, Russia, the Commonwealth of Independent States and the non-EU Balkan states.


Amryt’s lead drug candidate, AP101, is a potential treatment for Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment.  It is currently in Phase 3 clinical trials. The European and US market opportunity for EB is estimated to be in excess of €1 billion.


Amryt’s earlier stage product AP102 is focused on developing novel, next generation somatostatin analogue (“SSA”) peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need, including acromegaly and Cushing’s disease.


In March 2018, Amryt in-licensed a pre-clinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Recessive Dystrophic Epidermolysis Bullosa, a subset of EB, and is also potentially relevant to other genetic disorders.


For more information on Amryt, please visit

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