Amryt Pharma heart drug wins approval from NHS England

Drug treats rare condition which can lead to early blocking of arteries and heart attacks

Irish biopharmaceutical group Amryt Pharma’s flagship product Lojuxta has been approved for funding as an NHS England treatment for adults.

Lojuxta is a drug for a rare condition called homozygous familial hypercholesterolaemia (HoFH), which can lead to early onset blocking of arteries and to heart attacks.

The decision means that, from this year, patients with the life-threatening genetic condition being treated on the NHS in England can be prescribed the medicine, which is able to reduce the production and uptake of bad cholesterol.

The clinical value of Lojuxta in managing adult HoFH has been demonstrated in clinical trials and in the real world.


It delivers significant reductions in bad cholesterol, enabling patients to reach target levels of cholesterol that they have not otherwise been able to reach.

Amryt said the outcome was “a vital step” in helping adults with HoFH across England, who are in urgent need of alternative treatment options, to receive a better standard of care.

The company said the drug had 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.

Amryt Pharma chief executive Joe Wiley said the move was another step on the road as the company looks to make the drug available in more territories.

“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,” he said.

“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 and endocrinologist at Manchester University Hospital NHS Foundation Trust, said the decision was “a vital step” in ensuring patients with HoFH have access to a “new effective treatment option”.

He said it lowered cholesterol in adults with HoFH by switching off the release of bad cholesterol from the liver and reducing 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,” he said.

Furthermore, it can “substantially reduce” the 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,” he added.

Colin Gleeson

Colin Gleeson

Colin Gleeson is an Irish Times reporter