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 in previous studies. 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.
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.”
Dr Handrean Soran, Consultant Physician & Endocrinologist at Manchester University Hospital NHS Foundation Trust, added: “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. ”