Amryt Pharma has published a long-term extension study of Lojuxta in treating a rare, genetic, life-threatening disease, Homozygous Familial Hypercholesterolaemia (HoFH), which followed patients for up to 5.7 years results, showed Lojuxta as being highly effective in lowering LDL-C levels, with acceptable tolerability and no new safety signals.
HoFH impairs the body’s ability to remove LDL cholesterol (bad cholesterol) from the blood. Lojuxta is an approved treatment for adult patients with HoFH, which was in-licenced by Amryt in December 2016. The majority of patients achieved the recommended LDL-C target level for all adult patients with HoFH, Amryt said.
The study has been published by Circulation, an international, peer-reviewed journal, as “Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholesterolemia”.
Dr Dirk Blom, Department of Lipidology, University of Cape Town, South Africa, said that thestudy confirms the efficacy of lomitapide seen in the pivotal phase 3 trial. He said that adding lomitapide to other lipid-lowering therapies is highly effective in sustainably reducing LDL-C levels with acceptable tolerability and no new safety signals. He also said that a good number of patients achieved their recommended LDL-C target levels. Ho neted that this, before the advent of lomitapide, was thought would be impossible to achieve in this difficult to treat condition. “Some patients were also able to discontinue or reduce the frequency of lipid apheresis – a procedure similar to renal dialysis that removes LDL-C directly from the blood,” Dr Blom said.
Dr Helen Phillips, Head of Medical Affairs at Amryt, said that with all new treatments, especially for those for life-long treatment, is critically important to understand the long-term safety profile of a drug. The data from this long term study, which followed patients over a median of 5.1 years, is reassuring since no new safety signals were seen. She said: “Patients with HoFH have an extremely high risk of cardiovascular events and we are pleased that treatment with Lojuxta, given alongside other lipid-lowering therapies, is so effective in reducing “bad” cholesterol – LDL-C”.