Amryt Pharma, announced the publication of a long-term extension study evaluating the benefits of Lojuxta (lomitapide) in the treatment of Homozygous Familial Hypercholesterolaemia.

Amryt Pharma, announced the publication of a long-term extension study evaluating the benefits of Lojuxta (lomitapide) in the treatment of Homozygous Familial Hypercholesterolaemia ("HoFH"), a rare, genetic, life-threatening disease, which impairs the body's ability to remove LDL cholesterol from the blood. Lojuxta is an approved treatment for adult patients with HoFH, which was in-licenced by Amryt in December 2016.

The study, 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. Notably, the majority of patients achieved the recommended LDL-C target level for all adult patients with HoFH. The study was published by Dirk Blom et al and analysed data in 19 HoFH patients who had previously completed the pivotal phase III study (Cuchel et al) in lomitapide. The primary end-point of the study was LDL-C lowering versus the start of the study when receiving standard therapy, assessed at Week 126 (2.4 years) with safety being assessed to the study end at a maximum of 5.7 years. The median treatment duration with lomitapide was 5.1 years (range, 2.1 - 5.7 years). Of the 19 patients, 14 or 74% were able to achieve the recommended LDL-C target levels on at least one occasion of less than 100mg/dL and 11 or 58% of patients achieved the more stringent target of less than 70 mg/dL, recommended if they have cardiovascular disease.

The efficacy was maintained with a mean reduction in LDL-C of 45.5% versus baseline and no new safety signals were identified in the 5.7 years of treatment. The most common adverse events reported were gastrointestinal but the incidence of treatment related adverse events was lower in the extension study than those observed in the pivotal phase 3 trial (42.1% versus 84.2%).

See- "Long-Term Efficacy and Safety of the Microsomal Triglyceride Transfer Protein Inhibitor Lomitapide in Patients With Homozygous Familial Hypercholesterolemia"- Dirk J. Blom, Maurizio R. Averna, Emma A. Meagher, Hendrik du Toit Theron. et al.,Circulation. 2017;136:332-335, originally published July 17, 2017. https://doi.org/10.1161/CIRCULATIONAHA.117.028208.