Irish-listed Amryt Pharma, has announced that a long-term study into the treatment of a rare, genetic, and life-threatening disease using Lojuxta has shown highly positive results.
Homozygous Familial Hypercholesterolaemia ("HoFH") impairs the body's ability to remove bad cholesterol from the blood.
Lojuxta, an approved treatment for adult patients with HoFH, was in-licenced by Amryt, a biopharmaceutical company focused on innovative therapies that transform the lives of patients with rare and orphan diseases, in December 2016.
The study, which followed patients for up to 5.7 years results, showed Lojuxta as being highly effective in lowering LDL cholesterol, also known as bad cholesterol, with acceptable tolerability and no new safety signals.
The majority of patients achieved the recommended bad cholesterol target level for all adult patients with HoFH.
"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," Dr Helen Phillips, head of medical affairs at Amryt, said.
The study was published by Dirk Blom and other researchers, and analysed data in 19 HoFH patients who had previously completed the pivotal phase 3 study in lomitapide.
The primary end-point of the study was bad cholesterol lowering versus the start of the study when receiving standard therapy, assessed at Week 126, with safety being assessed to the study end at a maximum of 5.7 years.
The median treatment duration with Lojuxta was 5.1 years (range, 2.1 - 5.7 years).
Othe 19 patients, 14 or 74pc were able to achieve the recommended bad cholesterol target levels on at least one occasion of less than 100mg/dL.
While 11 or 58pc 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 45.5pc 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 (42.1pc versus 84.2pc).
Earlier this year the company said that it had signed an exclusive licensing agreement with Aegerion Pharmaceuticals in December, which it said could have a transformational effect on the business.