AOP Orphan Launches Lojuxta for Treatment of Homozygous Familial Hypercholesterolaemia to the Austrian Market

The Austrian pharmaceutical company AOP Orphan is launching Lojuxta into the Austrian market. Lojuxta was authorized by the EMA (European Medical Agency) in July 2013. Lojuxta is indicated in adult patients with homozygous familial hypercholesterolaemia (HoFH) as an adjunct to a low-fat diet and other lipid-lowering medications, with or without LDL (low density lipoprotein) apheresis. Genetic confirmation of HoFH should be obtained whenever possible, and other forms of primary hyperlipoproteinaemia and secondary causes of hypercholesterolaemia (e.g., nephrotic syndrome, hypothyroidism) must be excluded.

HoFH: A disease of the LDL receptor with an unmet medical need despite current standard of care
HoFH is a rare genetic disorder inherited from both parents, and characterised by significantly elevated low density lipoprotein cholesterol (LDL-C) levels. Chronic exposure to high LDL-C may lead to premature, severe and progressive atherosclerosis, and may correlate with development of premature cardiovascular disease, such as: acute coronary syndrome, early myocardial infarction, aortic stenosis, ultimately premature and sudden death. As HoFH is a rare disease, it is not possible to conduct randomized controlled cardiovascular outcomes studies in this disease and there are no data on the effect of Lojuxta on cardiovascular outcomes.

HoFH patients have LDL receptors that are either non-functional or are defective in their functioning. Most other drug treatments such as statins work by increasing the number of LDL-receptors and if these are defective or negative, the drugs that work by these mechanisms typically have limited effectiveness in HoFH patients.

In addition, regular lipid apheresis (filtering of the lipids from the blood) is often required and must be repeated every 1 to 2 weeks. The use of lipid lowering drugs with or without apheresis, although effective, do not typically reduce LDL-C levels in HoFH patients to the targets recommended by the European Atherosclerosis Society (EAS) and therefore atherosclerosis may continue to progress.