Homozygous Familial Hypercholesterolaemia
The Austrian pharmaceutical pioneer AOP Orphan is now bringing Lojuxta® (lomitapide) Capsules to the Austrian market
The Austrian pharmaceutical company AOP Orphan, a European pioneer in the field of rare diseases, 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.
Lojuxta: A chance to get to a new level
Lojuxta was developed by the biopharmaceutical company Aegerion Pharmaceuticals, Inc., which has its corporate headquarters in Cambridge, Massachusetts (USA) and has operations in a number of countries, including several offices in the EU. Lojuxta’s mechanism of action is independent of the LDL receptors and is based on inhibiting the microsomal triglyceride transfer protein that is required to assemble lipid containing lipoproteins and as a consequence reduces the production of these lipoproteins including LDL-C. Lomitapide was shown in a pivotal clinical trial of adult HoFH patients to significantly reduce LDL-C, and, in conjunction with a low-fat diet and other lipid-lowering medications, either with or without LDL apheresis, has the potential to allow some HoFH patients to reach the EAS recommended LDL-C targets previously thought unattainable. Lojuxta is administered once a day in capsule form.
Based on its mechanism of action of blocking the release of lipoproteins from the liver and absorption from the intestines, the most common side effects associated with Lojuxta are gastrointestinal (GI) problems. In order to reduce the occurrence and severity of GI side effects, patients on Lojuxta are required to follow a low-fat diet and dosage should be increased gradually. In addition, Lojuxta can cause elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) and hepatic steatosis (hepatic fat). The long term consequences of the hepatic steatosis associated with Lojuxta treatment are unknown. There is concern that Lojuxta could induce steatohepatitis, which may progress to cirrhosis over several years, although cases of hepatic dysfunction or failure with Lojuxta have not been reported. Clinical data suggest that hepatic fat accumulation is reversible after stopping treatment with Lojuxta, but whether histological sequelae remain is unknown.
Regular monitoring is required for liver effects (specifically, elevations of aminotransferase, steatohepatitis and fibrosis) both prior to and regularly during treatment. The performance of these tests and their interpretation should involve collaboration between the treating physician and the hepatologist. See the Lojuxta prescribing information for complete information on liver monitoring and related treatment recommendations. Treatment with Lojuxta may only be initiated and monitored by a physician experienced in the treatment of lipid disorders.
AOP Orphan are specialists in providing and supporting such comprehensive therapeutic measures and can assist lipid centres in the Central European region with their therapy management through the provision of sup-porting materials for physicians and patients.
Patient organisation pins hopes for HoFH patients on new treatment approach
“As a patient organisation, we welcome the development of new treatments for HoFH,” said Gabriele Hanauer-Mader from the patient organisation FHchol Austria, “as elevations of LDL-C levels can lead to severe and progressive atherosclerosis, with the most severe form of atherosclerosis typically occurring in HoFH patients.”
Under the distribution agreement with Aegerion Pharmaceuticals, AOP Orphan has rights to Lojuxta in Austria, and in several Eastern European countries. AOP Orphan hereby demonstrates its leading role in the treatment of rare diseases.
About AOP Orphan
AOP Orphan was one of the first companies to enter the sector of Rare Diseases in Europe and continues to be a trailblazer in the field of Orphan Diseases to this day. The Orphan Diseases specialist researches, produces and sells innovative solutions, concentrating on three treatment areas: haematology and oncology, cardiology and pulmonology, neurology and psychiatry. AOP Orphan is active at an international level with an export ratio of 70% and is one of the leading players in the Orphan Drug market.
Aegerion Pharmaceuticals is a biopharmaceutical company dedicated to the development and commercialisation of innovative therapies for patients with debilitating rare diseases. Aegerion is listed on the US NASDAQ. Visit
www.aegerion.com for more information.
AOP Orphan Pharmaceuticals AG
T +43 1 503 72 44-24
F +43 1 503 72 44-61
This drug is subject to additional monitoring. This allows for rapid identification of new findings about its safety. Health professionals are encouraged to report any suspected case of an adverse reaction. For instructions on reporting adverse reactions, see section 4.8 of the SPC.