15 September 2015 - Lomitapide mesylate (trade name Lojuxta) has been approved since July 2013 for use by adults with homozygous familial hypercholesterolaemia available, where a diet and other medicines used to lower cholesterol levels do not provide adequate control. The Institute for Quality and Efficiency in Health Care (IQWiG) has checked in a dossier evaluation whether this drug with respect to the appropriate comparator therapy offers an additional benefit. Because the dossier contains no appropriate data are available, such additional benefits, however, can not be inferred.

Homozygous familial hypercholesterolaemia is a very infrequent, hereditary metabolic disorder in which the blood excessive amount of LDL contains cholesterol (low-density lipoprotein). Standard treatment is diet therapy along with a lipid-lowering medicinal product; if this is not enough, then treatment is hemodialysis (LDL apheresis) in combination with drug therapy.

The Federal Joint Committee (G-BA) has distinguished of this rating between several groups of patients. The distinction depends on whether drug and dietary options have been exhausted and if LDL apheresis is to be used.

The dossier contains but for none of these patient groups data that would be suitable for the evaluation of the added value. Because the manufacturer draws in zoom neither a direct comparative study, yet he leads an indirect comparison. Results from a pre-post comparison from which the manufacturer wants to derive an additional benefit, are unsuitable: Besides the fact that pre-post comparisons already have a low predictive value, the concrete procedure is inadequate here. It therefore follows from the dossier no evidence of an additional benefit of lomitapide mesylate.

For more details, go to: https://www.iqwig.de/de/presse/pressemitteilungen/pressemitteilungen/lomitapid-bei-hypercholesterinamie-kein-anhaltspunkt-fur-zusatznutzen.6899.html?&et_cid=4&et_lid=%25208 [German]