Drug efficacy
Evolocumab shows promising results in familial hypercholesterolaemia
Many patients with familial hypercholesterolaemia (FH) fail to achieve recommended levels of low-density lipoprotein (LDL) cholesterol despite pharmacological therapy.
Source: PDBbot / Wikimedia Commons
Evolocumab is a fully human monoclonal antibody to PCSK9 (pictured)
Two studies in The Lancet (online, 1 October 2014) show that evolocumab, a fully human monoclonal antibody to PCSK9, reduced LDL cholesterol significantly more than placebo when added to background lipid-lowering therapy in patients with FH, including those with the rare homozygous form of the disease.
The TESLA Part B study[1] included 49 patients with homozygous FH while RUTHERFORD-2[2] enrolled 331 patients with heterozygous FH. Evolocumab, administered either fortnightly or monthly, yielded rapid reductions in LDL cholesterol and was well tolerated.
Evolocumab has previously demonstrated efficacy in a broad range of statin-treated patients with primary hypercholesterolaemia.
Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2014.20066714
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