Update on PCSK9 Inhibitors and New Therapies

There remains an unmet need for patients, many at high CVD risk, who are unable to achieve ‘optimal’ LDL-C targets with statin therapy, or are intolerant to statins. Two new drugs were approved in 2013–2014 both of which inhibit production of LDL, or its precursor very (V)LDL; mipomersen, an apolipoprotein B antisense agent, and lomitapide, an inhibitor of microsomal triglyceride transport protein.9 However their use is strictly limited to the rare orphan population with homozygous familial hypercholesterolemia (HoFH) with prescribing controlled by a Risk Evaluation and Monitoring Strategy in the USA and a ‘named patient’ program in other countries.9 Thus the proprotein convertase subtilisin/ kexin type 9 (PCSK9) monoclonal antibodies (mAbs), alirocumab and evolocumab, approved in late 2015, now provide a new class of drugs which substantially and safely decrease LDL-C.