Khoury · Expert opinion on drug discovery 2020 · narrative review · n=?

Preclinical discovery and development of evolocumab for the treatment of hypercholesterolemia.

Cited 7 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Narrative review and expert opinion with no primary human data or systematic synthesis

PubMed 31973581 · doi:10.1080/17460441.2020.1704728 · record verified 2026-08-30

What was done

The authors reviewed the timeline and preclinical-to-clinical development of evolocumab, a monoclonal antibody directed against proprotein convertase subtilisin/kexin type 9 (PCSK9) used as an adjunct lipid-lowering therapy for familial hypercholesterolemia and atherosclerotic cardiovascular disease.

What was found

The abstract provides a descriptive overview without numerical outcome metrics. It notes that PCSK9 was identified in 2002, leading to evolocumab's regulatory approval in 2015 for lowering LDL-cholesterol and reducing cardiovascular events, while highlighting ongoing real-world challenges regarding drug affordability, accessibility, and cost-effectiveness.

Why it matters

It summarizes the translational pipeline of PCSK9 inhibition from molecular target discovery to clinical practice, emphasizing the implementation barriers faced by high-cost precision biologic therapies.

Limits

The abstract contains no empirical trial data, effect estimates, or systematic review methodology. It represents a narrative overview and expert opinion.

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