OBJECTIVES: Two novel biologics, alirocumab and evolocumab, have recently been licensed for the treatment of familial hypercholesterolaemia (FH). Unlike other treatments for this disease, neither licence covers the full paediatric population. Our aim was to conduct a systematic literature review (SLR) of clinical data on pharmacological interventions for FH exclusively in children and adolescents ≤18 years. METHODS: Searches were conducted in Epub-Ahead-of-Print, MEDLINE, MEDLINE In-Process, Embase and The Cochrane Library on 19/05/2016, with no date limit. English language articles were included if they presented randomised controlled trial (RCT) data on pharmacological interventions for FH, exclusively in patients aged ≤18 years. The eligibility of each article was assessed by two independent reviewers and data on study design, baseline characteristics and efficacy, including percentage reductions in total cholesterol (TC) and low-density lipoprotein (LDL-C), were extracted. RESULTS: A total of CONCLUSIONS: Statins were the most commonly investigated intervention, followed by BAS and ezetimibe. All interventions were found to reduce TC and LDL-C; however, network meta-analysis will yield greater insight into the comparable efficacy of these interventions. Interestingly, studies investigating biologics were not identified solely in patients aged ≤18 years, highlighting the potential for future investigation in this patient group.