BACKGROUND : Alirocumab reduces low-density lipoprotein
cholesterol (LDL-C) levels by up to 61%. The ODYSSEY
Open-Label Extension study investigated the effect of
alirocumab in patients with heterozygous familial hypercholesterolaemia
(HeFH) over 144 weeks.
METHODS : Eligible patients with HeFH had completed an
earlier double-blind, randomised, placebo-controlled parent
study. Patients were initiated on 75 mg alirocumab Q2W
subcutaneous (SC) unless baseline LDL-C was > 8.9 mmol/l,
in which case they received 150 mg alirocumab Q2W. Dose
titration to 150 mg Q2W was at the investigator’s discretion.
RESULTS : The study enrolled 167 patients and the parent study
mean (± SD) baseline LDL-C level was 3.65 ± 1.9 mmol/l.
Mean LDL-C level was reduced by 48.7% at week 144; mean
on-treatment LDL-C was 2.30 ± 1.24 mmol/l. Eight patients
reported injection-site reactions, with one treatment discontinuation.
Treatment emergent anti-drug antibodies were
identified in five patients but these did not affect the efficacy.
CONCLUSION : Alirocumab effectively and safely reduced LDL-C
in these patients.