Familial hypercholesterolaemia
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Repatha® in heterozygous familial hypercholesterolaemia (HeFH)

  • 60% LDL-C reduction vs placebo at the mean of Weeks 10 and 12 for every 2 week dose2
  • 67% of patients achieved LDL-C <70 mg/dl (<1.8 mmol/l) at the mean of Weeks 10 and 122
  • LDL-C reductions were similar in all patients regardless of sex, age, baseline LDL-C, BMI, or intensity of statin therapy2

Mean LDL-C percent change from baseline to mean of Weeks 10 and 12*2

RUTHERFORD-2 was an international, multicentre, double-blind, randomised, placebo-controlled, 12-week study in 329 patients with heterozygous familial hypercholesterolaemia on stable lipid-lowering therapies for at least 4 weeks with fasting LDL-C concentration of 2.6 mmol/l or higher.*2

Repatha® in homozygous familial hypercholesterolaemia (HoFH)
Repatha® is the first approved PCSK9 inhibitor that has been evaluated specifically in a Phase 3 randomised, controlled trial in patients with homozygous familial hypercholesterolaemia.3
The percentage change in LDL-C from baseline to Week 12 was –23.1% with Repatha® vs +7.9% with placebo, corresponding to a treatment difference of 30.9% (adjusted p<0.0001).†3

*Background therapy included statin therapy with or without ezetimibe or other lipid-lowering therapies. Subcutaneous Repatha® 140 mg every 2 weeks, Repatha® 420 mg monthly, or subcutaneous placebo every 2 weeks or monthly was administered. Co-primary endpoints were mean percent change from baseline in LDL-C at the mean of 10 and 12 weeks and 12 weeks alone. Secondary endpoints were the absolute change from baseline in LDL cholesterol and the percentage of patients achieving a target of LDL cholesterol lower than 1.8 mmol/l at the same timepoints. Mean LDL-C at baseline was 3.9 mmol/l in the placebo groups and 4.0–4.2 mmol/l in the Repatha® groups. Mean LDL-C at the mean of Weeks 10 and 12 was 3.8–4.1 mmol/l with placebo and 1.7–1.8 mmol/l with Repatha®.
49 HoFH patients aged 12–65 years receiving stable background statin therapy with or without ezetimibe and not receiving apheresis. Repatha® was administered at the recommended dose of 420 mg (3 x 140 mg) once monthly. The response to Repatha® was related to the underlying genetic defect causing HoFH, ranging from a 40.8% mean reduction in LDL cholesterol compared with placebo in the 57% (28/49) of patients with either one or two defective LDL receptor mutations to no response in patients with two negative or suspected negative mutations.
In the study, 2690 recent MI patients received Repatha®, 2254 of whom achieved LDL-C levels <55 mg/dl.
§25% RRR reported as the key secondary endpoint: composite of cardiovascular death, MI or stroke. HR: 0.75; 95% CI: 0.62-0.91; p=0.003. ARR: 3.2%; 95% CI: 1.2-5.2.
ACS = Acute coronary syndrome; ARR = Absolute risk reduction; BMI = Body mass index; CV = Cardiovascular; EAS = European Atherosclerosis Society; ESC = European Society of Cardiology; EVOPACS = EVOlocumab for Early Reduction of LDL-cholesterol Levels in Patients With Acute Coronary Syndromes; LDL-C = Low-density lipoprotein cholesterol; MI = Myocardial infarction; PCSK9 = Protein convertase subtilisin/kexin type 9; Q2W = Every 2 weeks; RRR = Relative risk reduction; RUTHERFORD = Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder.
  1. Repatha® (evolocumab) Summary of Product Characteristics. Last revised: 31 March 2021.
  2. Raal FJ, et al. Lancet. 2015;385:331-40.
  3. Raal FJ, et al. Lancet. 2015;385:341-50.
  4. Gencer B, et al. JAMA Cardiol. 2020;5(8):1-6. Main paper & Supplementary appendix.

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