For adults with HeFH on maximally tolerated statin therapy,

Repatha® Was Studied in Patients With HeFH Who Require Additional Lipid Lowering1,2

Study 3

Study 3 study design Study 3 study design

In a multicenter, double-blind, randomized, placebo-controlled trial:

  • Adult patients (18-80 yrs) with a clinical diagnosis of HeFH requiring additional LDL-C lowering†, ‡ were randomized to Repatha® or placebo1,2

  • Primary endpoint was percent change from baseline in LDL-C at week 122

  • Post-lipid stabilization period baseline characteristics:1

- Mean age = 51

- Mean baseline LDL-C = 156 mg/dL

- Gender = 42% female, 58% male

- All patients were taking statin therapy

- Atherosclerotic CVD = 38%

- High-intensity statin = 76%

– Mean age = 51

– Gender = 42% female, 58% male

– Atherosclerotic CVD = 38%

– Mean baseline LDL-C = 156 mg/dL

– All patients were taking statin therapy

– High-intensity statin = 76%

*Baseline was measured after the lipid stabilization period and before administration of first dose of study drug, n = 329.1

At screening, eligible patients included those with HeFH diagnosed by the Simon Broome diagnostic criteria on a stable statin dose with or without other approved lipid-modifying therapy for at least 4 weeks before screening.2

Use of LDL or plasmapheresis within 16 weeks prior to enrollment and treatment with fibrates within 6 weeks of treatment were not allowed. Patients were stratified by other, non-statin lipid-lowering therapy and LDL-C level prior to randomization; in adults, the Simon Broome criteria include an LDL-C of > 190 mg/dL (without therapy) plus clinical criteria (including patient or family history of tendon xanthomas, plus either family history of early CAD or family history of total cholesterol > 290 mg/dL).2,3

Repatha® Every 2 Weeks + a Statin Helped Patients Escape High LDL-C, With 61% Greater LDL-C Reduction Than Statin Alone1

Study 3

Study 3 results Study 3 results
  • Repatha® every 2 weeks helped up to 68% of patients achieve LDL-C < 70 mg/dL2

  • Repatha® once monthly lowered LDL-C an additional 60% when combined with background therapy1

Background therapy was statin therapy with or without other lipid-lowering therapies.

Mean baseline was 156 mg/dL.1

References: 1. Repatha® (evolocumab) Prescribing Information, Amgen. 2. Raal FJ, Stein EA, Dufour R, et al. Lancet. 2015;385:331-340. 3. Scientific Steering Committee on behalf of the Simon Broome Register Group. Br Med J. 1991;303:893-896.


See More

Contraindication: Repatha® is contraindicated in patients with a history of a serious hypersensitivity reaction to Repatha®.

Allergic reactions: Hypersensitivity reactions (e.g. rash, urticaria) have been reported in patients treated with Repatha®, including some that led to discontinuation of therapy. If signs or symptoms of serious allergic reactions occur, discontinue treatment with Repatha®, treat according to the standard of care, and monitor until signs and symptoms resolve.

Adverse reactions: The most common adverse reactions (> 5% of Repatha®-treated patients and more common than placebo) were: nasopharyngitis, upper respiratory tract infection, influenza, back pain, and injection site reactions.

In a 52-week trial, adverse reactions led to discontinuation of treatment in 2.2% of Repatha®-treated patients and 1% of placebo-treated patients. The most common adverse reaction that led to Repatha® treatment discontinuation and occurred at a rate greater than placebo was myalgia (0.3% versus 0% for Repatha® and placebo, respectively).

Adverse reactions from a pool of the 52-week trial and seven 12-week trials:
Local injection site reactions occurred in 3.2% and 3.0% of Repatha®-treated and placebo-treated patients, respectively. The most common injection site reactions were erythema, pain, and bruising. The proportions of patients who discontinued treatment due to local injection site reactions in Repatha®-treated patients and placebo-treated patients were 0.1% and 0%, respectively.

Allergic reactions occurred in 5.1% and 4.7% of Repatha®-treated and placebo-treated patients, respectively. The most common allergic reactions were rash (1.0% versus 0.5% for Repatha® and placebo, respectively), eczema (0.4% versus 0.2%), erythema (0.4% versus 0.2%), and urticaria (0.4% versus 0.1%).

Neurocognitive events were reported in less than or equal to 0.2% in Repatha®-treated and placebo-treated patients.

In a pool of placebo- and active-controlled trials, as well as open-label extension studies that followed them, a total of 1,988 patients treated with Repatha® had at least one LDL‑C value < 25 mg/dL. Changes to background lipid-altering therapy were not made in response to low LDL-C values, and Repatha® dosing was not modified or interrupted on this basis. Although adverse consequences of very low LDL-C were not identified in these trials, the long-term effects of very low levels of LDL-C induced by Repatha® are unknown.

Musculoskeletal adverse reactions were reported in 14.3% of Repatha®-treated patients and 12.8% of placebo-treated patients. The most common adverse reactions that occurred at a rate greater than placebo were back pain (3.2% versus 2.9% for Repatha® and placebo, respectively), arthralgia (2.3% versus 2.2%), and myalgia (2.0% versus 1.8%).

Immunogenicity: Repatha® is a human monoclonal antibody. As with all therapeutic proteins, there is a potential for immunogenicity with Repatha®.

Repatha® is indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (CVD), who require additional lowering of low density lipoprotein cholesterol (LDL-C).

The effect of Repatha® on cardiovascular morbidity and mortality has been published. Inclusion of the results in the approved labeling is under evaluation with the FDA.

Please see full Prescribing Information.

Back to Top