Amgen Announces Long-Term Safety and Efficacy Data from Two Open-Label Extension Studies of Repatha® (evolocumab) in ASCVD Patients

Compiled by newborn

Recently, Amgen announced the topline results of two open-label extension (OLE) studies from the Phase 3 FOURIER cardiovascular outcomes trial of the PCSK9 inhibitor class lipid-lowering drug Repatha (Repatha, generic name: evolocumab). The studies aimed to evaluate the long-term safety and tolerability of Repatha over a 5-year treatment period in adult patients with clinically evident atherosclerotic cardiovascular disease (ASCVD).

FOURIER-OLE Study Composed of Study 20130295 (NCT02867813) and Study 20160250 (NCT03080935), the former enrolled 5,035 patients in Eastern Europe and the United States, while the latter enrolled 1,600 patients in Western Europe. Both studies demonstrated that Repatha regimens of 140 mg every two weeks or 420 mg monthly were safe and well-tolerated. In the two OLE studies, patients received Repatha treatment for approximately five years; in FOURIER and the two OLE studies, some patients received Repatha treatment for a cumulative duration of up to 8.5 years. No new long-term safety issues were observed.

Moreover, during the OLE study period, a medically significant and sustained reduction in low-density lipoprotein cholesterol (LDL-C) levels was observed, with more than 85% of patients achieving LDL-C levels of <40 mg/dL.

Other study endpoints included exploratory analyses of non-HDL cholesterol, apolipoprotein B, total cholesterol, lipoprotein (a), triglycerides, HDL cholesterol, VLDL cholesterol, and apolipoprotein A-1 levels, as well as related cardiovascular events.

Detailed study results will be shared with regulatory authorities and presented at a medical conference later this year. In addition, the ongoing VESALIUS-CV (NCT03872401) outcomes trial is also evaluating the long-term LDL-C lowering effects of Repatha.

Repatha is a monoclonal antibody drug that targets and binds to proprotein convertase subtilisin/kexin type 9 (PCSK9), inhibiting the binding of circulating PCSK9 to low-density lipoprotein (LDL) receptors (LDLR). This prevents PCSK9-mediated LDLR degradation, allowing LDLR to recycle back to the surface of hepatocytes. By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby reducing LDL cholesterol (LDL-C) levels.

The results of the two OLE studies announced this time confirm the good safety of long-term use of Repatha in lowering LDL-C. To date, Repatha has been approved in more than 75 countries worldwide, including China, and over one million patients have received treatment with Repatha.

Reference Source: AMGEN ANNOUNCES RESULTS FROM TWO OPEN LABEL EXTENSION STUDIES OF REPATHA® (EVOLOCUMAB)