Findings from the Phase III VESALIUS-CV study reinforce the cholesterol-lowering benefit of Repatha in high-risk patients without prior cardiovascular disease, for which the drug was approved in August 2024.
Amgen’s PCSK9 blocker Repatha significantly lowered the risk of heart complications in patients who had no prior history of cardiovascular disease, according to a topline Phase III readout on Thursday, further cementing the drug as a primary prevention option for cardiovascular disease.
The pharma did not provide specific data in its news release, claiming only that Repatha elicited a “statistically and clinically significant” improvement in a composite endpoint including heart attack, ischemic stroke and death from coronary heart disease. Amgen did not report any new safety signals of concern.
“These Ph3 data reinforce the label expansion decision by FDA,” analysts at Leerink Partners told investors in a note on Thursday, referring to a regulatory decision in August 2024 that allowed the use of Repatha to lower bad cholesterol levels in adult patients regardless of their history of heart disease. At the time, Amgen noted that the approval “removes a prior requirement for a patient to have been diagnosed with cardiovascular disease.”
“Some physicians already prescribe Repatha off-label for primary prevention,” Leerink wrote in its investor note, nevertheless noting that “the formal label expansion should help drive broader adoption.” Thursday’s readout, the analysts added, “will help broaden Repatha’s reach into the preliminary prevention space.”
The readout comes from the Phase III VESALIUS-CV trial, which enrolled more than 12,300 patients who were deemed high-risk: they had elevated levels of LDL cholesterol, diabetes mellitus or peripheral arterial disease at baseline, among other risk factors. None of the patients had had a previous myocardial infarction or stroke. Participants were followed for a median duration of approximately 4.5 years.
Amgen has promised to present full findings from VESALIUS-CV on Nov. 8 at the American Heart Association Scientific Sessions.
Designed to be injected under the skin, Repatha is a monoclonal antibody that blocks the PCSK9 protein, which otherwise tags LDL receptors on liver cells for destruction. By disrupting this pathway, Repatha facilitates the clearance of LDL cholesterol from the blood. The drug was first approved in August 2015 to lower levels of bad cholesterol in patients with established cardiovascular disease.
