On the heels of an FDA label expansion in August, Amgen is touting another potential avenue to extend the value of its decade-old PCSK9 cholesterol med Repatha.
In the late-stage Vesalius-CV study, Repatha (evolocumab) helped significantly curb the risk of major adverse cardiovascular events (MACE) over standard of care treatment alone in people who hadn’t had a heart attack or stroke before, Amgen announced Thursday.
The trial followed more than 12,000 high-risk patients over a median period of roughly four and a half years, with around 85% of those participants also receiving maintenance doses of a high-intensity or moderate therapy to manage their “bad” cholesterol, or LDL-C, Amgen said in an Oct. 2 press release.
That background therapy population is “particularly noteworthy,” analysts at Citi wrote in a Thursday note to clients, because it provides a glimpse into the larger primary prevention market Amgen could potentially unlock with the data.
Nevertheless, the Citi team isn’t expecting the data to deliver an “inflection in Repatha sales.” Rather, the results from Vesalius-CV could offer a “near-term additive” to the medication’s overall data package, they said.
Amgen did not indicate that it was imminently heading to regulators with the data. Repatha is already allowed to treat patients in the primary prevention setting thanks to an FDA nod in August.
A heart or stroke occurs in the U.S. every 40 seconds, with 75% of those representing first-time cardiovascular events, Amgen noted in its press release. High LDL-C is one of the “most modifiable risk factors for heart attack or stroke.” Still, most patients with high cardiovascular risk factors struggle to get those “bad” cholesterol levels in check, the company said, citing a recent report from the American Heart Association.
In a 2017 data drop, Amgen previously demonstrated Repatha’s ability to reduce the risk of major cardiovascular events in people with established atherosclerotic disease (ASCVD) and a history of major cardiovascular events like heart attack or stroke. With the Vesalius-CV trial—which Amgen says met both its primary endpoints—the company aimed to determine whether those risk reduction benefits would also be seen in patients without a history of heart attack or stroke.
The two key endpoints in Amgen’s study concerned the time to first occurrence of a composite of coronary heart disease (CHD) death, heart attack or ischemic stroke, as well as the time to first occurrence of a composite of CHD death, heart attack, ischemic stroke or any ischemia-driven arterial revascularization.
Amgen only teased the top-line readout in its announcement, noting that it plans to present full results from the Vesalius-CV study at a meeting of the American Heart Association in November.
"These results mark an important milestone in the fight against cardiovascular disease, the leading cause of death worldwide,” Amgen’s executive vice president of R&D, Jay Bradner, M.D., said in a statement.
He added that the new data “demonstrate that Repatha has the potential to reach tens of millions more patients earlier in their journey, before a life-altering event occurs."
Repatha was first approved in 2015 to treat certain patients with high cholesterol. The drug, which is a member of the PCSK9 heart med class, picked up its first cardiovascular nod in late 2017 when the FDA approved Repatha to prevent heart attacks, strokes and coronary revascularizations in adults with established cardiovascular disease.
More recently, the FDA in August broadened the drug’s label, removing the prior diagnosis requirement for cardiovascular disease and updating it to include adults at increased risk for MACE thanks to uncontrolled LDL-C.
The FDA nod pushed Repatha into the primary prevention setting for adults at risk of cardiovascular events, whereas its previous, secondary prevention nod required patients to have established cardiovascular disease to qualify for the therapy.
The overall market for primary prevention is “vast,” analysts at Leerink Partners wrote in June, noting that there are “tens of millions of patients globally still not reaching LDL-C goals despite available therapies.”
Repatha’s sales have been on the rise of late, with Amgen reporting a 36% increase to $2.22 billion for all of 2024. That momentum has carried through to 2025, with Amgen reporting in August that Repatha’s second-quarters sales had grown 31% to $696 million versus the same period last year.
Still, several storm clouds are gathering on the horizon for Amgen’s aging heart blockbuster.
Notably, the drug is expected to face price controls under the Inflation Reduction Act (IRA) in 2029, while biosimilars are poised to start chipping away at the drug’s U.S. sales stronghold at the beginning of the next decade, the Leerink team said earlier this year.
Repatha could keep hold of its market exclusivity a bit longer outside the U.S.—until May 2032—if certain patents survive until then, the analysts added.