The US Food and Drug Administration has approved a significant label update for Novartis' Leqvio (inclisiran), enabling the twice-yearly injectable therapy to be used as monotherapy alongside diet and exercise for reducing low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia. The FDA proactively requested this label update based on robust LDL-C lowering data for PCSK9-targeting therapies.
Expanded Treatment Options
The updated label removes the previous requirement for Leqvio to be used on top of or in combination with statin therapy. This change positions Leqvio as a first-line treatment option for patients with high cholesterol, potentially expanding its use earlier in the treatment journey.
"This first-line label update reinforces Leqvio's proven ability to effectively lower LDL-C, a critical risk factor for heart disease," said Victor Bultó, President, US, Novartis. "With this new indication enabling Leqvio's use as monotherapy along with diet and exercise, we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey."
Addressing Treatment Gaps
The approval comes at a critical time when up to 80% of atherosclerotic cardiovascular disease (ASCVD) patients in the US struggle to reach the LDL-C guideline-recommended target of less than 70 mg/dL. This treatment gap is particularly concerning given that ASCVD accounts for 85% of all cardiovascular deaths and represents a greater burden in the US than any other chronic disease.
The need for more effective cholesterol management has been heightened by the latest 2025 ACC/AHA Joint Committee Clinical Practice Guideline for the Management of Patients with Acute Coronary Syndromes, which recommends more aggressive treatment to achieve LDL-C targets.
Unique Dosing Advantage
Leqvio's twice-yearly, healthcare provider-administered dosing schedule is uniquely positioned to help support patient adherence and long-term LDL-C management, including goal attainment. After an initial dose and another at three months, patients receive subsequent doses every six months.
Label Refinements
Additional updates to the label include revising "primary hyperlipidemia" to the more specific term "hypercholesterolemia" throughout the prescribing information, to more accurately focus on LDL-C reduction.
Clinical Context
Cardiovascular disease affects hundreds of millions of people globally and claims more lives than cancer, chronic lung disease, and diabetes combined. Around 80% of premature cardiovascular deaths can be prevented by addressing factors that cause or worsen cardiovascular disease. ASCVD is caused by the development and growth of plaques in the inner lining of arteries, with atherosclerotic plaque mainly composed of LDL-C that accumulates over time.
