Inclisiran Gains Indication as Stand-alone Therapy to Reduce LDL Cholesterol

Inclisiran (Leqvio) may now be used as a first-line treatment option for patients with high cholesterol levels, according to an announcement from drugmaker Novartis.

The US Food and Drug Administration approved the revised label for inclisiran, a small interfering RNA that reduces the production of proprotein convertase subtilisin/kexin type 9 (PCSK9) in the liver, based on studies showing that treatment effectively lowers LDL levels when used as a stand-alone therapy.

While statins are the cornerstone of primary and secondary prevention, inclisiran could play a role for patients who can’t tolerate statin therapy or those unwilling to take the drugs, say experts. Prior to the update, inclisiran, which is injected twice per year by a physician, was indicated for use in combination with statin therapy.

To date, there are no studies showing that using inclisiran alone, or even in combination with statin therapy, reduces the risk of major adverse cardiovascular events. Several trials are currently underway, however. The VICTORION-1 PREVENT study is testing whether inclisiran monotherapy is superior to placebo for reducing the risk of MACE in high-risk patients without prior cardiovascular disease—those results aren’t expected until 2029.

In secondary prevention, the VICTORION-2 PREVENT study is testing whether inclisiran added to high-intensity statin therapy reduces the risk of major adverse cardiovascular events in patients with established CVD. Enrollment is also complete in ORION-4, another cardiovascular outcomes study of inclisiran in about 16,000 patients with established cardiovascular disease. These trials are expected to be presented in 2027 and 2026, respectively.