FDA approves add-on therapy to lower cholesterol among certain high-risk adults

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FDA has approved Leqvio (inclisiran) injection as a treatment to be used along with diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Leqvio works to reduce circulating levels of LDL-C, commonly known as “bad cholesterol.”

Leqvio is approved at a 284 mg dose administered as an initial under-the skin injection, a second dose at three months, and continued treatment once every six months after that point.

Diseases or Conditions

HeFH is a rare, life-threatening condition in which patients have a mutation in a small group of genes that controls the way the body clears cholesterol. As a result, patients have extremely high levels of LDL-C. People with HeFH generally have cholesterol levels two to three times higher than normal. These individuals are at increased risk of cardiovascular events, such as heart attack, stroke, and coronary artery disease. HeFH occurs in approximately 1 in 250 individuals.

ASCVD involves the buildup of cholesterol plaque in arteries. Approximately 18.3 million American adults (8%) have ASCVD. Clinical ASCVD is an umbrella term and includes conditions such as acute coronary syndromes (sudden, reduced blood flow to the heart), peripheral arterial disease, heart attack and stroke.

Effectiveness

The effectiveness of Leqvio was studied in three randomized, double-blind, placebo-controlled trials that enrolled 3,457 adults with HeFH or clinical ASCVD. Enrolled participants were taking maximally tolerated statin therapy but required additional LDL-C lowering based on their risk for cardiovascular events. In all three studies, the main effectiveness outcome measure was the percent change in LDL-C from the beginning of the trial to day 510 (month 17). In each trial, participants received under-the-skin injections of either 284 mg Leqvio or a placebo on four separate days: day 1, day 90 (month 3), day 270 (month 9), and day 450 (month 15).

Study 1 enrolled 1,561 adults with ASCVD. At day 510, the Leqvio group had an average LDL-C decrease of 51% whereas the placebo group had an average LDL-C increase of 1%. Study 2 enrolled 1,414 adults with ASCVD. At day 510, the Leqvio group had an average LDL-C decrease of 46% whereas the placebo group had an average LDL-C increase of 4%. Study 3 enrolled 482 adults with HeFH. At day 510, the Leqvio group had an average LDL-C decrease of 40% whereas the placebo group had an average LDL-C increase of 8%.

The effect of Leqvio on cardiovascular morbidity (suffering from a disease) and mortality (death) has not been determined.

Safety Information

Common side effects of Leqvio include injection site reaction, joint stiffness, urinary tract infection, diarrhea, bronchitis, pain in extremity, and difficulty breathing.

See the prescribing information for additional information on risks associated with Leqvio.