Novartis has secured approval from the US Food and Drug Administration (FDA) for its small interfering RNA (siRNA) therapy Leqvio (inclisiran) for its use in lowering low-density lipoprotein cholesterol (bad cholesterol or LDL-C).

Leqvio FDA approval is for the drug to be administered in two doses a year, after an initial dose followed by another one three months later.

Leqvio is approved by the FDA as an adjunct to diet and maximally tolerated statin therapy for treating adults having clinical atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) who need further lowering of bad cholesterol.

Vas Narasimhan — Novartis CEO said: “Leqvio is a revolutionary approach to lower LDL-C, and creates new possibilities for how healthcare systems can impact cardiovascular disease, a defining public health challenge of our time.

“We now have the opportunity, working together with partners, to provide this first-ever approved LDL-C–lowering siRNA-based therapy to tackle ASCVD at scale across the United States.”

According to Novartis, the effect that Leqvio has on cardiovascular morbidity and mortality is being studied in clinical trials that are presently in progress.

Novartis said that Leqvio brings down the amount of bad cholesterol in the bloodstream by boosting the natural ability of the liver to block the production of a protein that is instrumental in sustaining high levels of circulating cholesterol.

Leqvio, which will be available in the US from January 2022, is a subcutaneous injection to be administered by a healthcare provider.

Novartis bagged Leqvio FDA approval courtesy of the findings of the phase 3 ORION-9, -10 and -11 clinical trials. The three clinical studies saw the participation of 3,457 participants having atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia and with increased LDL-C while undertaking a maximally tolerated dose of statin therapy.

In the phase 3 clinical trials at month 17, Leqvio provided effective and sustained reduction of bad cholesterol of up to 52%, compared to placebo. The siRNA therapy was found to be well-tolerated with a safety profile that was comparable to placebo.

Novartis had been given global rights for developing, manufacturing, and commercializing Leqvio under a license and collaboration deal with Alnylam Pharmaceuticals.

John Maraganore — CEO of Alnylam Pharmaceuticals said: “The approval of Leqvio, a potentially transformational medicine for lowering LDL-C is a historic event for Alnylam and its RNAi therapeutics platform.

“The demonstrated ability of Leqvio to lower LDL cholesterol up to 52% versus placebo on top of maximally tolerated statins with just two doses per year after an initial dose and another at three months represents a breakthrough that carries the potential to treat millions of people with ASCVD who are struggling to control elevated LDL cholesterol.”

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