What is the mechanism of action for Leqvio (inclisiran)?

On the surface of liver cells are receptors for LDL cholesterol that play a critical role in regulating blood cholesterol levels. The more receptors that are present, the quicker cholesterol can be removed from the bloodstream. Receptor numbers are under the control of a protein, called PCSK9 which breaks down LDL receptors, meaning more cholesterol is left in the blood. Leqvio works by blocking this PCSK9 protein which means more LDL receptors are present on liver cells which increases the uptake of LDL cholesterol and lowers LDL cholesterol in the blood, reducing cholesterol levels.

Leqvio blocks the PCSK9 protein by interfering with the translation of PCSK9 messenger RNA (mRNA) which limits the production of PCSK9. It is a double-stranded small interfering ribonucleic acid (siRNA), which is conjugated with triantennary N-Acetylgalactosamine (GalNAc) to allow better uptake by liver cells.

Leqvio is administered by subcutaneous (under the skin) injection every 6 months and is used in combination with a daily oral statin (such as atorvastatin or rosuvastatin) together with a healthy diet. It belongs to the class of medicines known as small interfering ribonucleic acids (siRNAs).