Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

RCT (n=482) found that patients on subcutaneous injections of inclisiran, a small interfering RNA targeting hepatic PCSK9 synthesis (300mg) on days 1, 90, 270, and 450 had significantly lower levels of LDL cholesterol vs. placebo, with an acceptable safety profile.

SPS commentary:

In terms of the two primary end points:

 

  • At day 510, percent change in LDL cholesterol was a reduction of 39.7% in inclisiran group and an increase of 8.2% in the placebo group, (p<0.001).

 

  • The time-averaged percent change in LDL cholesterol level between day 90 and day 540 was a reduction of 38.1% in inclisiran group and an increase of 6.2% in placebo group (p<0.001).

 

Data from the ORION-10 and ORION-11 trials (n=1561 and 1617, respectively) also published in the journal found that reductions in LDL cholesterol levels of ~50% were obtained with inclisiran administered subcutaneously every 6 months.

New England Journal of Medicine

Resource links:

ORION-10 and ORION-11 trials