Novartis receives positive CHMP opinion for Leqvio, a potential first-in-class siRNA for the treatment of high cholesterol.

Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting marketing authorization of Leqvio (inclisiran) for the treatment of adults with hypercholesterolemia or mixed dyslipidemia, marking an important milestone towards it becoming available in the EU. Inclisiran is a potential first-in-class small interfering RNA (siRNA) with a new mechanism of action which delivers effective and sustained low-density lipoprotein cholesterol (LDL-C) reduction for patients with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk equivalent and heterozygous familial hypercholesterolemia (HeFH) a major driver of heart attacks, strokes and deaths. “This investigational medicine could significantly change how high LDL-C is treated,” said Professor Ulf Landmesser, M.D., Director of Charité Center for Cardiovascular Diseases, Berlin. “Many patients struggle to keep their LDL-C at recommended levels, and long-term exposure to high LDL-C is a major driver of ASCVD. The unique mechanism of action of inclisiran provides effective and sustained LDL-C reduction, a causal factor of atherosclerotic disease progression. With only two doses a year, and as an injection administered by healthcare professionals, it is anticipated to remove adherence challenges commonly encountered with self-administered treatments.” This CHMP opinion is based on results from the ORION clinical research program including Phase III trials, which involved more than 3,600 patients on a maximally tolerated statin dose and assessed the safety, efficacy and tolerability of inclisiran. Inclisiran demonstrated effective and sustained LDL-C reduction of up to 52% (P<0.0001) with two doses per year, after an initial dose and one at 3 months, in adults with ascvd, ascvd risk equivalent and or hefh. further, the reduction in ldl-c achieved with inclisiran was sustained through 17 months, with a safety and tolerability profile similar to placebo. additional post hoc analysis showed low variability among the patients treated with inclisiran: 88% of them reached guideline recommended targets at any timepoint during the study (observed values).>