PCSK9 siRNA recommended by CHMP for EU approval

News - Oct. 28, 2020

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on inclisiran for treatment of patients with hypercholesterolemia or mixed dyslipidemia and recommended granting marketing authorization of inclisiran.

Inclisiran is small interfering RNA (siRNA) against PCSK9, resulting in effective and sustained LDL-c reduction in patients with atherosclerotic CVD (ASCVD), ASCVD risk equivalent and heterozygous FH.

“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.”

The opinion of the CHMP is based on results from the ORION clinical research program, involving >3600 patients on maximally tolerated statins. The ORION program assessed the safety, efficacy and tolerability of inclisiran. Inclisiran resulted in effective and sustained LDL-c reduction up to 52% with two doses per year, after a starting dose and one at 3 months, in patients with ASCVD, ASCVD risk equivalent and/or HeFH. LDL-c reduction was sustained through 17 months, with a good safety and tolerability profile. A post-hoc analysis demonstrated low variability among patients on inclisiran: 88% of patients reached guideline recommended targets at any timepoint during the study.

The CHMP recommended granting marketing authorization of inclisiran for the treatment of patients with primary hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidemia, as an adjunct to diet: in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-c goals, or alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant or for whom a statin is contraindicated.

The European Commission (EC) has the authority to approve medicine for the EU and will review the CHMP opinion. Inclisiran is also under review by the US Food and Drug Administration for treatment of primary hyperlipidemia (including HeFH) in patients with increased LDL-c while taking maximally tolerated statin therapy.

Source: press release Novartis, October 16, 2020

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