Novartis has been granted a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for its cholesterol drug Leqvio.
Leqvio (inclisiran) is a potential first-in-class treatment for high cholesterol in Europe for patients with hypercholesterolemia or mixed dyslipidemia.
The drug is a small interfering RNA (sIRNA) with a new mechanism of action that allows it to deliver effective and sustained low-density lipoprotein cholesterol (LDL-C) reduction for patients with a number of cardiovascular risk factors.
It works by blocking the synthesis of PCSK9 in the liver rather than targeting the protein itself, which allows more receptors on the liver cell surface to capture LDL cholesterol to break down.
The positive opinion is based on results from Novartis’ ORION clinical research programme, including phase 3 trials which involved over 3,600 patients on a maximum statin dose.
In these trials, Leqvio demonstrated LDL-C reduction of up to 52% with two doses per year administered as a subcutaneous injection, following an initial dose and one at three months, in adults with atherosclerotic cardiovascular disease (ASCVD), ASCVD risk equivalent and heterozygous familial hypercholesterolemia (HeFH).
In addition, LDL-C reduction achieved with Leqvio treatment was sustained after 17 months, with a similar safety and tolerability profile to placebo. Another trial, dubbed ORION-4, is currently underway to determine if the reduction in LDL-C is coupled with an improvement in cardiovascular outcomes.
Novartis picked up Leqvio following its $9.7bn acquisition of The Medicines Company, which completed earlier this year.
Novartis is also aiming for approval from the US Food and Drug Administration (FDA), where Leqvio is currently under review for the treatment of primary hyperlipidaemia in adults with high LDL levels that have not been brought down by a maximum statin dose.
Earlier this year Novartis announced that it had teamed up with the NHS in the UK, with the aim of acceleraating access to Leqvio following if it is approved by the EMA.
The planned clinical trial will use NHS data to identify patients across the country who are at risk of heart disease and who have not improved with conventional treatment regimens, including statin therapy.