The Swiss drugmaker acquired Leqvio in January 2020 through its $9.7-billion takeover of The Medicines Company. Alnylam Pharmaceuticals initially developed the treatment, and under the terms of a strategic collaboration between it and private-equity investors Blackstone, 50% of Alnylam's Leqvio-related royalties will flow to Blackstone.
Novartis said Wednesday that the FDA has cleared its PCSK9-targeting siRNA Leqvio (inclisiran) as an add-on therapy to help decrease cholesterol levels in certain high-risk adults. The drug, which will be available in early January, is indicated for use along with maximally tolerated statins for patients with heterozygous familial hypercholesterolaemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL cholesterol.
"Leqvio is a revolutionary approach…and creates new possibilities for how healthcare systems can impact cardiovascular disease," remarked CEO Vas Narasimhan, adding "we now have the opportunity, working together with partners, to provide this first-ever approved LDL-cholesterol–lowering siRNA-based therapy to tackle ASCVD at scale across the US."
Follows initial rejection last year
Novartis refiled its application for Leqvio this summer after the FDA issued a complete response letter about a year ago, citing "unresolved" issues at a third-party production facility. The company said that in its resubmission, it listed its own site in Schaftenau, Austria, as the manufacturing location for the final finished product.
The FDA decision is based on results of the ORION clinical development programme, including the Phase III ORION-9, ORION-10 and ORION-11 studies, where Leqvio provided "effective and sustained" LDL-cholesterol reductions of up to 52%, versus placebo, despite patients being on maximally tolerated statin therapy. The drug also had a comparable safety profile to placebo, with the most common side effects being mild-to-moderate injection-site reactions, joint pain, urinary tract infections, diarrhoea, chest cold, pain in legs or arms and shortness of breath.Leqvio is administered by subcutaneous injection as an initial dose, followed by a second dose three months later, with continued treatment given twice yearly after that point. The European Medicines Agency authorised it in December last year. Meanwhile, Leqvio's effect on cardiovascular outcomes is currently being investigated in two ongoing trials.