Benzinga | Dec 22, 2021 04:26PM EST

Alnylam Pharma Reports Comments On FDA Approval Of Leqvio Approved To Reduce Low-Density Lipoprotein Cholesterol

-- Leqvio(r) Becomes Fourth RNAi Therapeutic Approved by U.S. FDA and First and Only for Lowering LDL-C in Atherosclerotic Cardiovascular Disease (ASCVD) Which Can Affect Up to 30 Million Americans --

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today highlighted the significance of the U.S. Food and Drug Administration's (FDA) approval of Leqvio(r) (inclisiran), the fourth small interfering RNA (siRNA) therapy (or RNAi therapeutic) approved in the U.S., and the first and only to lower low-density lipoprotein cholesterol (also known as "bad cholesterol" or LDL-C). Leqvio is indicated in the U.S. as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with clinical atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) who require additional lowering of LDL-C. Alnylam scientists discovered inclisiran and published the first clinical data. Alnylam also supported early clinical development. As of January 2020, Novartis has obtained global rights to develop, manufacture and commercialize inclisiran under a license and collaboration agreement. Novartis AG continues to develop inclisiran and commercialize Leqvio worldwide, with Alnylam eligible to receive tiered royalties between 10 and 20 percent on global sales.

Leqvio is the fourth Alnylam-discovered medicine using its RNAi therapeutic platform to be approved to date. The Leqvio approval marks the first U.S. approval of an RNAi therapeutic indicated to treat a major risk factor for a highly prevalent disease. Alnylam launched its first RNAi therapeutic in 2018 with the FDA approval of ONPATTRO(r) (patisiran) for treatment of the polyneuropathy caused by hATTR amyloidosis, a progressive and life-threatening, rare, genetic disease. Leqvio was approved to lower LDL cholesterol and has the potential to benefit millions of people with ASCVD and also those with HeFH around the world.

"The approval of Leqvio, a potentially transformational medicine for lowering LDL-C is a historic event for Alnylam and its RNAi therapeutics platform. The demonstrated ability of Leqvio to lower LDL cholesterol up to 52% versus placebo on top of maximally tolerated statins with just two doses per year after an initial dose and another at three months represents a breakthrough that carries the potential to treat millions of people with ASCVD who are struggling to control elevated LDL cholesterol," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "More broadly, we believe the approval of Leqvio validates the future potential of RNAi therapeutics in large population diseases, and facilitates Alnylam's advancement toward its bold Alnylam P5x25 strategy and goals. We are proud to share in the significance of this milestone with Novartis who made this approval a reality."