Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics[3] for genetically defined diseases.

[17] In 2009, the company formed alliances with Cubist Pharmaceuticals and Kyowa Hakko Kirin to market a drug targeted at respiratory syncytial virus.

[37] In December 2021, Alnylam submitted a clinical trial authorisation (CTA) application to the Medicines and Healthcare Products Regulatory Agency in the United Kingdom to initiate a Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of Alzheimer's disease and cerebral amyloid angiopathy.

[38] On December 22, 2021, Novartis announced that the US Food and Drug Administration (FDA) approved Leqvio (inclisiran), a small interfering RNA (siRNA) therapy to lower low-density lipoprotein cholesterol.

Leqvio is indicated in the United States 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.

Novartis obtained global rights to develop, manufacture and commercialize Leqvio under a license and collaboration agreement with Alnylam Pharmaceuticals.

[33] In August 2018, with its commercial name Onpattro, patisiran received the U.S. regulatory approval to treat polyneuropathy in patients with hereditary ATTR amyloidosis.

Although the APOLLO-B study met key endpoints, the FDA questioned the clinical significance of the results particularly for patients not on background therapy with tafamidis.

The FDA is seeking the committee's input on the clinical meaningfulness and patient populations for patisiran use potentially challenging Pfizer's tafamidis dominance in ATTR-CM treatment.