Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review.

“We are excited that the FDA has accepted our NDA for vutrisiran, which was based on 9-month results from the HELIOS-A study. Today’s announcement marks another important milestone as we work to make vutrisiran available to hATTR amyloidosis patients with polyneuropathy,” said Rena Denoncourt, Vice President, TTR Franchise Lead. “If approved, once-quarterly, subcutaneously administered vutrisiran may represent a new treatment option that potentially reverses polyneuropathy manifestations of disease.”

Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast-Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. The Company plans to also submit regulatory filings in the EU, Brazil, and Japan in 2021 based on the HELIOS-A results.

About Vutrisiran

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The safety and efficacy of vutrisiran have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.