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Alnylam Pharma Submits MAA To EMA For Vutrisiran For Hereditary ATTR Amyloidosis With Polyneuropathy

RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) announced Monday the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with polyneuropathy.

Positive 9-month results from the HELIOS-A Phase 3 study of vutrisiran were presented in April 2021 at the American Academy of Neurology (AAN) Virtual Annual Meeting.

At 9 months, vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy, quality of life, and gait speed, and demonstrated an encouraging safety profile, relative to the external placebo group of the APOLLO study of patisiran.

As aligned with the EMA, the results of the 18-month analysis from the HELIOS-A study will be provided to the Agency during its evaluation of the MAA.

Vutrisiran has been granted Orphan Drug Designation in the European Union (EU) and U.S. for the treatment of ATTR amyloidosis.

In June 2021, the U.S. Food and Drug Administration (FDA) accepted the Company's New Drug Application (NDA) submission for review for vutrisiran, with an action date set for April 14, 2022 under the Prescription Drug User Fee Act (PDUFA). The Company also plans to submit regulatory filings in Brazil and Japan in 2021.

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary ATTR (hATTR) and wild-type ATTR (wtATTR) amyloidosis.

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