Vutrisiran

Vutrisiran, sold under the brand name Amvuttra, is a medication used for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.^[1]^[2] It is a small interfering RNA (siRNA) that interferes with the expression of the transthyretin (TTR) gene.^[3]

Vutrisiran was approved for medical use in the United States in June 2022.^[1]^[2]

History

The U.S. Food and Drug Administration (FDA) granted the application for vutrisiran orphan drug designation.^[5]

^ ^a ^b ^c "Amvuttra- vutrisiran injection". DailyMed. 21 June 2022. Retrieved 3 July 2022.
^ ^a ^b ^c "Alnylam Announces FDA Approval of Amvuttra (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults". Alnylam. 13 June 2022. Archived from the original on 14 June 2022. Retrieved 14 June 2022 – via Business Wire.
^ Aimo A, Castiglione V, Rapezzi C, Franzini M, Panichella G, Vergaro G, Gillmore J, Fontana M, Passino C, Emdin M (March 2022). "RNA-targeting and gene editing therapies for transthyretin amyloidosis". Nature Reviews. Cardiology. doi:10.1038/s41569-022-00683-z. PMID 35322226. S2CID 247623259.
^ World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl:10665/330896.
^ "Vutrisiran Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 25 May 2018. Retrieved 14 June 2022.

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