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'''Vutrisiran''', sold under the brand name '''Amvuttra''', is a [[medication]] used for the treatment of the [[polyneuropathy]] of [[hereditary transthyretin-mediated amyloidosis|hereditary transthyretin-mediated (hATTR) amyloidosis]] in adults.<ref name="Alnylam PR 20220613" /> It is a [[small interfering RNA]] (siRNA) that interferes with the expression of the [[transthyretin]] (TTR) gene.<ref name="Aimo_2022">{{cite journal | vauthors = Aimo A, Castiglione V, Rapezzi C, Franzini M, Panichella G, Vergaro G, Gillmore J, Fontana M, Passino C, Emdin M | title = RNA-targeting and gene editing therapies for transthyretin amyloidosis | journal = Nature Reviews. Cardiology | volume = | issue = | pages = | date = March 2022 | pmid = 35322226 | doi = 10.1038/s41569-022-00683-z }}</ref>
'''Vutrisiran''', sold under the brand name '''Amvuttra''', is a [[medication]] used for the treatment of the [[polyneuropathy]] of [[hereditary transthyretin-mediated amyloidosis|hereditary transthyretin-mediated (hATTR) amyloidosis]] in adults.<ref name="Alnylam PR 20220613" /> It is a [[small interfering RNA]] (siRNA) that interferes with the expression of the [[transthyretin]] (TTR) gene.<ref name="Aimo_2022">{{cite journal | vauthors = Aimo A, Castiglione V, Rapezzi C, Franzini M, Panichella G, Vergaro G, Gillmore J, Fontana M, Passino C, Emdin M | title = RNA-targeting and gene editing therapies for transthyretin amyloidosis | journal = Nature Reviews. Cardiology | volume = | issue = | pages = | date = March 2022 | pmid = 35322226 | doi = 10.1038/s41569-022-00683-z | s2cid = 247623259 }}</ref>


Vutrisiran was approved for medical use in the United States in June 2022.<ref name="Alnylam PR 20220613" />
Vutrisiran was approved for medical use in the United States in June 2022.<ref name="Alnylam PR 20220613" />

Revision as of 06:48, 1 July 2022

Vutrisiran
Clinical data
Trade namesAmvuttra
Other namesALN-65492
License data
Routes of
administration
Subcutaneous
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG

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] 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]

Vutrisiran is the international nonproprietary name (INN).[4]

History

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

References

  1. ^ 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.
  2. ^ "AMVUTTRA (vutrisiran) prescribing information" (PDF). Cambridge, MA: Alnylam Pharmaceuticals, Inc. June 2022. Archived (PDF) from the original on 14 June 2022. Retrieved 14 June 2022.
  3. ^ 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.
  4. ^ World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl:10665/330896.
  5. ^ "Vutrisiran Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 25 May 2018. Retrieved 14 June 2022.
  • "Vutrisiran". Drug Information Portal. U.S. National Library of Medicine.