Vutrisiran

Vutrisiran
Clinical data
Trade namesAmvuttra
Other namesALN-65492, ALN-TTRSC02
AHFS/Drugs.comMonograph
MedlinePlusa622061
License data
Pregnancy
category
Routes of
administration		Subcutaneous
Drug classSmall interfering RNA
ATC code
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.[7][5][8] It is a double stranded small interfering RNA (siRNA) (also called RNA interference, or RNAi therapeutic) that interferes with the expression of the transthyretin (TTR) gene.[9][10] Transthyretin is a serum protein made in the liver whose major function is transport of vitamin A and thyroxine.[11][12] Rare mutations in the transthyretin gene result in accumulation of large amyloid deposits of misfolded transthyretin molecules most prominently in peripheral nerves and the heart. Patients with hATTR typically present with polyneuropathy or autonomic dysfunction followed by cardiomyopathy which, if untreated, is fatal within 5 to 10 years.[12]

Vutrisiran was approved for medical use in the United States in June 2022,[5][8] in the European Union in September 2022,[6] and in Australia in June 2024.[1]

  1. ^ a b c "Amvuttra (vutrisiran)". Therapeutic Goods Administration (TGA). 5 July 2024. Retrieved 7 July 2024.
  2. ^ "Amvuttra, (Medison Pharma Australia Pty Ltd)". Therapeutic Goods Administration (TGA). 1 July 2024. Retrieved 7 July 2024.
  3. ^ "Details for: Amvuttra". Health Canada. 15 December 2023. Retrieved 3 March 2024.
  4. ^ "Summary Basis of Decision (SBD) for Amvuttra". Drug and Health Products Portal. 1 September 2012. Retrieved 9 May 2024.
  5. ^ a b c "Amvuttra- vutrisiran injection". DailyMed. 21 June 2022. Archived from the original on 3 July 2022. Retrieved 3 July 2022.
  6. ^ a b Cite error: The named reference Amvuttra EPAR was invoked but never defined (see the help page).
  7. ^ Tamargo J, Agewall S, Borghi C, Ceconi C, Cerbai E, Dan GA, et al. (May 2023). "New pharmacological agents and novel cardiovascular pharmacotherapy strategies in 2022". European Heart Journal - Cardiovascular Pharmacotherapy. 9 (4): 353–370. doi:10.1093/ehjcvp/pvad034. PMC 10236523. PMID 37169875.
  8. ^ a b "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.
  9. ^ Aimo A, Castiglione V, Rapezzi C, Franzini M, Panichella G, Vergaro G, et al. (October 2022). "RNA-targeting and gene editing therapies for transthyretin amyloidosis". Nature Reviews. Cardiology. 19 (10): 655–667. doi:10.1038/s41569-022-00683-z. hdl:11382/558829. PMID 35322226. S2CID 247623259.
  10. ^ Keam SJ (September 2022). "Vutrisiran: First Approval". Drugs. 82 (13): 1419–1425. doi:10.1007/s40265-022-01765-5. PMID 35997942. S2CID 251725617.
  11. ^ Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M (21 February 2020). "Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care". Therapeutics and Clinical Risk Management. 16: 109–123. doi:10.2147/TCRM.S219979. PMC 7041433. PMID 32110029.
  12. ^ a b Al Musaimi O, Al Shaer D, Albericio F, de la Torre BG (February 2023). "2022 FDA TIDES (Peptides and Oligonucleotides) Harvest". Pharmaceuticals. 16 (3): 336. doi:10.3390/ph16030336. PMC 10056021. PMID 36986436.