Biology:Vutrisiran
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| Trade names | Amvuttra |
| Other names | ALN-65492 |
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| Routes of administration | Subcutaneous |
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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 (hATTR) amyloidosis in adults.[1][3] It is a small interfering RNA (siRNA) that interferes with the expression of the transthyretin (TTR) gene.[4]
Vutrisiran was approved for medical use in the United States in June 2022,[1][3] and in the European Union in September 2022.[2]
History
The U.S. Food and Drug Administration (FDA) granted the application for vutrisiran orphan drug designation.[5]
Society and culture
Legal status
On 21 July 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Amvuttra, intended for treatment of hereditary transthyretin-mediated (hATTR) amyloidosis.[6] Amvuttra was designated as an orphan medicinal product on 25 May 2018.[6] The applicant for this medicinal product is Alnylam Netherlands B.V.[6] Vutrisiran was approved for medical use in the European Union in September 2022.[2][7]
Names
Vutrisiran is the international nonproprietary name (INN).[8]
References
- ↑ 1.0 1.1 1.2 "Amvuttra- vutrisiran injection". 21 June 2022. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8db0facb-81b6-4006-9239-27dc6409c5d3.
- ↑ 2.0 2.1 2.2 "Amvuttra EPAR". 18 July 2022. https://www.ema.europa.eu/en/medicines/human/EPAR/amvuttra. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ 3.0 3.1 "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. https://www.businesswire.com/news/home/20220603005487/en/Alnylam-Announces-FDA-Approval-of-AMVUTTRA%E2%84%A2-vutrisiran-an-RNAi-Therapeutic-for-the-Treatment-of-the-Polyneuropathy-of-Hereditary-Transthyretin-Mediated-Amyloidosis-in-Adults.
- ↑ "RNA-targeting and gene editing therapies for transthyretin amyloidosis". Nature Reviews. Cardiology 19 (10): 655–667. March 2022. doi:10.1038/s41569-022-00683-z. PMID 35322226.
- ↑ "Vutrisiran Orphan Drug Designations and Approvals". 25 May 2018. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=637618.
- ↑ 6.0 6.1 6.2 "Amvuttra: Pending EC decision". 21 July 2022. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/amvuttra. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "Amvuttra Product information". https://ec.europa.eu/health/documents/community-register/html/h1681.htm.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information 33 (1). 2019.
External links
- "Vutrisiran". Drug Information Portal. U.S. National Library of Medicine. https://druginfo.nlm.nih.gov/drugportal/name/vutrisiran.
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