Vutrisiran

Vutrisiran
Names
Trade namesAmvuttra
Other namesALN-65492
Clinical data
Main usesPolyneuropathy in hereditary transthyretin-mediated (hATTR) amyloidosis[1]
Side effectsJoint pain, shortness of breath, vitamin A deficiency[1]
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Routes of
use		Subcutaneous
Typical dose25 mg q 3 months[2]
External links
AHFS/Drugs.comMonograph
Legal
License data
Legal status

Vutrisiran, sold under the brand name Amvuttra, is a medication used to treat polyneuropathy in hereditary transthyretin-mediated (hATTR) amyloidosis.[1] It is used in adults who are still able to walk.[2] It is used by injection under the skin.[1]

Common side effects include joint pain, shortness of breath, and vitamin A deficiency.[1] Vitamin A supplements are recommended.[1] It is a small interfering RNA (siRNA) that decreases expression of the transthyretin (TTR) gene.[1][3]

Vutrisiran was approved for medical use in the United States and Europe in 2022.[1][2] In the United States it costs about 244,000 USD per dose.[4] It is made by Alnylam Pharmaceuticals.[5]

Medical uses

Dosage

It is generally used at a dose of 25 mg every 3 months.[2]

History

The U.S. Food and Drug Administration (FDA) granted the application for vutrisiran orphan drug designation.[6]

Society and culture

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.[7] Amvuttra was designated as an orphan medicinal product on 25 May 2018.[7] The applicant for this medicinal product is Alnylam Netherlands B.V.[7] Vutrisiran was approved for medical use in the European Union in September 2022.[2]

Names

Vutrisiran is the international nonproprietary name (INN).[8]

References

  1. 1 2 3 4 5 6 7 8 9 "Amvuttra- vutrisiran injection". DailyMed. 21 June 2022. Archived from the original on 3 July 2022. Retrieved 3 July 2022.
  2. 1 2 3 4 5 6 "Amvuttra EPAR". European Medicines Agency. 18 July 2022. Archived from the original on 13 October 2022. Retrieved 13 October 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  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. ↑ "Amvuttra Prices, Coupons, Copay & Patient Assistance". Drugs.com. Archived from the original on 8 September 2022. Retrieved 12 December 2022.
  5. ↑ "Vutrisiran". SPS - Specialist Pharmacy Service. 11 January 2021. Archived from the original on 14 October 2022. Retrieved 13 December 2022.
  6. ↑ "Vutrisiran Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 25 May 2018. Archived from the original on 14 June 2022. Retrieved 14 June 2022.
  7. 1 2 3 "Amvuttra: Pending EC decision". European Medicines Agency. 21 July 2022. Archived from the original on 28 July 2022. Retrieved 30 July 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  8. ↑ World Health Organization (2019). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 81". WHO Drug Information. 33 (1). hdl:10665/330896.
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