Skip to main content
SpringerLink
Log in

Vutrisiran: First Approval

  • AdisInsight Report
  • Published:
Drugs Aims and scope Submit manuscript

Abstract

Vutrisiran (AMVUTTRA™) is a subcutaneously administered transthyretin-directed small interfering ribonucleic acid (siRNA) therapeutic (also called RNA interference, or RNAi therapeutic) being developed by Alnylam Pharmaceuticals, Inc. for the treatment of amyloid transthyretin-mediated (ATTR) amyloidosis, including hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis. Vutrisiran was approved in June 2022 in the USA for the treatment of the polyneuropathy of hATTR amyloidosis in adults and received a positive opinion in the EU in July 2022 for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Vutrisiran is also under regulatory review for the treatment of the polyneuropathy of hATTR amyloidosis in adults in Japan and Brazil. This article summarizes the milestones in the development of vutrisiran leading to this first approval in hATTR amyloidosis.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Similar content being viewed by others

References

  1. Aimo A, Castiglione V, Rapezzi C, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol. 2022. https://doi.org/10.1038/s41569-022-00683-z.

    Article  PubMed  Google Scholar 

  2. Ando Y, Adams D, Benson MD, et al. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid. 2022. https://doi.org/10.1080/13506129.2022.2052838.

    Article  PubMed  Google Scholar 

  3. Habtemariam BA, Karsten V, Attarwala H, et al. Single-dose pharmacokinetics and pharmacodynamics of transthyretin targeting N-acetylgalactosamine-small interfering ribonucleic acid conjugate, vutrisiran, in healthy subjects. Clin Pharmacol Ther. 2021;109(2):372–82.

    Article  CAS  Google Scholar 

  4. Tschöpe C, Elsanhoury A. Treatment of transthyretin amyloid cardiomyopathy: the current options, the future, and the challenges. J Clin Med. 2022;11(8):2148.

    Article  Google Scholar 

  5. Luigetti M, Romano A, Di Paolantonio A, et al. Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: current perspectives on improving patient care. Ther Clin Risk Manag. 2020;16:109–23.

    Article  CAS  Google Scholar 

  6. Alnylam Pharmaceuticals Inc. Hereditary ATTR amyloidosis; 2021. https://www.hattramyloidosis.com/. Accessed 4 Jul 2022.

  7. Alnylam Pharmaceuticals Inc. AMVUTTRA (vutrisiran): US prescribing information; 2022. https://www.amvuttra.com/. Accessed 27 June 2022

  8. Alnylam Pharmaceuticals Inc. Alnylam announces FDA approval of AMVUTTRA™ (vutrisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults [media release]. 13 June 2022. https://investors.alnylam.com/.

  9. European Medicines Agency. Amvuttra (vutrisiran): Committee for Medicinal Products for Human Use (CHMP) summary of opinion; 2022. https://www.ema.europa.eu/en/documents/smop-initial/chmp-summary-positive-opinion-amvuttra_en.pdf. Accessed 28 Jul 2022.

  10. Alnylam Pharmaceuticals Inc. Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy [media release]. 22 Jul 2022. https://investors.alnylam.com/.

  11. Alnylam Pharmaceuticals Inc. Alnylam announces new advances in ATTR amyloidosis program [media release]. 11 May 2021. http://www.alnylam.com.

  12. Shilling R, Karsten V, Silliman N, et al. Study design and rationale of HELIOS-B: a phase 3 study to evaluate the clinical efficacy and safety of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy [abstract no. 1323–359]. J Am Coll Cardiol. 2020;75(11 Suppl. 1):3579.

  13. Blackstone Life Sciences. Blackstone and Alnylam close $150 million R&D financing to advance RNAi therapeutics for cardiovascular disease [media release]. 17 Aug 2020. http://www.blackstone.com.

  14. Blackstone Life Sciences, Alnylam Pharmaceuticals Inc. Blackstone and Alnylam enter into $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics [media release]. 13 April 2020. http://www.blackstone.com.

  15. Sanofi, Alnylam Pharmaceuticals Inc. Sanofi and Alnylam conclude research and option phase of 2014 rare disease alliance [media release]. 8 April 2019. http://www.sanofi.com.

  16. Alnylam Pharmaceuticals Inc. Alnylam and Sanofi enter into strategic restructuring of RNAi therapeutics rare disease alliance [media release]. 7 Jan 2018. https://investors.alnylam.com/.

  17. Genzyme, Alnylam Pharmaceuticals Inc. Genzyme and Alnylam expand collaboration on rare genetic diseases [media release]. 13 Jan 2014. http://www.genzyme.com.

  18. Alnylam Pharmaceuticals Inc. Genzyme exercises its right to purchase additional shares of Alnylam common stock [media release]. 26 Mar 2014. http://www.alnylam.com.

  19. Alnylam Pharmaceuticals Inc. Alnylam announces closing of previously announced alliance with Genzyme for discovery, development, and commercialization of RNAi therapeutics as genetic medicines [media release]. 27 Feb 2014. http://www.alnylam.com.

  20. Alnylam Pharmaceuticals Inc., Genzyme. Alnylam and Genzyme form alliance to develop and commercialize RNAi therapeutics in Asia [media release]. 22 Oct 2012. http://www.alnylam.com.

  21. Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyrethrin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2022. https://doi.org/10.1080/13506129.2022.2091985.

    Article  PubMed  Google Scholar 

  22. Garcia-Pavia P., Gillmore J, Kale P, et al. HELIOS-A: 18-month exploratory cardiac results from the phase 3 study of vutrisiran in patients with hereditary transthyretin-mediated amyloidosis. In: Heart Failure 2022 and World Congress on Heart Failure; 2022.

  23. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):11–21.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand

    Susan J. Keam

Authors
  1. Susan J. Keam
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Susan J. Keam.

Ethics declarations

Funding

The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest

During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Susan J. Keam is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability

Not applicable.

Additional information

This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

Supplementary Information

Below is the link to the electronic supplementary material.

Supplementary file1 (PPTX 503 KB)

Rights and permissions

Springer Nature or its licensor holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Keam, S.J. Vutrisiran: First Approval. Drugs 82, 1419–1425 (2022). https://doi.org/10.1007/s40265-022-01765-5

Download citation

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40265-022-01765-5

Search

Navigation