Skip to main content
SpringerLink
Log in

Lumasiran: First Approval

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

Abstract

Lumasiran (Oxlumo™) is a subcutaneously administered small interfering RNA (siRNA) targeting the mRNA for hydroxyacid oxidase 1 gene (HAO1; encodes glycolate oxidase) and was developed by Alnylam Pharmaceuticals for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1. On 19 November 2020, lumasiran received its first global approval in the EU for the treatment of PH1 in all age groups. On 23 November 2020, lumasiran was approved in the USA for the treatment of adult and paediatric patients with PH1. This article summarizes the milestones in the development of lumasiran leading to this first approval.

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. Liebow A, Li X, Racie T, et al. An investigational RNAi therapeutic targeting glycolate oxidase reduces oxalate production in models of primary hyperoxaluria. J Am Soc Nephrol. 2017;28(2):494–503.

    Article  CAS  Google Scholar 

  2. Cochat P, Rumsby G. Primary hyperoxaluria. N Engl J Med. 2013;369(7):649–58.

    Article  CAS  Google Scholar 

  3. Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov. 2020;19(10):673–94.

    Article  CAS  Google Scholar 

  4. Debacker AJ, Voutila J, Catley M, et al. Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug. Mol Ther. 2020;28(8):1759–71.

    Article  CAS  Google Scholar 

  5. European Commission. Commission implementing decision of 19.11.20 granting marketing authorisation under Regulation (EC) No 726/2004 of the European Parliament and of the Council for "Oxlumo—lumasiran, an orphan medicinal product for human use. 2020. http://ec.europa.eu/health/documents/community-register/html/h1496.htm. Accessed 24 Nov 2020.

  6. US FDA. FDA approves first drug to treat rare metabolic disorder. 2020. http://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-rare-metabolic-disorder?utm_medium=email&utm_source=govdelivery. Accessed 25 Nov 2020.

  7. European Medicines Agency. Oxlumo 94.5 mg/0.5 mL, solution for injection: summary of product characteristics. 2020. http://ec.europa.eu/health/documents/community-register/2020/20201119149780/anx_149780_en.pdf. Accessed 24 Nov 2020.

  8. Alnylam Pharmaceuticals. Oxlumo (lumasiran) injection, for subcutaneous use: US prescribing information. 2020. http://www.alnylam.com/wp-content/uploads/pdfs/OXLUMO-Prescribing-Information.pdf. Accessed 25 Nov 2020.

  9. Alnylam Pharmaceuticals. Alnylam Pharmaceuticals and Taiba Group partner to commercialize RNAi therapeutics in the Gulf States [media release]. 8 July 2020. https://investors.alnylam.com/press-release?id=24996.

  10. Alnylam Pharmaceuticals, Dicerna Pharmaceuticals. Alnylam and Dicerna form RNAi therapeutics collaboration on alpha-1 antitrypsin deficiency-associated liver disease and complete cross-license agreement for primary hyperoxaluria programs [media release]. 6 Apr 2020. https://investors.alnylam.com/press-release?id=24701.

  11. Alnylam Pharmaceuticals. Alnylam Pharmaceuticals and Medison Pharma partner to commercialize RNAi therapeutics in Israel [media release]. 21 Jan 2019. https://www.businesswire.com/news/home/20190121005029/en/Alnylam-Pharmaceuticals-and-Medison-Pharma-Partner-to-Commercialize-RNAi-Therapeutics-in-Israel.

  12. Frishberg Y, Deschenes G, Groothoff J, et al. Final results from the phase 1/2 trial of lumasiran and program updates. 2019. https://www.alnylam.com/wp-content/uploads/2019/06/Luma_OHF-Ph12_Capella_FINAL.pdf. Accessed 14 Dec 2020.

  13. Frishberg Y, Hulton S, Cochat P, et al. Results from the ongoing phase 2 open-label extension study of lumasiran, an investigational RNAi therapeutic, in patients with primary hyperoxaluria type 1 (PH1) [abstract no. PO1647 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):520–1.

  14. Saland J, Groothoff J, Frishberg Y, et al. 12-Month analysis of ILLUMINATE-A, a phase 3 study of lumasiran: sustained oxalate lowering and kidney stone event rates in primary hyperoxaluria type 1 [abstract no. PO2637 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):B8.

  15. Michael M, Deschênes G, Cochat P, et al. ILLUMINATE-B, a phase 3 open-label study to evaluate lumasiran, an RNAi therapeutic, in young children with primary hyperoxaluria type 1 (PH1) [abstract no. PO1624 and presentation]. J Am Soc Nephrol. 2020;31(Abstract Suppl):515.

  16. van't Hoff W, Cochat P, Groothoff J, et al. Safety and efficacy of lumasiran, an investigational RNA interference (RNAi) therapeutic, in adult and pediatric patients with primary hyperoxaluria type 1 [abstract no. SUN-325]. Kidney Internat Rep. 2019;4(7 Suppl):S295.

  17. Garrelfs S, Frishberg Y, Hulton S, et al. ILLUMINATE-A, A phase 3 study of lumasiran, an investigational RNAi therapeutic, in children and adults with primary hyperoxaluria type 1 (PH1) [abstract no. LB002 and presentation]. In: Nephrology Dialysis Transplantation. 2020.

Download references

Author information

Authors and Affiliations

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

    Lesley J. Scott & Susan J. Keam

Authors
  1. Lesley J. Scott
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Susan J. Keam
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Lesley J. Scott.

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. Lesley Scott and Susan Keam are salaried employees of Adis International Ltd/Springer Nature, and declare 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

Digital Features for this Adis Insight Report can be found at https://doi.org/10.6084/m9.figshare.13377347.

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.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Scott, L.J., Keam, S.J. Lumasiran: First Approval. Drugs 81, 277–282 (2021). https://doi.org/10.1007/s40265-020-01463-0

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40265-020-01463-0

Search

Navigation