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Review
. 2023 Mar;83(4):347-352.
doi: 10.1007/s40265-023-01845-0.

Etranacogene Dezaparvovec: First Approval

Young-A Heo  1
Affiliations
Review

Etranacogene Dezaparvovec: First Approval

Young-A Heo. Drugs. 2023 Mar.

Abstract

Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; Hemgenix®) is an adeno-associated virus vector-based gene therapy being developed by uniQure and CSL Behring for the treatment of haemophilia B. In November 2022, etranacogene dezaparvovec was approved in the USA for the treatment of haemophilia B [congenital factor IX (FIX) deficiency] in adults who are currently using FIX prophylaxis therapy, have current or historical life-threatening haemorrhage or have repeated, serious spontaneous bleeding episodes. In December 2022, etranacogene dezaparvovec also received positive opinion in the EU for the treatment of haemophilia B. This article summarizes the milestones in the development of etranacogene dezaparvovec leading to this first approval.

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References

    1. Bolous NS, Bhatt N, Bhakta N, et al. Gene therapy and hemophilia: where do we go from here? J Blood Med. 2022;13:559–80. - DOI - PubMed - PMC
    1. Shah J, Kim H, Sivamurthy K, et al. Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B. Curr Med Res Opin. 2022;39(2):227–37. - DOI - PubMed
    1. Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis. 2021;2:1–14.
    1. US Food & Drug Administration. FDA approves first gene therapy to treat adults with hemophilia B [media release]. 22 Nov 2022. https://www.fda.gov/ .
    1. European Medicines Agency. First gene therapy to treat haemophilia B [media release]. 16 Dec 2022. https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b .

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