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Valoctocogene Roxaparvovec: First Approval

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Abstract

Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy being developed by BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In August 2022, valoctocogene roxaparvovec was granted conditional marketing authorization in the EU for the treatment of severe haemophilia A [congenital factor VIII (FVIII) deficiency] in adults without a history of FVIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). This article summarizes the milestones in the development of valoctocogene roxaparvovec leading to this first approval for severe haemophilia A.

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References

  1. Fassel H, McGuinn C. Haemophilia: factoring in new therapies. Br J Haematol. 2021;194(5):835–50.

    Article  PubMed  Google Scholar 

  2. BioMarin Pharmaceutical Inc. First gene therapy for adults with severe hemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), approved by European Commission (EC) [media release]. 24 Aug 2022. https://investors.biomarin.com/.

  3. BioMarin Pharmaceutical Inc. BioMarin receives European orphan drug designation for BMN 270, first investigational AAV-factor VIII gene therapy for patients with hemophilia A [media release]. 24 Mar 2016. https://www.biomarin.com/.

  4. BioMarin Pharmaceutical Inc. BioMarin receives orphan drug designation from FDA for first AAV-factor VIII gene therapy, BMN 270, for patients with hemophilia A [media release]. 1 Mar 2016. https://www.biomarin.com/.

  5. BioMarin Pharmaceutical Inc. BioMarin receives access to priority medicines (PRIME) regulatory support from EMA for BMN 270 gene therapy in hemophilia A [media release]. 1 Feb 2017. https://www.biomarin.com/.

  6. BioMarin Pharmaceutical Inc. FDA grants breakthrough therapy designation for BioMarin's valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy for hemophilia A [media release]. 26 Oct 2017. https://www.biomarin.com/.

  7. BioMarin Pharmaceutical Inc. BioMarin announces FDA Regenerative Medicine Advanced Therapy (RMAT) designation granted to valoctocogene roxaparvovec, investigational gene therapy for hemophilia A [media release]. 8 Mar 2021. https://www.biomarin.com/.

  8. BioMarin International Ltd. Valoctocogene roxaparvovec (ROCTAVIAN™): EU summary of product characteristics. 2022. https://ec.europa.eu/health/documents/community-register/2022/20220824156641/anx_156641_en.pdf. Accessed 12 Sep 2022.

  9. BioMarin Pharmaceutical Inc. BioMarin licenses factor VIII gene therapy program for hemophilia A from University College London and St. Jude Children's Research Hospital [media release]. 21 Feb 2013. https://www.biomarin.com/.

  10. Bunting S, Zhang L, Xie L, et al. Gene therapy with BMN 270 results in therapeutic levels of FVIII in mice and primates and normalization of bleeding in hemophilic mice. Mol Ther. 2018;26(2):496–509.

    Article  CAS  PubMed  Google Scholar 

  11. Sihn CR, Handyside B, Liu S, et al. Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers. Mol Ther Methods Clin Dev. 2022;24:142–53.

    Article  CAS  PubMed  Google Scholar 

  12. Long BR, Sandza K, Holcomb J, et al. The impact of pre-existing immunity on the non-clinical pharmacodynamics of AAV5-based gene therapy. Mol Ther Methods Clin Dev. 2019;13:440–52.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  13. Fonck C, Su C, Arens J, et al. Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy. Gene Ther. 2022. https://doi.org/10.1038/s41434-022-00318-5.

    Article  PubMed  Google Scholar 

  14. Zhang L, Handyside B, Murphy R, et al. Prednisolone does not regulate factor VIII expression in mice receiving AAV5-hFVIII-SQ: valoctocogene roxaparvovec. Mol Ther Methods Clin Dev. 2020;17:13–20.

    Article  CAS  PubMed  Google Scholar 

  15. Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med. 2022;386(11):1013–25.

    Article  CAS  PubMed  Google Scholar 

  16. Rangarajan S, Walsh L, Lester W, et al. AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 2017;377(26):2519–30.

    Article  CAS  PubMed  Google Scholar 

  17. Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N Engl J Med. 2020;382(1):29–40.

    Article  CAS  PubMed  Google Scholar 

  18. Pasi KJ, Laffan M, Rangarajan S, et al. Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A. Haemophilia. 2021;27(6):947–56.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  19. Long BR, Veron P, Kuranda K, et al. Early phase clinical immunogenicity of valoctocogene roxaparvovec, an AAV5-mediated gene therapy for hemophilia A. Mol Ther. 2021;29(2):597–610.

    Article  CAS  PubMed  Google Scholar 

  20. BioMarin Pharmaceutical Inc. FDA files ARUP Laboratories' AAV5 CDx for hemophilia A gene therapy treatment [media release]. 20 Feb 2020. https://www.biomarin.com/.

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

    Hannah A. Blair

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  1. Hannah A. Blair
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Correspondence to Hannah A. Blair.

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

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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. Hannah Blair is a salaried 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.

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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.

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Blair, H.A. Valoctocogene Roxaparvovec: First Approval. Drugs 82, 1505–1510 (2022). https://doi.org/10.1007/s40265-022-01788-y

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