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Review
. 2022 Sep;82(14):1505-1510.
doi: 10.1007/s40265-022-01788-y.

Valoctocogene Roxaparvovec: First Approval

Hannah A Blair  1
Affiliations
Review

Valoctocogene Roxaparvovec: First Approval

Hannah A Blair. Drugs. 2022 Sep.

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. - DOI - PubMed
    1. 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/ .
    1. 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/ .
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