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Valoctocogene roxaparvovec (Roctavian°) in haemophilia A?

 Marketing Authorisations   The gene therapy product valoctocogene roxaparvovec consists of a non-replicating adeno-associated virus serotype 5 (AAV5)-based vector that contains a DNA sequence encoding a variant of human factor VIII. It has been authorised in the European Union for the treatment of adults with severe haemophilia A on the basis of weak non-comparative data that suggest efficacy, but which declines over time.
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EDITORS' OPINION
Reality

Gene therapy is based on an attractive concept: the idea that if a disorder is caused by an alteration in a gene, then replacing the faulty gene might cure the disease. But in reality, after it is administered to the patient, how long will the "copy" of the gene result in synthesis of the functional protein?...

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©Prescrire 1 January 2025

Source: "Valoctocogene roxaparvovec (Roctavian°) in haemophilia A" Prescrire Int 2025; 34 (266): 10-11. Subscribers only.

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