Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5)-based gene therapy containing a coagulation factor VIII complementary DNA used to treat severe hemophilia A. The expression of hFVIII-SQ is driven by a liver-specific promoter, which enables hepatocytes to produce factor VIII protein and increase the levels of active factor VIII in blood.
- Mechanism
- Curator reviewed · 4 references
Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-specific promoter; and is used to treat hemophilia A, a condition characterized by the deficient activity of coagulation factor VIII. After valoctocogene roxaparvovec is infused into patients, the AAV5 vector delivers a B-domain deleted SQ form of a recombinant human factor VIII (hFVIII-SQ) to cells. This working copy of coagulation factor VIII is then expressed in the liver, producing an active form of factor VIII that is then released into the bloodstream. This leads to bleeding normalization and a lower risk of bleeding episodes. The long-term production of hFVIII-SQ is supported following valoctocogene roxaparvovec infusion.
- Primary indication
- Valoctocogene roxaparvovec is indicated for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).Curator reviewed · 2 structured indications
- First approval
- United States, 2023
- Code names
- BMN-270
Resolves to
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