Valoctocogene roxaparvovec
- Generic Name
- Valoctocogene roxaparvovec
- Brand Names
- Roctavian
- Drug Type
- Biotech
- CAS Number
- 1819334-78-5
- Unique Ingredient Identifier
- 681K1JDI8M
Overview
Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). 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. Valoctocogene roxaparvovec was approved by EMA in September 2022 and is indicated for the treatment of severe hemophilia A. It is not approved for use in the United States. Hemophilia A treatments such as prophylactic regimens of exogenous factor VIII or emicizumab improve the clinical outcomes of patients but do not eliminate breakthrough bleeding. As opposed to these therapies, valoctocogene roxaparvovec offers the advantage of continuous and measurable steady-state levels of coagulation factor VIII.
Background
Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). 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. Valoctocogene roxaparvovec was approved by EMA in September 2022 and is indicated for the treatment of severe hemophilia A. It is not approved for use in the United States. Hemophilia A treatments such as prophylactic regimens of exogenous factor VIII or emicizumab improve the clinical outcomes of patients but do not eliminate breakthrough bleeding. As opposed to these therapies, valoctocogene roxaparvovec offers the advantage of continuous and measurable steady-state levels of coagulation factor VIII.
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).
Associated Conditions
- Severe Hemophilia A
Clinical Trials
FDA Approved Products
Singapore Approved Products
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