Valoctocogene roxaparvovec
- Generic Name
- Valoctocogene roxaparvovec
- Brand Names
- Roctavian
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1819334-78-5
- Unique Ingredient Identifier
- 681K1JDI8M
- 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...
- 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
- Associated Therapies
- -
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