Valoctocogene roxaparvovec
| Gene therapy | |
|---|---|
| Target gene | Factor VIII |
| Vector | Adeno-associated virus |
| Nucleic acid type | DNA |
| Delivery method | Intravenous |
| Clinical data | |
| Trade names | Roctavian |
| Other names | BMN-270, Valrox, valoctocogene roxaparvovec-rvox |
| License data | |
| Routes of administration | Intravenous |
| Drug class | Antihemorrhagics |
| ATC code | |
| Legal status | |
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| Identifiers | |
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Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. It was developed by BioMarin Pharmaceutical. Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with hemophilia A. It is an adeno-associated virus vector-based gene therapy. It is given by intravenous infusion.
The most common side effects include increased levels of the liver enzymes alanine aminotransferase and aspartate aminotransferase (signs of possible liver problems), increased levels of the enzyme lactate dehydrogenase (sign of possible tissue damage), nausea (feeling sick), and headache.
Valoctocogene roxaparvovec was approved for medical use in the European Union in August 2022, and in the United States in June 2023.