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 | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A.[1][3] It was developed by BioMarin Pharmaceutical.[4][5][6] 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.[3] It is an adeno-associated virus vector-based gene therapy.[1] It is given by intravenous infusion.[3]
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.[3]
Valoctocogene roxaparvovec was approved for medical use in the European Union in August 2022,[3][4] and in the United States in June 2023.[7][8]
- ^ a b c "Roctavian- valoctocogene roxaparvovec-rvox injection, solution". DailyMed. 20 December 2019. Retrieved 28 August 2023.
- ^ "Roctavian". U.S. Food and Drug Administration (FDA). 30 June 2023. Archived from the original on 5 July 2023. Retrieved 4 July 2023.
- ^ a b c d e f Cite error: The named reference
Roctavian EPARwas invoked but never defined (see the help page). - ^ a b c "Roctavian Product information". Union Register of medicinal products. 25 August 2022. Archived from the original on 22 May 2023. Retrieved 6 September 2022.
- ^ Cite error: The named reference
Roctavian: Pending EC decisionwas invoked but never defined (see the help page). - ^ "Roctavian (formerly Valrox/BMN 270)". BioNews Services, LLC. Archived from the original on 9 July 2021. Retrieved 1 July 2021.
- ^ "FDA Approves First Gene Therapy for Adults with Severe Hemophilia A". U.S. Food and Drug Administration (FDA) (Press release). 30 June 2023. Archived from the original on 29 June 2023. Retrieved 4 July 2023. This article incorporates text from this source, which is in the public domain.
- ^ "U.S. Food and Drug Administration Approves BioMarin's Roctavian (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A" (Press release). BioMarin Pharmaceutical. 29 June 2023. Retrieved 4 July 2023 – via PR Newswire.