| Gene therapy | |
|---|---|
| Target gene | Factor IX |
| Vector | Adeno-associated virus |
| Nucleic acid type | DNA |
| Delivery method | Intravenous |
| Clinical data | |
| Trade names | Hemgenix |
| Other names | AMT-061, etranacogene dezaparvovec-drlb |
| License data | |
| Pregnancy category |
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| Routes of administration | Intravenous infusion |
| ATC code | |
| Legal status | |
| Legal status | |
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Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B.[5][6][7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX.[7] The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes.[7] Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.[7]
The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions and flu-like symptoms.[7][8]
Etranacogene dezaparvovec was approved for medical use in the United States in November 2022,[6][7][9] in the European Union in February 2023.[8] and in Canada in October 2023.[2]
- ^ a b "Hemgenix APMDS". Therapeutic Goods Administration (TGA). 29 May 2024. Retrieved 10 June 2024.
- ^ a b "Hemgenix Product information". Health Canada. 22 October 2009. Retrieved 3 March 2024.
- ^ "Summary Basis of Decision for Hemgenix". Health Canada. 5 May 2024. Retrieved 8 June 2024.
- ^ "Details for: Hemgenix". Health Canada. 23 October 2023. Retrieved 3 March 2024.
- ^ a b "Hemgenix- etranacogene dezaparvovec kit". DailyMed. 13 January 2023. Retrieved 21 January 2023.
- ^ a b c "Hemgenix". U.S. Food and Drug Administration (FDA). 22 November 2022. Retrieved 22 November 2022.
- ^ a b c d e f g "FDA Approves First Gene Therapy to Treat Adults with Hemophilia B". U.S. Food and Drug Administration (FDA) (Press release). 22 November 2022. Retrieved 22 November 2022.
This article incorporates text from this source, which is in the public domain.
- ^ a b c Cite error: The named reference
Hemgenix EPARwas invoked but never defined (see the help page). - ^ "U.S. Food and Drug Administration approves CSL's Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B". CSL (Press release). 22 November 2022. Retrieved 22 November 2022.
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