Gene therapy | |
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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 |
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Routes of administration | Intravenous infusion |
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Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B.[4][5][6] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX.[6] The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes.[6] 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.[6]
The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions and flu-like symptoms.[6][7]
Etranacogene dezaparvovec was approved for medical use in the United States in November 2022,[5][6][8] in the European Union in February 2023.[7] and in Canada in October 2023.[1]
Hemgenix EPAR
was invoked but never defined (see the help page).