Etranacogene dezaparvovec

Etranacogene dezaparvovec
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	US DailyMed: Etranacogene dezaparvovec;
Pregnancy; category	AU: B2;
Routes of; administration	Intravenous infusion
ATC code	B02BD16 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only / Schedule D; US: ℞-only; EU: Rx-only;
Identifiers
CAS Number	2156583-26-3;
DrugBank	DB16791;
UNII	Z5XCD5Q9RL;
KEGG	D12500;

Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. 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.

The most common adverse reactions include liver enzyme elevations, headache, mild infusion-related reactions and flu-like symptoms.

Etranacogene dezaparvovec was approved for medical use in the United States in November 2022, in the European Union in February 2023. and in Canada in October 2023.