Difference between revisions of "Etranacogene dezaparvovec (Hemgenix)"
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==Diseases for which it is used== | ==Diseases for which it is used== | ||
| − | *Hemophilia B | + | *[[Inherited coagulopathy|Hemophilia B]] |
==Patient drug information== | ==Patient drug information== | ||
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==History of changes in FDA indication== | ==History of changes in FDA indication== | ||
| − | *2022-11-22: [https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b | + | *2022-11-22: [https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b Approved] for the treatment of adults with [[Inherited coagulopathy|Hemophilia B (congenital Factor IX deficiency)]] who currently use [[:Category:Coagulation factors|Factor IX]] prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. |
==History of changes in EMA indication== | ==History of changes in EMA indication== | ||
| − | *2023-02-20: Granted conditional authorisation for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. | + | *2023-02-20: Granted conditional authorisation for the treatment of severe and moderately severe [[Inherited coagulopathy|Haemophilia B (congenital Factor IX deficiency)]] in adult patients without a history of Factor IX inhibitors. |
==Also known as== | ==Also known as== | ||
Latest revision as of 14:13, 5 June 2023
General information
Class/mechanism: adeno-associated virus vector-based gene therapy (adeno-associated virus serotype 5; AAV5) which delivers a gene encoding the Padua variant of human coagulation factor IX (hFIX-Padua) and results in the recipient producing circulating Factor IX.[1][2][3]
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias such as Micromedex, Lexicomp, Medscape, UpToDate (courtesy of Lexicomp), or the prescribing information.[1]
Diseases for which it is used
Patient drug information
- Etranacogene dezaparvovec (Hemgenix) package insert[1]
- Etranacogene dezaparvovec (Hemgenix) patient drug information (UpToDate)[4]
History of changes in FDA indication
- 2022-11-22: Approved for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
History of changes in EMA indication
- 2023-02-20: Granted conditional authorisation for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.
Also known as
- Generic name: etranacogene dezaparvovec-drlb