Etranacogene dezaparvovec
Identification
- Summary
Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy used for the treatment of hemophilia B (congenital factor IX deficiency).
- Generic Name
- Etranacogene dezaparvovec
- DrugBank Accession Number
- DB16791
- Background
Hemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX.3 Clotting factors, including factor IX, are necessary components of the signaling cascade responsible for blood clotting and subsequent wound healing.4 Symptoms of hemophilia B, therefore, involve a heightened susceptibility to bleeding episodes - in mild cases, bleeding may only occur after injury, while in more severe cases bleeding may occur after a minor injury or even spontaneously.4
Hemophilia B is the second most common type of hemophilia,4 with a prevalence of approximately one in 40,000.2 Men are most likely to experience symptomatic illness due to the X-linked provenance of the disorder.2 Treatment of hemophilia B primarily involves the routine replacement of factor IX using recombinant or donor-derived factor IX products that, while effective, may be burdensome for the patient due to the requirement of routine intravenous infusions.2,4
Etranacogene dezaparvovec (Hemgenix, CSL Bering LLC) is a gene therapy for the treatment of hemophilia B that provides a new treatment modality for its patients. The therapy involves a one-time infusion of a viral vector carrying a codon-optimized DNA sequence of the gain-of-function Padua variant of human Factor IX controlled by a liver-specific promotor 1.1 It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.1 Etranacogene dezaparvovec was approved by the FDA in November 2022 for the treatment of select patients with hemophilia B, becoming the first gene therapy approved for this indication.2 It is additionally notable for its cost per treatment - approximately 3.5 million USD - earning it the title of most expensive drug in the world.5 In December 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended etranacogene dezaparvovec be granted marketing authorization for the treatment of severe and moderately severe Haemophilia B.6 Etranacogene dezaparvovec was approved by the EMA in February 20237,8 and by Health Canada in October 2023.9
- Type
- Biotech
- Groups
- Approved, Investigational
- Biologic Classification
- Gene Therapies
Other gene therapies - Synonyms
- AAV5-hFIXco-Padua
- Etranacogene dezaparvovec
- External IDs
- AMT-061
Pharmacology
- Indication
Etranacogene dezaparvovec (Hemgenix) is indicated in the United States for the treatment of adults with hemophilia B who fit one of the following criteria:1
- Currently use factor IX prophylaxis therapy
- Have current or historical life-threatening hemorrhage
- Have repeated, serious spontaneous bleeding episodes
In the EU, etranacogene dezaparvovec for the treatment of severe and moderately severe hemophilia B in adult patients without a history of Factor IX inhibitors.8 In Canada, etranacogene dezaparvovec is indicated for adult patients with hemophilia B who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.10
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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Treatment of Hemophilia b •••••••••••• ••••• ••••• •••••• •• ••••••••••• ••••••• •••••••••• •••••••••• Treatment of Hemophilia b •••••••••••• ••••• ••••• •••••• •• ••••••••••• ••••••• •••••••••• •••••••••• Treatment of Hemophilia b •••••••••••• ••••• ••••••• ••••••••• •••••••• •••••••• •••••••••• •••••••••• Treatment of Hemophilia b •••••••••••• ••••• ••••••• •• ••••••• •••••••••• •••••••••• •••••••••• Treatment of Hemophilia b •••••••••••• ••••• ••••••• •••••••••• •••••••••• •••••••••• - Contraindications & Blackbox Warnings
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- Pharmacodynamics
Following a single intravenous infusion, median factor IX activity increased to 23.7% of normal by week 3, 33.8% at 3 months, 37.3% at 6 months, and peaked at 39.9% at 12 months.1 After 24 months, median activity remained at 33.9% of normal.1
- Mechanism of action
Etranacogene dezaparvovec is a non-replicating recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized DNA sequence of the gain-of-function Padua variant of human Factor IX (variant R338L) controlled by a liver-specific promotor 1.1 It is administered by a single intravenous infusion and delivers a copy of the gene in which hemophilia B patients are defective, leading to cell transduction and an eventual increase in circulating activity levels of factor IX.1
- Absorption
Not Available
- Volume of distribution
In a study to evaluate the biodistribution of etranacogene dezaparvovec in male mice and non-human primates, the highest levels of vector DNA were detected in the liver and adrenal glands, although it was also detected in all reproductive tissues examined.1
- Protein binding
Not Available
- Metabolism
- Not Available
- Route of elimination
Not Available
- Half-life
Not Available
- Clearance
In a clinical study, the maximum time to clearance of vector DNA was 22 weeks for urine, 26 weeks for saliva and nasal secretions, 40 weeks for feces, 52 weeks for semen, and 159 weeks for blood.1
- Adverse Effects
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- Toxicity
Not Available
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- No interactions found.
Products
- Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Hemgenix Injection, suspension; Kit 1013 {GC}/1 Intravenous Csl Behring 2022-11-22 Not applicable US Hemgenix Injection, suspension; Kit 1013 {GC}/1 Intravenous Csl Behring 2022-11-22 Not applicable US Hemgenix Injection, suspension; Kit 1013 {GC}/1 Intravenous Csl Behring 2022-11-22 Not applicable US Hemgenix Injection, suspension; Kit 1013 {GC}/1 Intravenous Csl Behring 2022-11-22 Not applicable US Hemgenix Injection, suspension; Kit 1013 {GC}/1 Intravenous Csl Behring 2022-11-22 Not applicable US
Categories
- Drug Categories
- Classification
- Not classified
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- Z5XCD5Q9RL
- CAS number
- 2156583-26-3
References
- General References
- FDA Approved Drug Products: Hemgenix (etranacogene dezaparvovec-drlb) suspension for intravenous infusion [Link]
- FDA News Release: FDA Approves First Gene Therapy to Treat Adults with Hemophilia B [Link]
- National Hemophilia Foundation: Hemophilia B [Link]
- National Organization for Rare Disorders (NORD): Hemophilia B [Link]
- CNN: FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world [Link]
- EMA Summary of Opinion: Hemgenix (etranacogene dezaparvovec) [Link]
- PR NewsWire: First Gene Therapy for Hemophilia B, CSL's HEMGENIX, Approved by the European Commission [Link]
- EMA Summary of Product Characteristics: HEMGENIX (etranacogene dezaparvovec) concentrate for solution for infusion [Link]
- Canadian Hemophilia Society (CHS) Contact: Health Canada approves Hemgenix gene therapy for hemophilia B [Link]
- Health Canada Product Monograph: Hemgenix (etranacogene dezaparvovec) suspension for intravenous infusion [Link]
- External Links
- 2626727
- Wikipedia
- Etranacogene_dezaparvovec
Clinical Trials
- Clinical Trials
Phase Status Purpose Conditions Count 2 Completed Treatment Hemophilia B 1 1, 2 Completed Treatment Hemophilia B 1
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Injection, solution, concentrate Intravenous 10000000000000 gc/ml Injection, suspension; kit Intravenous 1013 {GC}/1 Suspension Intravenous 10000000000000 genome copies / mL - Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Liquid
- Experimental Properties
- Not Available
Drug created at May 10, 2022 20:13 / Updated at November 25, 2023 12:39