Etranacogene dezaparvovec
- Generic Name
- Etranacogene dezaparvovec
- Brand Names
- Hemgenix
- Drug Type
- Biotech
- CAS Number
- 2156583-26-3
- Unique Ingredient Identifier
- Z5XCD5Q9RL
Overview
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. Clotting factors, including factor IX, are necessary components of the signaling cascade responsible for blood clotting and subsequent wound healing. 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. Hemophilia B is the second most common type of hemophilia, with a prevalence of approximately one in 40,000. Men are most likely to experience symptomatic illness due to the X-linked provenance of the disorder. 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. 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. It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity. 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. 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. 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. In February 2023, etranacogene dezaparvovec was approved by the EMA.
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. Clotting factors, including factor IX, are necessary components of the signaling cascade responsible for blood clotting and subsequent wound healing. 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. Hemophilia B is the second most common type of hemophilia, with a prevalence of approximately one in 40,000. Men are most likely to experience symptomatic illness due to the X-linked provenance of the disorder. 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. 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. It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity. 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. 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. 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. In February 2023, etranacogene dezaparvovec was approved by the EMA.
Indication
Etranacogene dezaparvovec (Hemgenix) is indicated for the treatment of adults with hemophilia B who fit one of the following criteria:
Associated Conditions
- Hemophilia B
- Moderately Severe Hemophilia B
- Severe Hemophilia B
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|
No clinical trials found
No clinical trials found for this drug
FDA Approved Products
Product Name | Manufacturer | Route | Strength | Approved | NDC Code |
|---|
No FDA products found
No FDA products found for this drug
EMA Approved Products
Medicine Name | EMA Number | Auth. Holder | Country | Drug Type | Status | Issued | Opinion | Revision |
|---|---|---|---|---|---|---|---|---|
EMEA/H/C/004827 | Germany | Orphan | Authorised | 2023/02/20 | 2022/12/15 | 6 |
HSA Approved Products
Product Name | Manufacturer | Dosage Form | Strength | Approved | Approval No. |
|---|
No HSA products found
No HSA products found for this drug
NMPA Approved Products
Product Name | Approval No. | Manufacturer | Dosage Form | Trade Name | Strength | Type | Status | Date | Import |
|---|
No NMPA products found
No NMPA products found for this drug
PPB Approved Products
Product Name | Registration Code | Company | Category | Sale Type | Reg. Date |
|---|---|---|---|---|---|
HK-68510 | Part 1, Schedule 1 & Schedule 3 Poison | POM | 2024/12/16 |
TGA Approved Products
Product Name | ARTG ID | Sponsor | Status | Reg. Date | Ingredient |
|---|---|---|---|---|---|
405360 | Active | 2024/03/19 | etranacogene dezaparvovec |