Etranacogene Dezaparvovec: Uses, Side Effects, Dosage - Drugvu

ℹ️ Compiled from public regulatory records · Last regulator revision: May 22, 2026🚩 Report this page

Etranacogene Dezaparvovec

Blood Coagulation Factors

Sold as Hemgenix

GB MHRAEU EMACA Health Canada

Drug class: Blood Coagulation Factors
Availability: See label
Routes: Intravenous
Markets covered: 3
Products on record: 3

Overview

Etranacogene Dezaparvovec is an active pharmaceutical ingredient in the Blood Coagulation Factors group (B02BD). The information below is compiled per regulator from the product labels on record, with direct links to the original documents.

Regulatory status by market

Market	Regulator	Products	Last revision
GB United Kingdom	MHRA	1	April 24, 2026
EU European Union	EMA	1	March 4, 2026
CA Canada	Health Canada	1	May 22, 2026

GBUnited Kingdom· MHRA

1 product

Uses

GBOfficial regulatory label· revised April 24, 2026[1]

Hemgenix is indicated for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.

How to take

EUEuropean Union· EMA

1 product

Uses

EUOfficial regulatory label· revised March 4, 2026[2]

Hemgenix is indicated for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.

How to take

CACanada· Health Canada

1 product

1 product on record with this regulator. Detailed label text (uses, dosage, side effects) is being ingested — the original document is linked under Sources [3].

Brands in Canada (1)

HEMGENIXCSL BEHRING CANADA INC

Sources & citations

[1]MHRA (UK) · PLGB150360160 · revised April 24, 2026
[2]European Medicines Agency · EMEA/H/C/004827 · revised March 4, 2026
[3]Health Canada (DPD) · 02542560 · revised May 22, 2026

Information on this page is compiled from public regulatory records. Drugvu is not affiliated with any regulator or pharmaceutical manufacturer. This is not medical advice. Always consult a qualified healthcare professional.

Side effects & warnings

GBOfficial regulatory label· Adverse reactions· revised April 24, 2026[1]

5% of patients), and influenza-like illness (very common; 14% of patients). 1). The Table 3 shows the overview of the ADRs from the clinical trials with etranacogene dezaparvovec. The ADRs are classified according the MedDRA System Organ Class and frequency.
The ADRs are listed based on the following convention for frequency categories: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000), and not known (cannot be estimated from the available data).
Within each frequency category, adverse reactions are presented in order of decreasing frequency. Table 3. Adverse drug reactions obtained from clinical studies with etranacogene dezaparvovec MedDRA System Organ Class (SOC) ADR (Preferred term) Frequency per patient Headache Very commonNervous system disorders Dizziness Common Gastrointestinal disorders Nausea Common Influenza like illness Very commonGeneral disorders and administration site conditions Fatigue, malaise Common Alanine aminotransferase increased, aspartate aminotransferase increased, C- reactive protein increased Very commonInvestigations Blood creatine phosphokinase increased, blood bilirubin increased Common Injury, poisoning and procedural complications Infusion related reaction (Hypersensitivity, infusion site reaction, dizziness, eye pruritus, flushing, abdominal pain upper, urticaria, chest discomfort, pyrexia) Very common* *The frequency results from pooled infusion related reactions of similar medical concept.
5%) within 24 hours post-dose. Hepatic laboratory abnormalities Table 4 describes hepatic laboratory abnormalities following administration of Hemgenix. 4). Table 4. 1%) Abbreviations: ULN = Upper Limit of Normal; CTCAE = Common Terminology Criteria for Adverse Events aHighest post-dose CTCAE Grades of values are presented bNot all patients with laboratory abnormality > ULN reached CTCAE Grade 1 due to elevated baseline levels cCTCAE Grade 1 dCTCAE Grade 2 eCTCAE Grade 3 fAdverse event data are derived from patients followed for up to two years post-dose.
3%) subjects. The infusion was temporarily interrupted in 3 patients and resumed at a slower infusion rate upon treatment with antihistamines and/or corticosteroids. 1). 8%) patients. 4). Nine of the 13 patients with ALT elevations received a tapered course of corticosteroid.
4 days (range: 51 to 130). Nine of the 13 patients with ALT elevations also experienced AST elevations. All treatment-emergent adverse events of elevated ALTs were non-serious and resolved within 3 to 127 days. After month 6 post-dose, no persistence of treatment-related transaminitis was observed and no steroid treatment was required.
4). Immunogenicity In the clinical studies with etranacogene dezaparvovec, no Factor IX inhibitor development was observed. An expected sustained humoral immune response to the infused AAV5 capsid was observed in all patients treated with etranacogene dezaparvovec.
1) Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store

GBOfficial regulatory label· Warnings and precautions· revised April 24, 2026[1]

4. 6). Hemgenix can be administered only once. 1). Therefore, haemostatic support with exogenous human Factor IX may be needed during the first weeks after etranacogene dezaparvovec infusion to provide sufficient Factor IX coverage for the initial days post-treatment.
g. weekly for 3 months) is recommended post-dose to follow the patient`s response to etranacogene dezaparvovec. When using an in vitro activated partial thromboplastin time (aPTT)-based one-stage clotting assay for determining Factor IX activity in patients’ blood samples, plasma Factor IX activity results can be affected by both the type of aPTT reagent and the reference standard used in the assay.
4). Therefore, the same assay and reagents are recommended to be used to monitor Factor IX activity over time. In case increased plasma Factor IX activity levels are not achieved, decrease, or bleeding is not controlled or returns, post-dose testing for Factor IX inhibitors is recommended along with Factor IX activity testing.
Special populations Elderly population No dose adjustments are recommended in elderly patients. 1). Renal impairment No dose adjustments are recommended in patients with any level of renal impairment. 2). 2). The safety and efficacy of etranacogene dezaparvovec in patients with severe hepatic impairment have not been studied.
3). 2). Patient with HIV No dose adjustments are recommended in HIV-positive patients. Limited data are available in patients with controlled HIV infection. Paediatric population The safety and efficacy of etranacogene dezaparvovec in children aged 0 to 18 years have not been studied.
No data are available. 9%) solution for infusion. Etranacogene dezaparvovec must not be administered as an intravenous push or bolus. 6. Infusion rate The diluted product should be administered at a constant infusion rate of 500 mL/hour (8 mL/min).
• In the event of an infusion reaction during administration, the infusion rate should be slowed or stopped to ensure patient tolerability. 4). 3). 6. 1. 4). 4). 4 Special warnings and precautions for use Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.

This is not medical advice. Consult a qualified healthcare professional.

Side effects & warnings

EUOfficial regulatory label· Adverse reactions· revised March 4, 2026[2]

5% of patients), and influenza-like illness (very common; 14% of patients). 1). The Table 3 shows the overview of the ADRs from the clinical trials with etranacogene dezaparvovec. The ADRs are classified according the MedDRA System Organ Class and frequency.
The ADRs are listed based on the following convention for frequency categories: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000), and not known (cannot be estimated from the available data).
Within each frequency category, adverse reactions are presented in order of decreasing frequency. Table 3. Adverse drug reactions obtained from clinical studies with etranacogene dezaparvovec MedDRA System Organ Class (SOC) ADR (Preferred term) Frequency per patient Nervous system disorders Headache Very common Dizziness Common Gastrointestinal disorders Nausea Common General disorders and administration site conditions Investigations Influenza like illness Very common Fatigue, malaise Common Alanine aminotransferase increased, aspartate aminotransferase increased, C-reactive protein increased Very common Blood creatine phosphokinase increased, blood bilirubin increased Common Injury, poisoning and procedural complications Infusion related reaction (Hypersensitivity, infusion site reaction, dizziness, eye pruritus, flushing, abdominal pain upper, urticaria, chest discomfort, pyrexia) Very Common* *The frequency results from pooled infusion related reactions of similar medical concept.
5%) within 24 hours post-dose. Hepatic laboratory abnormalities Table 4 describes hepatic laboratory abnormalities following administration of Hemgenix. 4). Table 4. 1%) Abbreviations: ULN = Upper Limit of Normal; CTCAE = Common Terminology Criteria for Adverse Events aHighest post-dose CTCAE Grades of values are presented bNot all patients with laboratory abnormality >ULN reached CTCAE Grade 1 due to elevated baseline levels cCTCAE Grade 1 13 dCTCAE Grade 2 eCTCAE Grade 3 fAdverse event data are derived from patients followed for up to two years post-dose.
3%) subjects. The infusion was temporarily interrupted in 3 patients and resumed at a slower infusion rate upon treatment with antihistamines and/or corticosteroids. 1). 8%) patients. 4). Nine of the 13 patients with ALT elevations received a tapered course of corticosteroid.
4 days (range: 51 to 130). Nine of the 13 patients with ALT elevations also experienced AST elevations. All treatment-emergent adverse events of elevated ALTs were non-serious and resolved within 3 to 127 days. After month 6 post-dose, no persistence of treatment-related transaminitis was observed and no steroid treatment was required.
4). Immunogenicity In the clinical studies with etranacogene dezaparvovec, no Factor IX inhibitor development was observed. An expected sustained humoral immune response to the infused AAV5 capsid was observed in all patients treated with etranacogene dezaparvovec.
1). Reporting of suspected adverse reactions Reporting suspected adverse drug reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.

EUOfficial regulatory label· Warnings and precautions· revised March 4, 2026[2]

4. 6). Hemgenix can be administered only once. 1). Therefore, haemostatic support with exogenous human Factor IX may be needed during the first weeks after etranacogene dezaparvovec infusion to provide sufficient Factor IX coverage for the initial days post-treatment.
g. weekly for 3 months) is recommended post-dose to follow the patient`s response to etranacogene dezaparvovec. When using an in vitro activated partial thromboplastin time (aPTT)-based one-stage clotting assay for determining Factor IX activity in patients’ blood samples, plasma Factor IX activity results can be affected by both the type of aPTT reagent and the reference standard used in the assay.
4). Therefore, the same assay and reagents are recommended to be used to monitor Factor IX activity over time. In case increased plasma Factor IX activity levels are not achieved, decrease, or bleeding is not controlled or returns, post-dose testing for Factor IX inhibitors is recommended along with Factor IX activity testing.
Special populations Elderly population No dose adjustments are recommended in elderly patients. 1). Renal impairment No dose adjustments are recommended in patients with any level of renal impairment. 2). 2). The safety and efficacy of etranacogene dezaparvovec in patients with severe hepatic impairment have not been studied.
3). 2). Patient with HIV 4 No dose adjustments are recommended in HIV-positive patients. Limited data are available in patients with controlled HIV infection. Paediatric population The safety and efficacy of etranacogene dezaparvovec in children aged 0 to 18 years have not been studied.
No data are available. 9%) solution for infusion. Etranacogene dezaparvovec must not be administered as an intravenous push or bolus. 6. Infusion rate The diluted product should be administered at a constant infusion rate of 500 mL/hour (8 mL/min).
• In the event of an infusion reaction during administration, the infusion rate should be slowed or stopped to ensure patient tolerability. 4). 3). 6. 1. 4). 4). 4 Special warnings and precautions for use Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.

This is not medical advice. Consult a qualified healthcare professional.

Overview

Regulatory status by market

GBUnited Kingdom· MHRA

EUEuropean Union· EMA

CACanada· Health Canada

Drugs in the same class

Sources & citations