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Hemgenix is a brand name for Etranacogene Dezaparvovec. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: 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.
Verbatim from this product's EMA label. Tap a section to expand.
Treatment should be initiated under the supervision of a physician experienced in the treatment of Haemophilia and/or bleeding disorders. 8). 3 Hemgenix should only be administered to patients who have demonstrated absence of Factor IX inhibitors.
In case of a positive test result for human Factor IX inhibitors, a re-test within approximately 2 weeks should be performed. If both the initial test and re-test results are positive, the patient should not receive Hemgenix. In addition, before administration of Hemgenix, baseline testing of liver health and assessment of preexisting neutralising anti-AAV5 antibody titre should be performed; see section
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.
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.
1. 4). 4).
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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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.
Initiation of treatment with Hemgenix Patients with pre-existing antibodies to the AAV5 vector capsid Prior to the treatment with Hemgenix patients should be assessed for the titre of preexisting neutralising anti-AAV5 antibodies. 1).
There is limited data in patients with neutralising anti-AAV5 antibodies above 1:898 (equivalent to the 1:678 titre based on the clinical study assay). 1). In the clinical studies with etranacogene dezaparvovec, for the patient sub-group with detectable preexisting neutralising anti-AAV5 antibodies up to a titre of 1:678 (tested using the clinical study assay, equivalent to 1:898 titre based on the neutralising anti-AAV5 antibody assay with extended measuring range), mean Factor IX activity levels were within the […]