Brand Name
Hemgenix
Generic Name
Etranacogene Dezaparvovec
View Brand Information FDA approval date: November 22, 2022
Form: Kit
What is Hemgenix (Etranacogene Dezaparvovec)?
HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for treatment of adults with Hemophilia B who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes. HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B who: Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage, or Have repeated, serious spontaneous bleeding episodes.
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Brand Information
HEMGENIX (etranacogene dezaparvovec)
1INDICATIONS AND USAGE
HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for 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.
2DOSAGE AND ADMINISTRATION
For single-use intravenous infusion only.
For patient selection:
- Perform Factor IX inhibitor titer testing.
- Perform liver health assessments, including:
2.1Dose
The recommended dose of HEMGENIX is 2 × 10
The multiplication factor 2 represents the per kilogram dose (2 × 10
Number of HEMGENIX vials needed = HEMGENIX dose (in mL) divided by 10 (round up to next whole number of vials).
The division factor 10 represents the extractable volume of HEMGENIX from each vial (10 mL).
The total volume of the patient's HEMGENIX dose to be diluted may be less than the total volume of vials needed.
Example calculation for 72 kg patient
HEMGENIX can be administered only once.
2.2Preparation
The vials are for single-dose only.
3DOSAGE FORMS AND STRENGTHS
HEMGENIX is a clear and colorless suspension for intravenous infusion.
HEMGENIX is provided in a kit containing 10 to 48 vials. Each kit constitutes a dosage unit based on the patient's body weight.
HEMGENIX has a nominal concentration of 1 × 10
4CONTRAINDICATIONS
None.
5ADVERSE REACTIONS
The most common adverse reactions (incidence ≥5%) reported in clinical studies were ALT elevations, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, malaise, and AST elevations.
The following adverse reactions are discussed in greater detail in other sections of the label:
- Infusion related reactions
- Hepatotoxicity
- Immune-mediated neutralization of the AAV5 vector capsid
5.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice.
The safety of HEMGENIX was evaluated in two clinical studies (the first study enrolled 3 subjects and the second study 54 subjects). Both studies enrolled adult male subjects with moderately severe or severe Hemophilia B (N = 57), who received a single intravenous dose of 2 × 10
No serious adverse reactions were reported
Infusion-related reactions were observed in 19 subjects. Infusions were temporarily interrupted in 3 subjects and resumed at a slower infusion rate after treatment with antihistamines and/or corticosteroids. In one subject, infusion was stopped and not resumed (see
There were 24 subjects who had elevated ALT values from Day 8 to 731 post-administration.
Five subjects had ALT elevations >2-3× ULN (range = 89 IU/L – 130 IU/L), one subject had an ALT elevation > 3-5× ULN (193 IU/L) and one subject had an ALT elevation > 5× ULN (275 IU/L). The subject who had the ALT elevation >5× ULN occurred 3 weeks after HEMGENIX administration.
Five subjects had AST elevations > 2-3× ULN (range = 71 IU/L – 118 IU/L), three subjects had AST elevations > 3-5× ULN (range = 127 IU/L – 163 IU/L) and one subject had an AST elevation > 5× ULN (327 IU/L). The subject who had the AST elevation > 5× ULN occurred 11 months post-HEMGENIX administration.
Seventeen subjects had elevations in ALT levels within the first 4 months after HEMGENIX infusion (range = 41 IU/L – 275 IU/L), eleven of these subjects' ALT levels resolved within 4 months post-infusion (range = 41 IU/L – 275 IU/L) and five of these subjects' ALT levels never normalized as of last follow-up (range of values at 2-year follow-up = 48 IU/L – 110 IU/L). Seven additional subjects had ALT elevations with onset between Months 6 to 24 (range = 42 IU/L-193 IU/L), five of these subjects had additional risk factors for having elevated transaminase levels including Hepatitis C and Human Immunodeficiency Virus (HIV). ALT levels never normalized as of last follow-up (range of values at 2-year follow-up = (59 IU/L- 193 IU/L) in three of the subjects with ALT elevations with onset between Months 6 to 24.
Nineteen subjects had elevations in AST levels within 3 months after HEMGENIX infusion (range = 32 IU/L- 163 IU/L). Nine of these subjects' AST elevations resolved within 4 months post-infusion (range = 35 IU/L – 163 IU/L), three resolved within 7 to 13 months post-infusion (range = 35 IU/L – 62 IU/L), and seven of these subjects' AST levels never normalized as of last follow-up (range of values at 2-year follow-up = 36 IU/L – 327 IU/L). The remaining 5 subjects with AST elevation had onset of between 6 months and 2 years post-infusion (range = 36 IU/L – 127 IU/L), and AST levels had not normalized as of the last follow-up for one subject (AST at 2-year follow-up = 127 IU/L) who had additional risk factors for having elevated transaminase levels.
Nine subjects with ALT elevations received a tapered course of corticosteroids. The mean duration of corticosteroid treatment for the elevated ALT was 81.4 days. Nineteen of the 24 subjects with ALT elevations also had a related AST elevation. Twenty-one subjects had elevated transaminase levels and were not treated with corticosteroids.
6DESCRIPTION
HEMGENIX (etranacogene dezaparvovec-drlb) is an adeno-associated viral vector-based gene therapy for intravenous infusion after dilution. HEMGENIX is a non-replicating recombinant AAV5 containing a codon-optimized DNA sequence of the gain-of-function Padua variant of human Factor IX (variant R338L), under control of a liver-specific promotor 1 (LP1).
HEMGENIX has a nominal concentration of 1 × 10
HEMGENIX is sterile, clear and colorless suspension, and contains no preservative. After dilution, HEMGENIX should be clear and colorless suspension.
7NONCLINICAL TOXICOLOGY
Nonclinical studies were initiated with a predecessor of HEMGENIX product, rAAV5 expressing the wild type human coagulation factor IX (rAAV5-hFIX). HEMGENIX was developed by introducing a 2-nucleotide change in the transgene for hFIX, generating the naturally occurring Padua variant of Factor IX (rAAV5-hFIX-Padua).
7.1Carcinogenesis, Mutagenesis, Impairment of Fertility
No traditional nonclinical carcinogenicity or mutagenicity studies were conducted with HEMGENIX; such studies were not indicated. No adverse effects were observed in mating rates and fertility indices in healthy naïve female mice following mating with males that were administered the predecessor of HEMGENIX
7.2Animal Toxicology and/or Pharmacology
A pharmacology study was conducted in a murine model of Hemophilia B (
Intravenous administration of HEMGENIX resulted in a no-observed-adverse-effect-level of 5 × 10
One out of 10 healthy mice administered 5 × 10
8CLINICAL STUDIES
The efficacy of HEMGENIX was evaluated in a prospective, open-label, single-dose, single-arm, multi-national study (N = 54). The study enrolled adult male subjects aged 19 to 75 years, with severe or moderately severe Hemophilia B, who received a single intravenous dose of 2 × 10
The 54 subjects prospectively completed a lead-in period of at least six months with the intent to receive standard of care routine Factor IX prophylaxis. These 54 subjects then received the indicated single intravenous dose of HEMGENIX. Subjects were then followed up monthly until Month 12, and then at 6-month intervals until Year 5. For the efficacy evaluation, data up to 18 months post-treatment were used. Of the 54 subjects, 53 subjects completed at least 18 months of follow-up in the ongoing study. One subject with numerous cardiovascular and urologic risk factors, aged 75 years at screening, died of urosepsis and cardiogenic shock at Month 15 post-dose (at age 77 years) unrelated to treatment. Another subject received around 10% of the intended dose of HEMGENIX due to an infusion-related hypersensitivity reaction.
The main efficacy outcome was a non-inferiority test of annualized bleeding rate (ABR) during Months 7 to 18 after HEMGENIX treatment compared with ABR during the lead-in period. All bleeding episodes, regardless of investigator assessment, were counted. Subjects were allowed to continue prophylaxis during Months 0 to 6. The estimated mean ABR during Months 7 to 18 after HEMGENIX treatment was 1.9 bleeds/year with a 95% confidence interval (CI) of (1.0, 3.4), compared with an estimated mean ABR of 4.1 [95% CI: 3.2, 5.4] during the lead-in period. The ABR ratio (Months 7 to 18 post-treatment / lead-in) was 0.46 [95% CI: 0.26, 0.81], demonstrating non-inferiority of ABR during Months 7 to 18 compared to the lead-in period.
Two subjects were not able to stop routine prophylaxis after HEMGENIX treatment. During Months 7 to 18, an additional subject received prophylaxis from Days 396-534 [approximately 20 weeks].
After a single-dose of HEMGENIX, increases in Factor IX activity were observed [
9PATIENT COUNSELING INFORMATION
Inform patients that:
- Pre-infusion blood tests will be necessary to look for Factor IX inhibitors. If these exist, the patient may not be a good candidate for HEMGENIX
- Prior to HEMGENIX treatment, a liver ultrasound and elastography will be performed. Patients found to have pre-existing risk factors for hepatocellular carcinoma will be monitored annually in the 5 years following infusion
- Infusion reactions can occur. Patients will be monitored during and for at least 3 hours following administration. If a reaction occurs, the infusion rate may be slowed or interrupted, then started at a slower rate
- HEMGENIX can elevate certain liver enzymes. Weekly blood tests will be required to monitor for this for 3 months after treatment. Corticosteroid treatment may be necessary if this occurs
- If post-infusion bleeding is not controlled or if bleeding returns, then blood tests will be performed for Factor IX activity and neutralizing Factor IX inhibitors
- Vector distribution in blood (within the body), and vector shedding in semen and other excreta and secreta can occur post-infusion. It is not known how long this will continue. Patients should not donate blood, organs, tissues, or cells for transplantation
10PRINCIPAL DISPLAY PANEL - 10 ml Vial Label
NDC 0053-0099-01
etranacogene dezaparvovec-drlb
1x10
Suspension for intravenous infusion
Rx only
CSL Behring
11PRINCIPAL DISPLAY PANEL - 10 ml Vial Carton
NDC 0053-0099-01
etranacogene dezaparvovec-drlb
1 x 10
Single-dose vial
Upon receipt store refrigerated at
Do Not Freeze
Protect from Light
Do Not Shake
Dilute before use
See enclosed prescribing information
Rx only
12PRINCIPAL DISPLAY PANEL - Kit Carton
etranacogene dezaparvovec-drlb
1 x 10
Single-dose vial
Upon receipt store refrigerated at 2°C to 8°C (36°F to 46°F)
Do Not Freeze
Protect from Light
Do Not Shake
Dilute before use
Manufactured by:
Manufactured for:
Distributed by:
CSL Behring
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