Hemophilia B Treatments
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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 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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Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
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An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
Summary: This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
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Brand Information
HEMGENIX (etranacogene dezaparvovec)
1INDICATIONS AND USAGE
HEMGENIX is 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 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
3CONTRAINDICATIONS
None.
4DESCRIPTION
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.
5NONCLINICAL 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).
5.1Carcinogenesis, 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
5.2Animal 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
6CLINICAL STUDIES
The efficacy of HEMGENIX was evaluated in a prospective, open-label, single-dose, single-arm, multi-national study (NCT03569891; N = 54). The study enrolled adult male patients aged 19 to 75 years, with severe or moderately severe Hemophilia B, who received a single intravenous dose of 2 × 10
The 54 patients prospectively completed a lead-in period of at least six months with the intent to receive standard of care regular Factor IX prophylaxis. These 54 patients then received the indicated single intravenous dose of HEMGENIX. Patients were then followed up weekly until Month 3, monthly until Month 12, and then at 6-month intervals until Year 5 (Month 60). Of the 54 patients, 53 patients completed at least 24 months of follow-up and 50 patients completed 60 months of efficacy follow-up.
The primary efficacy evaluation was non-inferiority in annualized bleeding rate (ABR) during Months 7 to 18 after HEMGENIX treatment compared with ABR during the lead-in period as described in Table 7. All bleeding episodes, regardless of investigator assessment, were counted. Patients were allowed to continue prophylaxis during Months 0 to 6. 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.
Four patients had regular or intermittent prophylaxis following HEMGENIX administration and are described below.
An unvalidated clinical trial assay was utilized to assess preexisting neutralizing anti-AAV5 antibodies. There were 21 patients with pre-treatment positive AAV5 antibody titers that ranged between 1:8.5-3212. Patients with detectable preexisting neutralizing anti-AAV5 antibodies up to titers of 1:678 showed mean Factor IX activity that was numerically lower compared to patients without detectable preexisting neutralizing anti-AAV5 antibodies. One patient with anti-AAV5 antibodies (1:3212) did not stop regular prophylaxis after HEMGENIX treatment due to lack of response.
One patient who received a partial dose administration continued regular prophylaxis. One patient resumed FIX prophylaxis beginning day 874. An additional patient received prophylaxis intermittently from Days 396-1825.
The adjusted ABR for months 7 to 60 after HEMGENIX treatment was 1.9 (95% CI: 1.0, 3.7).
Four patients did not complete the 5-year follow-up period: 2 patients due to treatment-unrelated death, 1 subject due to receiving a treatment-unrelated liver transplantation and 1 subject due to withdrawal of consent. Another subject received around 10% of the intended dose of HEMGENIX due to an infusion-related hypersensitivity reaction but continued through to the study end and was included in the efficacy analysis.
7PATIENT COUNSELING INFORMATION
Inform patients that:
- Pre-infusion blood tests will be necessary to look for Factor IX inhibitors. If these exist, the patient is not a 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-related and allergic 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
8PRINCIPAL DISPLAY PANEL - 10 ml Vial Label
NDC 0053-0099-01
etranacogene dezaparvovec-drlb
1x10
Suspension for intravenous infusion
Rx only
CSL Behring
9PRINCIPAL 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
10PRINCIPAL 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
11PRINCIPAL DISPLAY PANEL - NDC: 0053-0100-10_ 46-50 KG KIT VARIABLE LABEL
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