Prademagene Zamikeracel
FDA Drug Information • Also known as: Zevaskyn
- Brand Names
- Zevaskyn
- Route
- TOPICAL
- Dosage Form
- CELLULAR SHEET
- Product Type
- HUMAN PRESCRIPTION DRUG
Description
11 DESCRIPTION ZEVASKYN is composed of autologous cells isolated from skin punch biopsies of patients with mutations in the collagen type VII alpha 1 chain ( C OL 7A1 ) gene and which have been transduced ex vivo with a replication-incompetent retroviral vector (RVV) containing the full-length COL7A1 gene. The resulting gene-modified cell sheets express functional collagen VII (C7) protein. ZEVASKYN is manufactured using human- and animal-derived reagents. ZEVASKYN is provided as cellular sheets of 41.25 cm 2 (5.5 cm x 7.5 cm) secured to sterile petrolatum gauze using sterile ligation titanium clips for surgical application. The cellular sheets consist of autologous, viable, gene-modified cells. Up to twelve (12) sheets may be manufactured from the patient biopsies and supplied for potential use. Each ZEVASKYN sheet is packaged in a clamshell and sealed transport pouch containing sterile transport media. The transport media includes reagents derived from human and animal materials, including bovine pituitary extract, bovine transferrin, and human transferrin. The sheet also has a nylon suture, which functions as a visual indicator of the sheet’s topography. Neither ZEVASKYN sheets nor its excipients contain preservatives.
What Is Prademagene Zamikeracel Used For?
1 INDICATIONS AND USAGE ZEVASKYN is indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN is an autologous cell sheet-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). ( 1 )
Dosage and Administration
2 DOSAGE AND ADMINISTRATION For autologous topical application on wounds only The recommended dose of ZEVASKYN is based on the surface area of the wound(s). One sheet of ZEVASKYN covers an area of 41.25 cm 2 . ( 2.1 ) Up to twelve ZEVASKYN sheets may be manufactured from the patient biopsies and supplied for potential use. ( 2.1 ) Verify the patient’s identity prior to ZEVASKYN application. ( 2.2 ) See full prescribing information for ZEVASKYN preparation, and administration instructions. ( 2.2 , 2.3 ) Figure 1 2.1 Recommended Dose For autologous topical application on wounds only. The recommended dose of ZEVASKYN is based on the surface area of the wound(s). One sheet of ZEVASKYN covers an area of 41.25 cm 2 . Up to twelve ZEVASKYN sheets may be manufactured from the patient biopsies and supplied for potential use. 2.2 Receipt and Preparation Receipt of ZEVASKYN ZEVASKYN is shipped directly to the qualified treatment center sealed in transport packaging. If the patient is expected to be ready for same-day application, transport the packaging to the operating room. Hold at room temperature (15-25°C) until preparation to maintain cell viability. If the patient is not expected to be ready for same-day application, store the packaging containing ZEVASKYN in a secure onsite location at room temperature (15-25°C) until preparation to maintain cell viability. Preparation ZEVASKYN is to be prepared by the manufacturer in an appropriate healthcare setting for surgical application by a qualified healthcare provider. Manufacturer will conduct Quality Control (QC) testing to release the product immediately prior to surgery in the operating room. Verify patient’s identity during preparation. Hold ZEVASKYN at room temperature (15-25°C) until application. Apply all selected sheets in a single surgical session. 2.3 Administration Below is the list of standard surgical supplies needed for ZEVASKYN administration and not provided by the manufacturer: scalpel scissors forceps resorbable sutures non-adhesive dressings topical antibiotic ointment 1. Verify patient’s identity prior to ZEVASKYN application. Do not apply ZEVASKYN if the information on the patient-specific label(s) does not match the intended patient. 2. Prepare wound by debridement under general or other appropriate anesthesia to accommodate ZEVASKYN sheet(s). 3. Before surgical application, pick up ZEVASKYN sheet with forceps by grasping the titanium ligating clips (Figure 1). Handle ZEVASKYN sheets only with forceps and only by the titanium ligating clips. Figure 1. ZEVASKYN sheets removed with forceps for application 4. Do not trim ZEVASKYN sheets. 5. Apply unaltered ZEVASKYN sheets onto the wound bed only (nylon suture on the ZEVASKYN sheet facing away from wound) by affixing via resorbable sutures. Do not suture ZEVASKYN onto healthy intact skin. Do not overlap ZEVASKYN sheets on wounds. 6. Apply all selected sheets in a single surgical session. 7. Cover area of application of ZEVASKYN sheets...
Side Effects (Adverse Reactions)
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥5%) were procedural pain and pruritus. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Abeona Therapeutics Inc at 1-844-888-2236 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a product cannot be directly compared to the rates in the clinical trials of another product and may not reflect the rates observed in practice. The safety data described in this section reflects exposure of 11 patients to ZEVASKYN in the VIITAL study [see Clinical Studies ( 14 ) ] . The median number of sheets patients received was 6 (range 3-6), and the total exposure time was 6 months following ZEVASKYN application. The most common adverse reactions occurring in ≥5% of patients were procedural pain (n=3; 27%) and pruritus (n=1; 9%).
Contraindications
4 CONTRAINDICATIONS None. None.
Pregnancy and Breastfeeding
8.1 Pregnancy Risk Summary There are no available data with ZEVASKYN use in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with ZEVASKYN. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Clinical Considerations Women of childbearing potential should be advised to use an effective method of contraception to prevent pregnancy at the time of treatment with ZEVASKYN.
How Supplied
1 6 HOW SUPPLIED/STORAGE AND HANDLING 16.1 How Supplied ZEVASKYN sheets of 41.25 cm² (5.5 cm × 7.5 cm) affixed on a rectangular gauze and placed in a clear, thermoformed protective case (“clamshell”) containing sterile transport media sealed in packaging consisting of 4 levels of protection. Up to four ZEVASKYN sheets are provided in a single transport container, with up to three containers per manufactured lot, for a total of up to twelve sheets. All available sheets per manufactured lot are supplied under NDC 84103-007-01. Confirm patient identity on the drug product container upon receipt. Each sheet is supplied ready for use and is intended for application on the patient from whom the biopsy was derived for manufacturing the ZEVASKYN sheet. Due to ZEVASKYN’s autologous nature, in case of a manufacturing failure, a second manufacturing of ZEVASKYN may be attempted and would require a repeat biopsy. 16.2 Storage and Handling Store and transport ZEVASKYN at room temperature (15-25°C). ZEVASKYN is stable for 84 hours at room temperature and must be used within 84 hours. Dispose of any compromised or mishandled product. Dispose of unused ZEVASKYN as surgical biohazardous waste in accordance with local requirements. Dispose of materials that have come into contact with ZEVASKYN as surgical biohazardous waste in accordance with local requirements. 16.1 How Supplied ZEVASKYN sheets of 41.25 cm² (5.5 cm × 7.5 cm) affixed on a rectangular gauze and placed in a clear, thermoformed protective case (“clamshell”) containing sterile transport media sealed in packaging consisting of 4 levels of protection. Up to four ZEVASKYN sheets are provided in a single transport container, with up to three containers per manufactured lot, for a total of up to twelve sheets. All available sheets per manufactured lot are supplied under NDC 84103-007-01. Confirm patient identity on the drug product container upon receipt. Each sheet is supplied ready for use and is intended for application...
About This Information
This drug information is sourced from FDA-approved labeling via the openFDA database. It is intended for educational and reference purposes only. This is not medical advice. Always consult your healthcare provider before making decisions about medication. Drug information may be updated by the FDA; check with your pharmacist for the most current information.
What are side effects?
Side effects are unwanted reactions that can occur when taking a medication. They range from mild (headache, nausea) to severe (allergic reactions, organ damage). Not everyone experiences side effects, and severity varies. Report any concerning side effects to your doctor.
What are drug interactions?
Drug interactions occur when a medication is affected by another drug, food, or supplement. Interactions can make medications less effective or cause dangerous side effects. Always tell your doctor about all medications and supplements you take.