Prademagene zamikeracel FDA Approval Status
Last updated by Judith Stewart, BPharm on April 23, 2024.
FDA Approved: No
Generic name: prademagene zamikeracel
Previous Name: EB-101
Company: Abeona Therapeutics Inc.
Treatment for: Epidermolysis Bullosa
Prademagene zamikeracel (pz-cel) is an autologous, engineered cell therapy in development for the treatment for patients with recessive dystrophic epidermolysis bullosa.
- Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications. RDEB is caused by a defect in the COL7A1 gene, which results in the inability to produce functioning type VII collagen that helps anchor the dermal and epidermal layers of the skin.
- Pz-cel treatment involves using gene transfer to deliver COL7A1 genes into the patient’s own skin cells (keratinocytes) then transplanting them back to the patient to enable normal Type VII collagen expression and to facilitate wound healing.
- Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA.
- On April 22, 2024, Abeona Therapeutics Inc. announced that the U.S. Food and Drug Administration had issued a Complete Response Letter in response to the Company’s Biologics License Application for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa noting that certain additional information needed to satisfy Chemistry Manufacturing and Controls requirements must be satisfactorily resolved before the application can be approved.
Development timeline for prademagene zamikeracel
Further information
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
