The U.S. Food and Drug Administration (FDA) is slated to decide on the approval of Abeona Therapeutics' pz-cel (prademagene zamikeracel) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) by April 29, 2025. This decision follows the FDA's acceptance of Abeona's resubmitted biologics license application (BLA) for the cell therapy, offering renewed hope for a differentiated treatment option for individuals with RDEB.
Vish Seshadri, PhD, CEO of Abeona, stated, "The FDA acceptance of our BLA resubmission moves us one step closer to providing pz-cel as a differentiated treatment option to address the persistent unmet needs of people with RDEB in the U.S."
Addressing Previous Manufacturing Concerns
This marks Abeona's second attempt to secure FDA approval for pz-cel. Earlier this year, the agency declined approval due to concerns regarding the cell therapy's manufacturing process. The initial application had been granted priority review, expediting the review timeline to six months.
Seshadri added, "We look forward to continuing to work with the FDA to finalize the review of the pz-cel application."
Understanding Recessive Dystrophic Epidermolysis Bullosa (RDEB)
RDEB is a genetic disorder caused by mutations in the COL7A1 gene, which provides instructions for producing type VII collagen, a protein crucial for maintaining skin integrity. The deficiency in collagen production leads to fragile skin, resulting in wounds and blistering.
How Pz-cel Works
Pz-cel, previously known as EB-101, involves collecting skin cells from patients and genetically modifying them to carry a healthy copy of the COL7A1 gene. These modified cells are then grown into a sheet that can be grafted onto the patient's wounds. The aim is to promote normal collagen production, leading to improved wound healing.
Clinical Trial Data Supporting Pz-cel
Abeona's BLA submission is supported by data from a Phase 1/2a study (NCT01263379) and the Phase 3 VIITAL trial (NCT04227106). Results from these trials demonstrated that pz-cel application to large, chronic wounds resulted in improved healing and pain reduction in both adult and pediatric RDEB patients.
The FDA had previously granted pz-cel several designations, including orphan drug, rare pediatric disease, regenerative medicine advanced therapy, and breakthrough therapy designations, to accelerate its development and address the unmet needs in RDEB treatment.
