FDA decision on Abeona’s pz-cel for RDEB now expected by April 29
Formerly EB-101, pz-cel involves genetically engineering patient's collected cells
The U.S. Food and Drug Administration (FDA) is expected to decide whether or not to approve the cell therapy pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB) by April 29, 2025.
Abeona Therapeutics, the developer of pz-cel, announced that the FDA has agreed to review a resubmitted biologics license application (BLA) for the therapy.
“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.,” Vish Seshadri, PhD, CEO of Abeona, said in a company press release.
FDA had earlier concerns about therapy’s manufacturing process
This marks the second time Abeona has sought FDA approval of pz-cel. The agency earlier this year said it couldn’t grant approval due to concerns about the cell therapy’s manufacturing process. The previous application had already been accepted by the FDA under priority review, which shortens the review process down to six months (FDA reviews usually take about 10 months).
“We look forward to continuing to work with the FDA to finalize the review of the pz-cel application,” Seshadri said.
RDEB is caused by mutations in the gene COL7A1, which provides instructions for making a type of collagen, or a protein that’s vital to maintaining the structural integrity of the skin. In RDEB, deficiency in collagen production causes the skin to be unusually fragile, leading to symptoms such as wounds and blistering.
Pz-cel, formerly known as EB-101, involves collecting skin cells from a patient and genetically engineering them to contain a healthy version of the COL7A1 gene. The modified cells are then grown on a thin sheet that can be grafted onto a patient’s wounds to help promote normal collagen production and ultimately facilitate better wound healing.
Abeona’s application seeking approval of the cell therapy is based on data from two clinical trials, a Phase 1/2a study (NCT01263379) and a Phase 3 trial called VIITAL (NCT04227106). Across both these trials, results showed that applying pz-cel to large, long-lasting wounds led to improved healing and lessened pain in adults and children with RDEB.
The FDA had previously granted pz-cel several designations to incentivize and speed up its development, including orphan drug, rare pediatric disease, regenerative medicine advanced therapy, and breakthrough therapy designations.
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