Abeona planning potential US launch of pz-cel to treat RDEB
Developer readying for FDA decision on therapy's approval in April
Abeona Therapeutics is preparing for the potential launch of pz-cel (prademagene zamikeracel) to treat recessive dystrophic epidermolysis bullosa (RDEB), after the U.S. Food and Drug Administration (FDA) initiated discussions on the medication label and post-marketing requirements, according to a company press release.
Last fall, the FDA indicated April 29 as the deadline by which it will decide whether or not to approve pz-cel for RDEB. That date was set after the agency accepted Abeona’s resubmitted biologics license application (BLA) for the cell therapy.
If it’s now approved, the company anticipates the first patient will receive pz-cel in the third quarter of this year, or by the end of September. Additionally, the company may be eligible for a priority review voucher upon approval, which can be used to speed the review of a future application for another therapy or be sold to a third party.
In “recent progress” noted by the company, Abeona has also obtained two additional patents from the U.S. Patent and Trademark Office. One covers the use of pz-cel to treat RDEB until June 2037, with the other covering the packaging and transport system for the therapy until July 2040.
“The RDEB community continues to highlight the unmet medical need for new therapies that could provide long-lasting healing and pain reduction, even in tough large, chronic RDEB wounds,” said Vish Seshadri, PhD, Abeona’s CEO. “With a [decision] date of April 29, 2025, the BLA review by the FDA is on track as discussions are underway regarding post-marketing requirements and commitments as well as the draft label.”
Pz-cel shown to successfully treat RDEB in clinical trials
RDEB is caused by mutations in the COL7A1 gene that lead to a deficiency or dysfunction in a type of collagen, a protein that helps maintain the structural integrity of the skin. This leads to unusually fragile skin that easily blisters and tears.
Pz-cel, formerly known as EB-101, is a cell therapy that Abeona is developing to treat RDEB. It involves collecting skin cells from a patient and genetically modifying them to insert a healthy version of the COL7A1 gene. The genetically modified cells are then grown on a thin sheet that can be grafted onto a patient’s wounds, helping restore collagen production and facilitate wound healing.
Abeona’s BLA application seeking the therapy’s approval was based on data from two clinical trials — a Phase 1/2a study (NCT01263379) and a Phase 3 trial called VIITAL (NCT04227106) — showing that applying pz-cel to large, chronic wounds improved healing and reduced pain in adults and children with RDEB.
The therapy was also found to be well-tolerated, with no serious treatment-related adverse events reported.
In anticipation of the therapy’s approval, the company is making efforts to select five well-recognized epidermolysis bullosa treatment centers across the U.S. to serve as pz-cell qualified treatment centers, as well as educating stakeholders and engaging payers to ensure patients’ access to treatment.
Additionally, Abeona has hired and trained personnel to support pz-cel’s manufacturing, supply chain, quality control, and commercialization. It has also entered into a lease agreement for an additional facility, in Ohio, to expand the manufacturing capacity of the therapy.
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