Abeona seeks FDA approval for EB-101 cell therapy for RDEB
If granted priority review, decision could be made by mid-2024
The biologics license application (BLA) submitted by the company was filed along with a request for priority review, which, if granted, would shorten the time to a decision from the standard 10 months to six months.
The FDA usually decides whether to accept or reject the submission within 60 days, or two months. If it’s accepted and priority review is granted, a potential approval could take place by the second quarter of 2024.
“The BLA submission for EB-101 is a historic milestone for Abeona and a critical step toward making our investigational EB-101 product an option for RDEB patients,” Vish Seshadri, PhD, Abeona’s CEO, said in a company press release.
One-time therapy could ‘provide years of wound healing,’ developer says
The submission by Abeona comes following an August meeting with the FDA in which the regulatory agency agreed that existing clinical data — provided in a briefing package prior to the meeting — were adequate to support the BLA.
“We appreciate the FDA’s level of engagement and constructive guidance in the months leading up to the pre-BLA meeting,” Seshadri said.
RDEB, a severe form of epidermolysis bullosa, is caused by mutations in COL7A1, a gene coding part of type VII collagen. This protein is essential for binding the layers of skin together. When type VII collagen is missing, the skin becomes fragile and can tear and blister easily.
EB-101 works by delivering a working (healthy) version of the COL7A1 gene to a patient’s keratinocytes, the main cell type in the skin’s outer layer. The working gene is packaged aboard a delivery vehicle, a viral vector, and unloaded into the cells in a lab.
The modified cells are then grown in a sheet that can be surgically transplanted back to the patient onto a wound. The skin graft is expected to produce type VII collagen, thus helping the wound to heal.
Seshadri called EB-101 “the first individualized cell therapy with potential to provide years of wound healing and pain reduction following a one-time application.”
The company’s BLA was largely backed by data from VIITAL (NCT04227106), a pivotal Phase 3 clinical study that included 11 people with RDEB. The participants, ages 6 to 40, had wounds that had remained open for at least six months.
[EB-101 is] the first individualized cell therapy with potential to provide years of wound healing and pain reduction following a one-time application.
The study included a total of 43 pairs of wounds, all of them 20 square centimeters (about 3 square inches) in area or larger. For each pair, one wound was randomly assigned to be treated with up to six EB-101 sheets, depending on how large it was, while the other was left untreated.
A main goal of the study was to check how many wounds achieved 50% or more healing in 24 weeks, or about six months.
The results showed that a significantly higher proportion of wounds treated with EB-101 reached the 50% healing mark compared with untreated wounds (81.4% vs. 16.3%).
EB-101 also eased patient-reported pain in the wound sites, the other main goal of the study. The gene-corrected cell therapy was tolerated well, with no serious treatment-related side effects noted.
The FDA has granted EB-101 regenerative medicine advanced therapy, breakthrough therapy, orphan drug, and rare pediatric disease designations. If EB-101 is approved, Abeona may qualify for a priority review voucher, which can be used to speed the review of a future application, or be sold to a third party.