Abeona provides new data to FDA on EB-101 ahead of meeting
Application for approval of RDEB therapy expected later this year
Abeona Therapeutics has submitted a data briefing package on its cell therapy candidate EB-101 to the U.S. Food and Drug Administration (FDA) ahead of a meeting with the agency planned for this month.
The meeting’s purpose is to discuss Abeona’s planned application seeking approval of EB-101 to treat recessive dystrophic epidermolysis bullosa (RDEB), typically one of the most severe forms of the skin disorder. The company is planning to submit a biologics license application or BLA for EB-101 later this year.
“We look forward to discussing various clinical and chemistry, manufacturing, and controls aspects of our data package at the pre-BLA meeting, and anticipate submitting the EB-101 BLA in the third quarter of 2023,” Vish Seshadri, CEO of Abeona, said in a company press release.
The updated data was submitted at the request of the FDA, Seshadri said.
FDA had requested updated data on therapy’s production process
RDEB is caused by mutations in the gene COL7A1, which carries instructions for making a part of the protein type VII collagen. This protein normally helps to provide structural support to skin tissue; in RDEB, the protein is lacking, leading to symptoms such as fragile skin and chronic wounds that don’t heal well.
EB-101 is designed to restore production of this structural protein. The therapy works by first collecting skin cells from a patient, then engineering them in a lab so they carry a healthy copy of the COL7A1 gene. The modified cells are then affixed to a cellular sheet that is surgically transplanted onto a wound, allowing the gene-corrected cells to produce functional type VII collagen and promote wound healing.
To deliver a healthy copy of the COL7A1 gene to skin cells, EB-101 uses a retroviral vector — a virus that has been engineered to deliver the therapeutic gene rather than cause an infection. Clinical trials testing EB-101 used retroviral vectors made at two facilities, either in-house by Abeona or at Indiana University, in the U.S.
The newly submitted data were requested by the FDA to confirm that the vector works the same regardless of where it is made.
“In response to the FDA’s request, we have generated additional data to establish comparability between retroviral vectors from two different sources that were used in the EB-101 clinical studies, and have included this data in the briefing package for our pre-BLA meeting with the FDA,” Seshadri said.
Abeona’s BLA will be based mainly on data from the Phase 3 trial VIITAL (NCT04227106). The study enrolled 11 people with RDEB who had large wounds that had not healed for at least six months. In the trial, each patient received EB-101 to treat some wounds, while other wounds were left untreated for comparison.
The results showed that, after six months, more than 80% of wounds given EB-101 had healed by at least half. By comparison, less than 20% of untreated wounds showed this extent of healing. Patients reported that the therapy also reduced pain and itching at wounds.