RDEB cell therapy pz-cel on track for May FDA decision

Abeona addressing FDA concerns raised during plant inspection

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Abeona Therapeutics said its gene-corrected cell therapy, called pz-cel (prademagene zamikeracel), for recessive dystrophic epidermolysis bullosa (RDEB), is on track for a May 25 decision from the U.S. Food and Drug Administration (FDA), and the company is addressing concerns raised during a plant inspection.

The FDA conducted a two-week inspection of the facility where the medication will be produced and noted concerns with “process controls,” Abeona said in a press release.

The treatment is being considered for FDA approval under the priority review process, which is designed to get important medications to market quickly.

Five FDA inspectors completed the pre-license inspection (PLI), a requisite for bringing a new biological medication to market in the U.S., at the Cleveland plant March 1, Abeona said. The FDA subsequently issued Abeona a Form 483, which is a formal documentation of concerns observed during the inspection. Abeona said it submitted a response to the FDA on March 15, outlining steps it plans to take to address those concerns.

“We appreciate the FDA’s collaborative conduct of the PLI, and look forward to working with them through the remainder of the BLA [biologics license application] review,” said Vish Seshadri, PhD, CEO of Abeona.

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Checking in with my son with EB after his 15th birthday

Trial shows therapy effective in RDEB

Abeona reiterated that it expects a final decision from the FDA on May 25, as previously announced, adding the agency does not plan to hold an advisory committee meeting. Such meetings are an optional review step in which outside experts offer their opinions on data for experimental therapies.

The company said it “continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel,” including “payer engagement,” and it has done market research that supports reimbursement coverage “at a price commensurate with the value of approved gene therapies.”

Abeona applied to the FDA seeking approval of pz-cel based primarily on data from a Phase 3 clinical trial called VIITAL (NCT04227106). The trial compared the cell therapy against no treatment in more than 80 chronic wounds in 11 people with RDEB, and results showed significantly better healing in wounds with the cell therapy.

The FDA has completed inspections of two of the trial sites where the VIITAL study took place — one at Stanford University School of Medicine in Palo Alto, California, and the other at the University of Massachusetts Medical School in Worcester, Massachusetts — and raised no formal concerns, Abeona said.

RDEB is caused by mutations in the COL7A1 gene, which is important for maintaining the structural integrity of the skin. In pz-cel (formerly called EB-101), a patient’s skin cells are collected and engineered in a lab so they’ll express a healthy version of this gene. The modified cells are then grown on a sheet that can be grafted onto wounds to promote healing.