FDA review of RDEB cell therapy pz-cel remains on schedule

Agency reaffirmed in January it had no plans for advisory committee meeting

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA)’s review of pz-cel (prademagene zamikeracel), a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), is progressing on schedule.

The therapy’s developer Abeona Therapeutics announced it had conducted a biologics license application (BLA) mid-cycle meeting with the FDA and the agency has also completed a bio-research monitoring (BIMO) inspection. Everything remains on track for an FDA decision on May 25. A BIMO inspection is an in-depth audit to double-check the validity and integrity of the data Abeona is using in support its application.

“The Abeona team is committed to working with the FDA in its review of the pz-cel BLA, with the goal of bringing this therapy to patients as soon as possible,” Vish Seshadri, Abeona’s CEO, said in a company press release.

At the Jan. 25 mid-cycle meeting, the FDA reaffirmed it has no plans to convene an advisory committee meeting for pz-cel, which is an optional step where the FDA asks a panel of outside experts to interpret available data, the company said. Pz-cel is also not expected to have Risk Evaluation and Mitigation Strategies (REMS), monitoring protocols that are put in place for some treatments that carry a substantial risk of serious side effects.

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How does pz-cel work in RDEB?

The FDA hasn’t yet given Abeona its formal report on the BIMO inspection, but no notable concerns were raised, the company said.

RDEB is caused by mutations in the COL7A1 gene, which provides instructions for making part of the type VII collagen protein that’s integral to maintaining the structure of skin. Formerly known as EB-101, pz-cel works by collecting skin cells from a patient and engineering them to carry a healthy copy of COL7A1. The engineered cells are then grown on a cellular sheet that can be grafted onto patients’ wounds.

Abeona’s application to the FDA was based mainly on data from the Phase 3 VIITAL clinical trial (NCT04227106). The study enrolled 11 people with RDEB with more than 80 chronic wounds among them. Some wounds were treated with pz-cel and others were left untreated. Significantly more wounds treated with pz-cel healed by at least 50% (81.4% vs. 16.3%), results showed. Benefits were also noted in the percentage of wounds healed by 75% or completely, and in significant reductions in patient-reported pain during wound dressing changes.