Abeona Completes Resubmission of Pz-cel Cell-Based Gene Therapy BLA for RDEB

Credit: US Food and Drug Administration

Abeona Therapeutics has finished resubmitting a biologics license application (BLA) forprademagene zamikeracel (pz-cel), an investigational cell-based gene therapy, for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB).¹

The FDA previously issued a complete response letter in April for Abeona’s previous BLA submission, over 1 year ago in September 2023, at which point the FDA requested more chemistry, manufacturing, and controls (CMC) information, specifically with regard to validation requirements for specific manufacturing and release testing methods.² There were no issues identified with clinical efficacy or safety data and no new trials or clinical data were requested. During a Type A meeting in August 2024, Abeona aligned with the FDA on the content of the resubmission and necessary CMC information.¹

“We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance,” Vish Seshadri, Chief Executive Officer of Abeona, said in a statement.¹ “We have incorporated the Agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the Complete Response Letter, including observations from the completed Pre-License Inspection of our manufacturing facility. Upon acceptance of the BLA, we look forward to assisting the FDA in the completion of its review.”

The BLA resubmission is supported by phase 3 data from the pivotal VIITAL study (NCT04227106) and data from a Phase 1/2a study (NCT01263379). VIITAL reported results in late 2022 that demonstrated achievement of the trial’s primary endpoints of achieving over 50% wound healing and achieving a greater magnitude of pain reduction benefit at 6 months after treatment compared to baseline and control wounds. The therapy has been well-tolerated with no serious treatment-related adverse events observed.³

If approved, pz-cel would become the second gene therapy available for the treatment of RDEB, following beremagene geperpavec’s (B-VEC) landmark approval in 2023.⁴ B-VEC is marketed under the name Vyjuvek and was developed by Krystal Biotech. Pz-cell is administered via a single-surgical application with autologous cells and B-VEC has a topical HSV-based gene delivery requiring weekly administration.

"I think many more local primary care pediatricians and dermatologists will start to treat epidermolysis bullosa patients now that they have an easy corrective therapy they can prescribe. This will be a benefit for epidermolysis bullosa patients as well," M. Peter Marinkovich, MD, primary investigator and associate professor of dermatology, faculty member of the Program in Epithelial Biology and the Stanford Cancer Biology Program, as well as director of the Stanford Bullous Disease and Psoriasis Clinics at Stanford University, told HCPLive’s sister site CGTLive at the time of approval. "It's especially important to start treating at a young age, in order to help prevent chronic wounding and fibrosis issues."

REFERENCES

1. Abeona Therapeutics® Completes Pz-cel Biologics License Application Resubmission to U.S. Food and Drug Administration. News release. Abeona Therapeutics. October 29, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/291/abeona-therapeutics-completes-pz-cel-biologics-license

2. Abeona Therapeutics Provides Regulatory Update on Pz-cel. News release. Abeona Therapeutics. April 22, 2024. Accessed April 22, 2024. https://finance.yahoo.com/news/abeona-therapeutics-provides-regulatory-pz-200500676.html

3. Abeona Therapeutics announces positive topline results with both co-primary endpoints met in pivotal phase 3 VIITAL™ study of EB-101. News release. Abeona Therapeutics. November 3, 2022. Accessed April 22, 2024. https://www.globenewswire.com/news-release/2022/11/03/2547492/0/en/Abeona-Therapeutics-Announces-Positive-Topline-Results-with-Both-Co-Primary-Endpoints-Met-in-Pivotal-Phase-3-VIITAL-Study-of-EB-101.html

4. Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa. News release. Krystal Biotech. May 19, 2023. Accessed May 19, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene