FDA accepts BLA resubmission for pz-cel to treat recessive dystrophic epidermolysis bullosa

FDA accepts BLA resubmission for pz-cel to treat recessive dystrophic epidermolysis bullosa | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

A resubmission Biologics License Application for prademagene zamikeracel (pz-cel) as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) has been accepted by the FDA, according to a press release from Abeona Therapeutics.¹

With this acceptance, the federal agency has assigned a target action date of April 25, 2025, for an approval decision.

Pz-cel, an investigational autologous, COL7A1 gene-corrected epidermal sheet, was granted FDA Priority Review on November 28, 2023, with an original target action date of May 25, 2024.² However, following a Complete Response Letter (CRL) that was issued in April 2024, any potential approval decision was delayed.³

The CRL was issued after the FDA requested additional information regarding Chemistry Manufacturing and Control requirements being “satisfactorily resolved” prior to a potential approval for pz-cel, according to prior coverage from Contemporary Pediatrics.³

The BLA resubmission was supported by efficacy and safety data following a 1-time administration of pz-cel demonstrated in the phase 3 VITAL study (NCT04227106), as well as a phase 1/2a study (NCT01263379) with up to 8 years of follow up.¹

Both studies demonstrated that 1-time administration of pz-cel on "large and chronic" wounds revealed wound healing and pain reduction of RDEB, a rare connective tissue disorder characterized by skin wounds that result in pain and can lead to systemic complications.²

"In this Phase 3 trial of patients with RDEB, supported by Abeona and completed at Stanford University and the University of Massachusetts, investigators implanted sheets impregnated with the EB101 gene," stated Bernard A. Cohen, MD, at the time of Priority Review Designation.²

Cohen is a professor of pediatrics and dermatology atJohns Hopkins University School of Medicine in Baltimore, Maryland, and is a editorial advisory board member of Contemporary Pediatrics.²

"This turns on production of Collagen type VII and results in production of anchoring fibrils that attach the epidermis to dermis," said Cohen. "The results of treated ulcers compared with control, untreated ulcers were dramatic with healing of chronic wounds within 12 weeks and a decrease in pain."²

According to Abeona, pz-cel would be the first autologous, cell-based gene therapy for RDEB and the first RDEB treatment designed to provide VII expression at wound sites via a stably integrated copy of the COL7A1 gene, if approved.¹

Pz-cel has also been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Orphan Drug Designations by the FDA.¹

References:

1. Abeona Therapeutics announces FDA acceptance of BLA resubmission of pz-cel for the treatment of recessive dystrophic epidermolysis bullosa. Abeona Therapeutics. Press release. November 12, 2024. Accessed November 13, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/294/abeona-therapeutics-announces-fda-acceptance-of-bla

2. Fitch, J. Pz-cel granted FDA Priority Review to treat recessive dystrophic epidermolysis bullosa. Contemporary Pediatrics. November 28, 2023. Accessed November 13, 2024. https://www.contemporarypediatrics.com/view/pz-cel-granted-fda-priority-review-to-treat-recessive-dystrophic-epidermolysis-bullosa

3. Fitch, J. CRL issued for pz-cel as potential recessive dystrophic epidermolysis bullosa treatment. Contemporary Pediatrics. April 23, 2024. Accessed November 13, 2024. https://www.contemporarypediatrics.com/view/crl-issued-pz-cel-recessive-dystrophic-epidermolysis-bullosa-treatment