Abeona Therapeutics has resubmitted its Biologics License Application (BLA) to the FDA for prademagene zamikeracel (pz-cel; EB-101), an investigational autologous gene-corrected epidermal sheet therapy intended for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows a Complete Response Letter (CRL) received earlier this year. This therapy represents a potential advancement in treating this debilitating genetic skin disorder.
The company's decision to resubmit the BLA was made after reaching an agreement with the FDA during a Type A meeting in August 2024. The discussion focused on providing additional Chemistry, Manufacturing, and Controls (CMC) information.
Addressing FDA Concerns
The CRL, issued in April 2024, requested further CMC information, specifically regarding validation requirements for certain manufacturing and release testing methods. It's important to note that the FDA did not raise concerns about the clinical efficacy or safety data included in Abeona's original BLA submission for pz-cel, nor did they request additional clinical trials or data.
Vish Seshadri, PhD, MBA, CEO of Abeona, stated, "We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance. We have incorporated the agency’s feedback and are confident that our resubmission package addresses all the CMC items identified in the CRL, 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."
Clinical Data Supporting the BLA
Abeona's BLA for pz-cel was initially submitted in September 2023 and granted priority review on November 27, 2024. The application is supported by data from the phase 3 VIITAL study (NCT04227106) and a phase 1/2a study (NCT01263379).
In November 2022, results from the VIITAL study showed that pz-cel treatment significantly improved wound healing and reduced pain in 11 participants with 43 wound pairs. The study met its primary endpoints, demonstrating over 50% wound healing and a greater magnitude of pain reduction at 6 months post-treatment compared to baseline and control wounds. Pz-cel was well-tolerated, with no serious treatment-related adverse events reported.
The Patient Perspective
Brett Kopelan, executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, commented in November 2022, "I am incredibly enthused to see new clinical evidence of EB-101’s potential to treat the more difficult chronic and large wounds. Our patient community needs options to address not only the healing of wounds but also the chronic pain and the acute treatment related pain of daily wound care associated with these wounds. Today’s standard of care comprises hours of brutal and painful wound care, and EB-101’s promise to be a transformational option for RDEB patients is truly exciting."
