NEW YORK – Abeona Therapeutics on Tuesday said that it has resubmitted a biologics license application (BLA) to the US Food and Drug Administration for its investigational gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB).
The FDA in April issued a complete response letter to Cleveland-based Abeona's original BLA for prademagene zamikeracel, or pz-cel for short, in which the regulator asked for additional chemistry, manufacturing, and controls (CMC) information. Abeona said it held a Type A meeting with the FDA in August to discuss the information it should include in its resubmission.
"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," Abeona CEO Vish Seshadri said in a statement.
Pz-cel uses a retroviral vector to incorporate a functional collagen-producing COL7A1 gene into patients' autologous skin cells that are then transplanted back to wound areas in an effort to treat RDEB. The rare genetic skin disorder is caused by mutations in the COL7A1 gene, which hinder production of functioning type VII collagen that's needed for strengthening the layers of the skin.
Abeona expects the FDA will set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel if it accepts the new BLA. After accepting the original BLA, the FDA had granted the gene therapy priority review and said it expected to decide whether to grant it marketing approval by May 25.