Abeona Therapeutics

The FDA accepted Abeona Therapeutics' resubmission of a BLA for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of April 29, 2025. Pz-cel is an autologous, COL7A1 gene-corrected epidermal sheet therapy made from patients' own skin cells, granted multiple designations by the FDA. The BLA is supported by phase 3 and phase 1/2 studies showing efficacy and safety in RDEB patients.

Abeona Therapeutics announced the FDA accepted its BLA resubmission for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of April 29, 2025.

Abeona Therapeutics announced FDA acceptance of its BLA resubmission for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of April 29, 2025.

Abeona Therapeutics resubmits BLA for pz-cel; RiboX Therapeutics receives IND clearance for RXRG001; Nanoscope Therapeutics' MCO-010 improves visual acuity in RP patients; HuidaGene Therapeutics' HG202 receives IND clearance for nAMD; CARsgen's clinical holds removed for BCMA, Claudin18.2, and GPRC5D CAR-T therapies; Lyell Immunopharma acquires ImmPACT Bio, focusing on IMPT-314 and LYL119; EvolveImmune collaborates with AbbVie to develop therapeutic antibodies for cancer.

Abeona Therapeutics resubmitted the BLA for prademagene zamikeracel (pz-cel) after a Type A meeting with the FDA in August 2024, addressing CMC concerns from the April 2024 CRL. The FDA did not request additional clinical trials or data, focusing solely on CMC validation. The original BLA was submitted in September 2023 and accepted with priority review in November 2024, supported by phase 3 and 1/2a study data showing significant wound healing and pain reduction in RDEB patients.

Abeona Therapeutics resubmits Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to FDA for recessive dystrophic epidermolysis bullosa (RDEB) treatment, incorporating Agency feedback and addressing Chemistry Manufacturing and Controls items.

Abeona Therapeutics resubmits BLA to FDA for pz-cel, its gene therapy for RDEB, incorporating FDA feedback and addressing CMC items. The resubmission is supported by Phase 3 and Phase 1/2a clinical data, with no new clinical trials requested. Abeona expects a new PDUFA target action date upon BLA acceptance.

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Abeona Therapeutics receives a Complete Response Letter from the FDA for its Biologics License Application for prademagene zamikeracel (pz-cel) due to unresolved Chemistry Manufacturing and Controls (CMC) requirements, with no deficiencies identified in clinical efficacy or safety data. The company plans to resubmit the BLA in the third quarter of 2024.