FDA Issues Complete Response Letter for Pz-Cel to Treat Epidermolysis Bullosa

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Prademagene zamikeracel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information on manufacturing practices.

The FDA has issued a complete response letter for the biologics license application (BLA) for prademagene zamikeracel (pz-cel) to treat patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is rare connective tissue disorder. The disorder causes severe and painful skin wounds and can lead to systemic complications. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen.

Developed by Abeona Therapeutics, pz-cel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information for the Chemistry Manufacturing and Controls (CMC) requirements.

Company officials said in a news release that they had planned to CMC data prior to BLA approval, with plans to provide full validation reports after approval in mid-2024. FDA officials, however, said in the CRL that he proposed timing of the data submission would not allow sufficient review before the May 25, 2024, PDUFA date.

Abeona said the information requested by the FDA pertains to validation requirements for certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA. Regulators did not request any new clinical trials or clinical data to support the approval of pz-cel.

Vish Seshadri

Vish Seshadri

“We anticipate completing the BLA resubmission in the third quarter of 2024 with necessary updates to fully satisfy all the deficiencies outlined in the CRL,” said Vish Seshadri, CEO of Abeona.

FDA Sets Review Date for Therapy for Rare Connective Tissue Disorder

The BLA is supported by clinical efficacy and safety data from the pivotal phase 3 VIITAL study and confirmatory evidence from a phase 1/2a study. Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.

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