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A Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)

Trial Profile

A Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 05 Feb 2024

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At a glance

  • Drugs Prademagene zamikeracel (Primary)
  • Indications Epidermolysis bullosa dystrophica
  • Focus Proof of concept; Registrational; Therapeutic Use
  • Sponsors Abeona therapeutics
  • Most Recent Events

    • 01 Feb 2024 According to an Abeona Therapeutics media release, company announced, as part of the review process by the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), completion by the FDA of both a Bioresearch Monitoring (BIMO) inspection of Abeona and the BLA mid-cycle review meeting.
    • 27 Nov 2023 According to an Abeona Therapeutics media release, the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024 based upon clinical efficacy and safety data from the pivotal Phase 3 VIITAL study (NCT04227106) and confirmatory evidence from a Phase 1/2a study (NCT01263379).
    • 26 Sep 2023 According to an Abeona Therapeutics media release, company has submitted a BLA to the U.S. Food and Drug Administration seeking approval of EB-101 for patients with recessive dystrophic epidermolysis bullosa. As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDAs review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review. Company expects potential BLA approval in the second quarter of 2024.
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