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: 16 Aug 2024
Price :
$35
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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
- 12 Aug 2024 According to an Abeona Therapeutics media release, On August 8, 2024, company completed a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss Abeona's forthcoming resubmission of its Biologics License Application (BLA) for prademagene zamikeracel in development for recessive dystrophic epidermolysis bullosa.
- 12 Aug 2024 According to an Abeona Therapeutics media release, in premeeting communications and during the Type A meeting, company shared data and reports addressing nearly all of the deficiencies noted in the CRL and gained the FDAs preliminary alignment pending formal review. Company continues to expect to resubmit the BLA in the second half of 2024. Upon acceptance of the BLA, company expects the FDA to set a Prescription Drug User Fee Act (PDUFA) action date 6 months from the date of submission.
- 12 Aug 2024 According to an Abeona Therapeutics media release, In April 2024, Abeona received a CRL from the FDA based on the need for additional Chemistry Manufacturing and Controls (CMC) information. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel.