FDA Rejects Abeona’s PZ-CEL Topical Gene Therapy, Delaying Further Progress

1. Abeona Therapeutics Inc. has faced a setback as the U.S. Food and Drug Administration (FDA) rejected its topical gene therapy, PZ-CEL (EB-101), for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).

2. The FDA's rejection was based on concerns regarding the Chemistry, Manufacturing, and Controls (CMC) section of the Investigational New Drug (IND) application, which led to a delay in the therapy's progress.

3. PZ-CEL is a novel gene therapy designed to deliver a functional COL7A1 gene to patients with RDEB, a rare and severe genetic skin disorder.

4. Abeona Therapeutics Inc. will need to address the FDA's concerns and make necessary adjustments to the CMC section of the IND application before resubmitting it for approval.

5. The delay in the approval process will impact the timeline for further clinical trials and the potential commercialization of PZ-CEL.

6. Epidermolysis Bullosa is a group of rare, inherited skin disorders characterized by fragile skin and recurrent blister formation, leading to significant morbidity and early mortality.

7. PZ-CEL is one of the few gene therapies being developed for the treatment of RDEB, and its potential rejection by the FDA could have significant implications for patients and the broader gene therapy landscape.