Rare Daily Staff

Abeona Therapeutics said it received a clinical hold letter from the U.S. Food and Drug Administration denying approval for the company to begin its planned phase 3 clinical trial of its cell therapy EB-101 for the treatment of recessive dystrophic epidermolysis bullosa until it submits additional data points on transport stability of EB-101 to clinical sites.

Recessive dystrophic epidermolysis bullosa, or RDEB, is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB.

EB-101 is an experimental, autologous, gene-corrected cell therapy ready to enter late-stage development for the treatment of REDB. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal type VII collagen expression and facilitate wound healing. Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 in the United States, and Orphan Drug designation in both the United States and European Union.

Over the past 12 months, Abeona has worked closely with the FDA to address and narrow open Chemical, Manufacturing and Controls (CMC) items and has been working to resolve this one item identified in the FDA clinical hold letter. Abeona expects to receive CMC clearance for the trial by the end of the year.

“Initiating the VIITAL pivotal phase 3 trial for EB-101 is the company’s top priority,” said João Siffert, CEO of Abeona. “Efforts to gather supplemental data points on transport stability of EB-101 are ongoing and we are confident that the requested additional data will be submitted to the FDA promptly. Looking ahead, we believe that completion of our CMC work coupled with the durable safety and efficacy data, now out to five years in some patients, will ultimately be critical to support a future Biologics License Application.”

Photo: João Siffert, CEO of Abeona

X