FDA places hold on Abeona late-stage study

Sept. 23, 2019

Abeona Therapeutics announced that the FDA has placed a clinical hold on its planned Phase 3 clinical trial evaluating autologous cell therapy EB-101 in patients with recessive dystrophic epidermolysis bullosa.

The gene and cell therapy biotech said that the FDA will not provide approval for the company to begin its planned Phase 3 clinical trial for EB-101 until it submits additional data points on transport stability of EB-101 to clinical sites to the agency.

EB-101 is an investigational, autologous, gene-corrected cell therapy being studied for the treatment of epidermolysis bullosa — a rare connective tissue disease characterized by fragile skin that blisters quickly. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU

According to Texas-based Abeona, over the last 12 months, the drugmaker 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.  The drugmaker intends to resolve the issue as soon as feasible and maintains its expectation of FDA sign-off on the trial in Q4.

Read the press release