Abeona Receives Breakthrough Designation for Rare Skin Disease Gene Therapy

A gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease that causes the skin to be very fragile and blister easily, has been granted Breakthrough Therapy designation status by the FDA.

Abeona’s president and CEO, Timothy J. Miller, PhD, expressed gratitude at the FDA’s designation.

“EB-101 is an autologous gene-corrected cell therapeutic approach that utilizes a patient’s own cells and genetically engineering them to produce the correct version of collagen, which helps hold skin on to the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” Miller said in a statement.

The breakthrough designation was granted based on data from the phase 1/2 EB-101 clinical trial, which showed significant wound healing in treated wounds for more than 2 years after treatment. The designation, which is granted to drugs used to treat serious or life-threatening conditions, allows Abeona to expedite the development and review of EB-101.

RDEB is the most severe form of epidermolysis bullosa. EB-101 is a skin graft that includes a healthy COL7A1 gene, which is mutated in patients with RDEB.

In the phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject. The wounds were assessed at predefined time points to determine wound healing, and the researchers found significant wound healing—defined as more than 75% closure—in 94% of patients at 3 months, 67% of patients at 6 months, and 50% of patients at 12 months.

When the trial results were released in April 2017, and presented at the Society for Investigative Dermatology in Portland, Oregon, Miller outlined the benefits of the treatment.

“The EB-101 clinical data update highlights the wound closure and collagen biomarker expression continues for over 2 years in multiple patients, which are critically important parameters of efficacy in patients with RDEB,” he said.