Rare Disease Report

Abeona Gene Therapy for Epidermolysis Bullosa Continues to Impress the FDA

AUGUST 29, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy to Abeona Therapeutics’ EB-101 gene therapy for epidermolysis bullosa (EB). 
EB patients have extremely delicate skin, often referred to as “butterfly skin,” because it becomes fragile – like the wings of a butterfly – and can fall apart at the touch. The disorder makes patients susceptible to blisters and poor healing of wounds. Recessive dystrophic epidermolysis bullosa is the most severe form of the condition, and is the result of a mutation in COL7A1 gene, which encodes for collagen VII. Abeona’s EB-101 is a skin graft that includes a healthy COL7A1 gene.

Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions and it was granted based on data from the Phase 1/2 clinical trial showing significant wound healing (greater than 50% healed) over a 2-year period.

The EB-101 program has already been granted Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA).

 “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,” stated Timothy J. Miller, Ph.D., Abeona’s President and CEO. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal Phase 3 trial as we advance EB-101 for patients with this debilitating disease.”

In May of this year, data from the Phase 1/2 trial were presented at the Society of Investigative Dermatology (SID) conference by Stanford collaborators, and demonstrated that EB-101 treated wounds were significantly healed >50% for more than two years post-administration. The data included:

An example of wound healing that occurred using the gene therapy is shown below.

Look for out exclusive interview with Dr. Miller later this week when we talk to him about EB-101 and the company’s other gene therapies.

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