Rare Disease Report

Abeona Gets RMAT Designation for Epidermolysis Bullosa Gene Therapy

JANUARY 29, 2018
Mathew Shanley
This morning, Abeona Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted EB-101, its gene therapy for epidermolysis bullosa (EB), Regenerative Medicine Advanced Therapy (RMAT) designation.

In August, the company received Breakthrough Therapy Designation for the EB-101 program, after it had already been granted Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA).

The autologous gene-corrected cell therapy is essentially a skin graft that includes a healthy COL7A1 gene, the gene that is most commonly mutated in patients with recessive dystrophic EB. It uses a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin on the body.

Patients with EB suffer from extremely delicate skin – often called “butterfly skin” because of its fragility – that can fall apart at the touch. Those who suffer from the condition are susceptible to blisters and poor wound healing.

“(The therapy) reduces the number of painful blisters caused by injury and has demonstrated improved wound healing in our Phase 1/2 clinical trial for over 2 years,” said Timothy J. Miller, Ph.D., President and CEO of Abeona in a press release.

In May, data from Abeona’s Phase 1/2 trial of the gene therapy were presented at the Society of Investigative Dermatology (SID) conference by Stanford collaborators, and demonstrated that EB-101 can provide significant wound healing (more than 50%) over a 2-year period. The trial met its primary endpoints for safety and efficacy, as well as secondary endpoints including expression of collagen C7 and restoration of anchoring fibrils at 3 and 6-months post-administration.

"The receipt of the RMAT and Breakthrough designations, both over the last six months, reaffirms the significance of the EB-101 clinical trial results and the need to advance promising therapies in areas of considerable unmet medical need. We are pleased that the FDA granted the RMAT designation, which will help accelerate the development of EB-101, and look forward to continuing our collaborative discussions in defining the pathway forward for the Phase 3 trial set to begin later this year.”

The RMAT designation was established under the 21st Century Cures Act and is an expedited program for the advancement and approval of regenerative medicine products where preliminary clinical evidence indicates the potential to address unmet medical needs for life-threatening diseases or conditions. With it, Abeona is permitted to work more closely and frequently with the FDA, and EB-101 may now be eligible for priority review and accelerated approval.

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