Epidermolysis Bullosa Drug Gains Regenerative Medicine Advanced Therapy Designation

Abeona Therapeutics recently announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to EB-101, an experimental gene therapy being assessed in patients with recessive dystrophic epidermolysis bullosa (RDEB), according to a press release.

“EB-101 is an autologous gene-corrected cell therapy approach that utilizes a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin on to the body,” said Timothy J. Miller, PhD, president and CEO of Abeona. “This 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.”

RMAT, which was created under the 21^st Century Cures Act, expedites the approval of regenerative medicine products that show the potential to address unmet clinical needs, according to the FDA. This designation allows manufacturers to work closely with the FDA and also allows the products to receive priority review and accelerated approval.

Abeona said that it is continuing to collaborate with the FDA in designing a phase 3 clinical trial of EB-101, according to the release.

EB-101 is an autologous, ex-vivo gene-corrected cell therapy that works by inserting the COLA7A1 gene into the patient’s keratinocytes to address the cause of RDEB, according to Abeona.

In the phase 1/2 clinical trial, EB-101 was administered to patients with non-healing chronic wounds, which were then evaluated for wound healing.

The trial met its primary endpoints of safety and efficacy compared with the results of another study that included 128 patients with RDEB and 1500 chronic and recurring wounds. Secondary endpoints included collagen C7 expression and restoration of anchoring fibrils at 3 and 6 months.

The researchers found that all patients achieved wound healing—defined as >50% closure after EB-101 therapy—at 3 months, according to the release. At 6 months, 90% of patients achieved wound healing.

At 12 months and 24 months, 83% and 88% of patients achieved wound healing, respectively, according to the release.

Thus far, the EB-101 development program has been granted Breakthrough Therapy, Orphan Drug Designation, and Rare Pediatric Disease designations.

"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,” Dr Miller said. “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.”