Rare Daily Staff

The U. S. Food and Drug Administration granted Abeona Therapeutics Regenerative Medicine Advanced Therapy designation for its EB-101, the company’s gene-corrected autologous cell therapy for patients with recessive dystrophic epidermolysis bullosa, a genetic condition that causes painful blistering of the skin.

People with recessive dystrophic epidermolysis bullosa lack functional type IIV collagen due to a mutation in COL7A1 gene. EB-101 is an autologous gene-corrected cell therapy that utilizes a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin onto the body.

A phase 1/2 clinical trial showed that the therapy reduces the number of painful blisters caused by injury and has demonstrated improved wound healing for more than two years, the company said.

“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,” said Timothy Miller, president and CEO of Abeona.

Miller said the designation will help accelerate the development of EB-101. The company is continuing discussions with the FDA in defining the pathway forward for the phase 3 trial set to begin later this year.

Established under the 21st Century Cures Act, the RMAT designation 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.

Similar to Breakthrough Therapy designation, the RMAT allows companies developing regenerative medicine therapies to work more closely and frequently with the FDA, and RMAT-designated products may be eligible for priority review and accelerated approval. In November 2017, the FDA expanded the RMAT designation for gene therapies.

January 29, 2018

Photo: Timothy Miller, president and CEO of Abeona Therapeutics