Abeona Therapeutics, a clinical-stage biopharmaceutical company, has received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its for its gene therapy in epidermolysis bullosa in EB-101.
Gene therapy
“EB-101 is an autologous gene-corrected cell therapy approach that utilises a patient’s own cells and genetically re-engineers them to produce the missing collagen protein, which helps hold skin on to the body. This reduces the number of painful blisters caused by injury and has demonstrated improved wound healing in our Phase I/II clinical trial for over 2 years,” said Timothy J. Miller, PhD, president and CEO of Abeona. “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 III trial set to begin later this year.”
RMAT is similar to breakthrough therapy designation in that companies can work more closely and frequently with the FDA during the development of their regenerative medicine therapies. Additionally, any RMAT-designated products may be eligible for priority review and accelerated approval.