First allogeneic cell therapy to receive US FDA BTD and RMAT designations

USA and Japan based privately held company Aurion Biotech announced that the US Food and Drug Administration (FDA) has granted both Breakthrough Therapy designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) designation for AURN001, the company’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease.

BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

RMAT is part of the 21st Century Cures Act. An investigational drug is eligible for RMAT designation if: it meets the definition of regenerative medicine therapy; is intended to treat, modify, reverse, or cure a serious condition; and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such conditions.