EC grants orphan drug designation to investigational cell therapy in living-donor kidney transplantation

On July 13, 2026, the European Commission (EC) granted orphan drug designation (ODD) to MIC-Lx, an investigational modified immune cell therapy for living-donor kidney transplantation, following a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) and a recent EMA priority medicines (PRIME) designation. MIC-Lx is designed to induce donor-specific immune tolerance before transplantation, with the aim of protecting the transplanted organ, reducing dependence on lifelong systemic immunosuppression, and preserving recipient immune competence. 

The designation follows ongoing evaluation of MIC-Lx in the randomized, controlled, phase IIb TOL-2 trial (NCT05365672) in living-donor kidney transplantation (N = 63 donor–recipient pairs). All donor–recipient pairs have received treatment, and the trial has entered follow-up, with topline results expected in the first half of 2027. A single administration was evaluated in the completed phase Ib TOL-1 trial (NCT02560220) in kidney transplant recipients, which demonstrated sustained safety and tolerability while preserving normal immune responses.