Drug Approval

TolerogenixX wins EMA orphan drug designation for kidney transplant therapy MIC-Lx

TolerogenixX is developing MIC-Lx as a personalized cell therapy designed to induce donor-specific immune tolerance before living-donor kidney transplantation

German clinical-stage biopharma company TolerogenixX has secured Orphan Drug Designation from the European Commission for its lead therapy candidate MIC-Lx, marking a major regulatory milestone in the development of a potential new approach to kidney transplantation.

The designation, granted following a positive recommendation from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), strengthens MIC-Lx’s regulatory and commercial position in Europe and follows the recent achievement of EMA PRIME designation.

TolerogenixX is developing MIC-Lx as a personalized cell therapy designed to induce donor-specific immune tolerance before living-donor kidney transplantation — with the goal of reducing patients’ reliance on lifelong systemic immunosuppressive medicines.

Current transplant patients typically require lifelong immunosuppression to prevent organ rejection. While effective, these treatments can increase the risk of serious long-term complications, including infections, malignancies, cardiovascular disease and chronic kidney damage.

MIC-Lx is based on TolerogenixX’s proprietary Modified Immune Cell (MIC) technology, which aims to protect transplanted organs while preserving the patient’s broader immune function.

"Receiving both PRIME and Orphan Drug Designation from the EMA is an important recognition of our first-in-class approach and supports the ongoing development of MIC-Lx," said Matthias Schaier, Chief Executive Officer of TolerogenixX. "Our goal is to enable long-term graft protection without lifelong systemic immunosuppression, thereby addressing one of the key challenges in transplantation medicine."

Christian Morath, Chief Scientific Officer of TolerogenixX, added: "Kidney transplant recipients still face the long-term burden of systemic immunosuppression, including infection risk, malignancies, cardiovascular complications, and chronic graft injury.

"MIC-Lx is designed to induce donor-specific immune tolerance before transplantation, with the goal of protecting the graft while preserving broader immune competence. The Orphan Drug Designation further supports the clinical rationale for advancing this approach."