The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to TolerogenixX lead cell therapy candidate MIC-Lx for individualised immuno-suppression in living donor kidney transplantation.
Kidney transplantation is the preferred treatment for end-stage renal disease, yet patients require life-long systemic immunosuppressive therapy to prevent organ rejection. These treatments are associated with significant side effects, including infections, malignancies and metabolic complications. TolerogenixX’s MIC-Lx therapy—modified immune cells for living-donor transplants—aims to induce donor-specific immune tolerance, potentially allowing transplant recipients to significantly reduce chronic immunosuppression.
The PRIME scheme supports medicines addressing serious diseases with significant unmet medical need and provides early and enhanced regulatory interaction with the EMA to accelerate clinical development and patient access.
The PRIME designation for MIC-Lx therapy follows a positive assessment by the Committee for Advanced Therapies (CAT) and endorsement by the Committee for Medicinal Products for Human Use (CHMP) during its February 2026 meeting.
According to the EMA assessment, available clinical and non-clinical data support the hypothesis that modified donor immune cells treated with a cytostatic agent may induce a tolerogenic immune state in transplant recipients.
Clinical phase I and ongoing phase II studies in renal transplant patients with follow-up data of up to 10 years support the proposed mechanism of action and indicate absence of donor-specific antibody responses; substantial reduction of immunosuppressive therapy, and fewer opportunistic infections, TolerogenixX says in a press release.
Five-year follow-up data from the phase I study were published in Frontiers in Immunology in 2023 and showed long-lasting donor-specific immune modulation accompanied by a sustained increase in regulatory B lymphocytes.
“The EMA PRIME designation is an important recognition of the potential of our MIC technology to improve the current treatment paradigm in transplantation,” said Matthias Schaier, CEO of TolerogenixX. “Our goal is to enable donor-specific immune tolerance and reduce the burden of chronic immunosuppression for transplant patients. Clinical data generated so far indicate that our MIC therapy may provide an effective treatment option for kidney transplant recipients while reducing the side effects typically associated with long-term immunosuppression.”
The company plans to publish data from an ongoing phase IIb trial in the first half of 2027.
