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TolerogenixX Has Received EMA Orphan Drug Designation for MIC-Lx, Strengthening its Regulatory Pathway in Kidney Transplantation
- Supporting the development of its proprietary MIC-Lx Modified Immune Cells technology as a novel approach to reduce lifelong systemic immunosuppression and improve long-term outcomes for transplant recipients
- Strengthening MIC-Lxâs regulatory and commercial positioning in Europe
TolerogenixX, a German clinical-stage biopharmaceutical company developing personalized cell therapies inducing antigen-specific immune tolerance, today announced that the European Commission has granted MIC-Lx, the Company’s lead product candidate, Orphan Drug Designation (ODD) for living-donor kidney transplantation following a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).
The Orphan Drug Designation follows the recent granting of EMA PRIME designation for MIC-Lx, marking a second key regulatory milestone and further strengthening the Company’s pathway toward full regulatory approval.
Both designations recognize the significant unmet medical need in kidney transplantation addressed by MIC-Lx. Despite major advances in transplantation medicine, recipients today generally require lifelong systemic immunosuppressive therapy to prevent organ rejection. While effective, these therapies are associated with substantial long-term complications, including infections, malignancies, cardiovascular disease and chronic kidney damage, which continue to limit long-term patient outcomes.
Unlike conventional immunosuppressive therapies, MIC-Lx is designed to induce donor-specific immune tolerance before transplantation, enabling targeted protection of the transplanted organ while preserving the patient’s overall immune competence. Based on TolerogenixX’s proprietary Modified Immune Cell (MIC) technology, MIC-Lx aims to reduce dependence on lifelong systemic immunosuppression and improve graft survival and patients’ long-term quality of life.
“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 Prof. Dr. 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.â
âKidney transplant recipients still face the long-term burden of systemic immunosuppression, including infection risk, malignancies, cardiovascular complications, and chronic graft injury,â said Prof. Dr. Christian Morath, Chief Scientific Officer of TolerogenixX. âMIC-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.â
In addition to recognizing the therapy’s clinical rationale, Orphan Drug Designation provides regulatory and commercial incentives that support the development of innovative medicines for rare diseases. These include protocol assistance from the EMA, significant fee reductions during development and regulatory review, and, subject to marketing authorization, up to ten years of market exclusivity in the European Union.
MIC-Lx is currently being evaluated in a randomized, controlled Phase IIb TOL-2 trial in living-donor kidney transplantation. Following the successful treatment of all 63 donor-recipient pairs, the study has entered its follow-up phase, with topline results expected in the first half of 2027.
MIC-Lx has already completed a clinical Phase Ib trial (TOL-1) in kidney transplant recipients demonstrating sustained safety and tolerability after a single administration while preserving normal immune responses.
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About TolerogenixX
TolerogenixX is a privately held biopharmaceutical company developing novel, personalized therapies to induce antigen-specific immune tolerance in transplantation and autoimmune diseases, creating the foundation for a broader immune tolerance platform. Its proprietary MIC (modified immune cells) technology aims to modulate the immune system in a targeted manner, potentially reducing the need for long-term systemic immunosuppression.
The Companyâs lead candidate MIC-Lx has completed a Phase Ib clinical trial (TOL-1) in kidney transplant recipients demonstrating sustained safety and tolerability after a single administration while preserving normal immune responses. A Phase IIb study (TOL-2) is currently ongoing. TolerogenixX was founded in 2016 and is headquartered in Heidelberg, Germany.
About MIC treatment
TolerogenixX’s proprietary MIC treatment is a personalized cell therapy designed to modulate the immune system through a novel mode of action. It not only benefits transplant recipients, but it could also benefit patients with autoimmune diseases such as systemic lupus erythematosus and multiple sclerosis. Unlike conventional immunosuppressive therapies, the platform is designed to preserve the patient’s overall immune competence.
MIC production is fast, safe, and effective. Using cells obtained by leukapheresis, MIC can be manufactured within 24 hours. Thanks to its standardized process, MIC production can be easily scaled up and supplied globally using the proprietary approach developed by TolerogenixX.