European Medicines Agency Grants Orphan Drug Designation to Marker Therapeutics' MT-401 for AML Treatment
Tuesday, July 11, 2023
Marker Therapeutics, Inc. (Nasdaq: MRKR) has recently announced that its T cell-based immunotherapy, zedenoleucel (MT-401), has been granted Orphan Drug Designation by the European Medicines Agency (EMA) for the treatment of acute myeloid leukemia (AML). AML is a life-threatening disease with high relapse rates and poor outcomes following initial treatment and stem cell transplantation. MT-401, a multi-tumor-associated antigen (multiTAA)-specific T cell product candidate, is designed to target four antigens that are upregulated in AML while minimizing tumor escape.
Orphan Drug Designation is granted in the European Union to drugs intended for the treatment of rare and severe conditions affecting a limited number of individuals. This designation provides regulatory and financial incentives to facilitate the development and approval process. It includes ten years of market exclusivity after approval, reduced regulatory fees, and scientific advice from the EMA during the drug development phase.
Nadia Agopyan, Senior Vice President of Regulatory Affairs at Marker Therapeutics, highlighted the significance of this regulatory milestone and the company's commitment to advancing the development of MT-401 for the treatment of AML. In addition to the Orphan Drug Designation from the EMA, MT-401 was granted orphan drug designation by the U.S. Food and Drug Administration in 2020.
Juan F. Vera, President and CEO of Marker Therapeutics, expressed pride in receiving the Orphan Drug Designation from the EMA and emphasized the promising results observed in the Phase 2 clinical trial of MT-401 in patients with AML following stem cell transplantation. The unique targeting technology of MT-401 has the potential to provide a new treatment option for AML patients before relapse occurs.
Marker Therapeutics' multiTAA-specific T cell platform is a non-genetically modified cell therapy approach that selectively expands tumor-specific T cells capable of recognizing a broad range of tumor antigens. Clinical trials have demonstrated the safety and efficacy of autologous and allogeneic multiTAA-specific T cell products in various hematological malignancies and solid tumors. Notably, these T cells can be administered on an outpatient basis, setting them apart from other cell therapies that require hospitalization and close monitoring.
