Pharma Focus Europe

Maat Pharma Secures European Medicines Agency Orphan Drug Designation for MaaT033, With a Focus on Enhancing Survival in Hematopoietic Stem Cell Transplantation Patients

Friday, September 08, 2023

MaaT Pharma, a clinical-stage biotechnology firm traded on EURONEXT under the symbol MAAT, has announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) for their product, MaaT033. This designation is aimed at improving overall survival outcomes in patients undergoing Hematopoietic Stem Cell Transplantation (HSCT), recognizing the potential significant benefit it could offer to this patient group. MaaT033 is the second clinical-stage asset and a first-in-class therapeutic by the company to receive this designation. MaaT Pharma has previously obtained ODD status for MaaT013 in both the United States and Europe.

Philippe Moyen, COO of MaaT Pharma, expressed his appreciation for the EMA's decision, emphasizing the necessity for new treatment options for patients with liquid tumors. He highlighted the potential of microbiome-based drugs and their growing value in the company's clinical portfolio, pledging continued collaboration with regulatory agencies to expedite the development of safe and innovative microbiome therapies.

MaaT033 is an oral Microbiome Ecosystem Therapy derived from donors, characterized by its high richness and diversity. It contains anti-inflammatory Butycore species and is currently in development as an adjunctive therapy to enhance the overall survival of patients undergoing HSCT and other cellular therapies. Its primary objective is to ensure the optimal function of the microbiota and address a broader patient population in a chronic treatment context. The European Medicines Agency (EMA) has granted MaaT033 Orphan Drug Designation.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a medical procedure used in the treatment of liquid tumors. It involves replacing cancerous cells with healthy stem cells, but the rigorous conditioning treatments used in the process can damage the patient's gut microbiome. This damage has been associated with reduced survival rates, an increased risk of graft-vs-host disease, and higher susceptibility to infections due to compromised immune function. In 2021, nearly 20,000 allo-HSCT transplantations were reported in Europe, according to the European Society for Blood and Marrow Transplantation (EBMT), and this number continues to rise.

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