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Mocravimod granted Orphan Drug Designation by the FDA
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On November 27, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to mocravimod (also known as KRP203), a novel synthetic sphingosine-1-phosphate receptor modulator, to improve outcome after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with hematologic malignancies.1
Mocravimod was previously granted Orphan Drug Designation by the U.S. FDA and the European Medicines Agency (EMA) for the treatment of patients with acute myeloid leukemia undergoing allo-HSCT.2 The safety and efficacy of mocravimod, both as an adjunctive and maintenance therapy, for patients with acute myeloid leukemia receiving allo-HSCT is currently being investigated in the phase III MO-TRANS trial (NCT05429632), which will enroll around 250 adult patients.1
- Priothera–US FDA grants Orphan Drug Designation to mocravimod to improve the outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with hematologic malignancies. https://priothera.com/priothera-us-fda-grants-orphan-drug-designation-to-mocravimod-to-improve-the-outcome-of-allogeneic-hematopoietic-stem-cell-transplantation-allo-hsct-in-patients-with-hematologic-malignanci/. Published Nov 27, 2023. Accessed Nov 27, 2023.
- Priothera–FDA and EMA Grant Orphan Drug Designation to mocravimod for the treatment of Acute Myeloid Leukemia (AML) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). https://priothera.com/priothera-fda-and-ema-grant-orphan-drug-designation-to-mocravimod-for-the-treatment-of-acute-myeloid-leukemia-aml-in-patients-undergoing-allogeneic-hematopoietic-stem-cell-transplantation/. Published Mar 3, 2022. Accessed Nov 27, 2023.
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