Mocravimod granted Orphan Drug Designation by the FDA

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