Rafael Pharmaceuticals granted Orphan Drug Designation by EMA for devimistat
Rafael Pharmaceuticals announced that the European Medicines Agency, or EMA, has granted orphan drug designation to devimistat, the company's lead Altered Metabolism Directed, or AMD, drug candidate, for the treatment of older patients with relapsed or refractory Acute Myeloid Leukemia, or AML. AML is a hematologic malignancy characterized by the accumulation of clonal myeloid progenitor cells in the blood or bone marrow. Malignant hematopoietic stem and myeloid progenitor cells proliferate uncontrollably and disrupt normal hematopoiesis, resulting in impairments in the ability of these myeloid progenitor cells to differentiate. Many patients who achieve a remission and complete consolidation therapy still have a guarded prognosis. This is driven by the fact that over 50% of patients will experience a relapse, and most of them will die from AML within a year. There is no consensus standard treatment for relapsed or refractory disease, but most fit patients are treated with a high dose cytarabine based regimen.