• The European Medicines Agency (EMA) has granted orphan drug designation to Rafael Pharmaceuticals' devimistat for treating refractory or relapsed Burkitt's lymphoma.
• Burkitt's lymphoma, a rare and aggressive form of non-Hodgkin's lymphoma, disproportionately affects children, representing up to 30% of pediatric NHL cases.
• Devimistat targets the mitochondrial tricarboxylic acid cycle, crucial for tumor cell proliferation, and has received multiple orphan drug designations from both EMA and FDA.
• A Phase 2 trial is underway to investigate devimistat's efficacy in relapsed or refractory Burkitt's lymphoma, offering hope for patients with limited treatment options.