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Devimistat receives orphan drug designation for the treatment of patients with relapsed or refractory Burkitt lymphoma by the European Medicines Agency
Burkitt lymphoma (BL) is an extremely rare subtype of non-Hodgkin Lymphoma (NHL), representing 2% of NHL diagnoses in the UK. Moreover, BL is more common in children than adults, with up to 30% of cases of childhood represented by BL. At the moment, treatment options for such patients are limited after first line of therapy.
Devimistat has received orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer, acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), peripheral T-cell lymphoma (PTCL), soft tissue sarcoma, biliary tract cancer, and BL, and by the European Medicines Agency (EMA) for the treatment of pancreatic cancer, and AML. Devimistat targets the mitochondrial tricarboxylic acid (TCA) cycle, which affects the metastatic activity of tumor cells by increasing cellular stress and cellular sensitivity to various chemotherapeutic agents. This synergistic effect may allow for devimistat to be combined with lower doses of cytotoxic chemotherapeutic agents to improve their efficacy with reduced side effects.
This orphan drug designation brings new hope for improved survival for patients with relapsed or refractory (R/R) BL1.
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