The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to SEL24/MEN1703, an orally available, dual PIM/FLT3 inhibitor designed for the treatment of Acute Myeloid Leukemia (AML). Licensed by the Menarini group from Ryvu Therapeutics, SEL24/MEN1703 is currently undergoing investigation as a single agent with hopes to be an effective solution for patients with relapsed or refractory cases of AML. Throughout the multi-stage trial, the novel drug has shown an ability to maintain a safety profile up to the recommended dose, in addition to initial evidence of its ability to act as an anti-leukemic agent. The FDA’s ODD status, the designation given to SEL24/MEN1703, is intended to encourage companies to use therapeutic development and diagnostics to address conditions such as AML, rare diseases that impact less than 200,000 people in the US.
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