FDA Grants Orphan Designation to ES-3000 for AML

The U.S. Food and Drug Administration granted a second orphan drug designation to ES-3000 (an orally bioavailable small molecule) for the treatment of acute myeloid leukemia. The drug previously received orphan drug designation for the treatment of chronic myeloid leukemia.

ES-3000 will be used in conjunction with standard agents to target leukemic stem cells for more durable remissions, according to the drug’s manufacturer. ES-3000 works by reducing β-catenin expression – a protein that is critical for the survival of leukemic stem cells.

Source: Escend Pharmaceuticals press release, December 28, 2016.

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