December 28, 2016
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FDA grants orphan drug designation to ES-3000 for AML

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The FDA granted a second orphan drug designation to ES-3000 for the treatment of acute myeloid leukemia, according to the drug’s manufacturer.

ES-3000 (Escend Pharmaceuticals) — an orally bioavailable small molecule which ablates leukemic stem cells by reducing beta-catenin expression through a novel mechanism of action — previously received orphan drug designation for the treatment of chronic myeloid leukemia.

“This accomplishment validates the potential significance ES-3000 can have in shifting the AML treatment paradigm,” Saira Bates, co-founder and CEO of Escend, said in a press release. “The treatment of patients with AML is complicated by relapse and refractory disease which is often attributed to the resistance of leukemic stem cells to standard therapeutics.”

ES-3000 will be used in conjunction with standard agents to target leukemic stem cells for more durable remissions, Bates added.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.