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FDA grants orphan drug designation to GMI-1271 for AML

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The FDA today granted orphan drug designation to GMI-1271 for the treatment of patients with acute myeloid leukemia, according to a press release from the drug’s manufacturer.

GMI-1271 (GlycoMimetics) — a novel and proprietary E-selectin antagonist — is currently being evaluated in a phase 1/2 open-label multicenter study of patients with AML who are also receiving chemotherapy. GlycoMimetics completed a phase 1 study of GMI-1271 in healthy volunteers last year.

"Having the FDA designate GMI-1271 as an orphan drug for the treatment of AML is an important accomplishment for GlycoMimetics. This is a significant regulatory milestone for our program," Helen Thackray, MD, FAAP, vice president of clinical development and chief medical officer of GlycoMimetics, said in a press release. "We look forward to advancing GMI-1271 through clinical trials targeting E-selectin for multiple … hematologic cancers."

The FDA’s 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.