FDA Grants Orphan Drug Designation To GlycoMimetics' GMI-1271 For Treatment of Acute Myleogenous Leukemia

GlycoMimetics, Inc. GLYC announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to GMI-1271, a novel and proprietary E-selectin antagonist in the company's pipeline for treatment of patients with acute myeloid leukemia (AML). GlycoMimetics is currently recruiting patients in a Phase 1/2, open-label multicenter study designed to evaluate the safety, pharmacokinetics and efficacy of GMI-1271 in combination with chemotherapy in adult patients with AML; a Phase 1 study in healthy volunteers was completed 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," said Helen Thackray, M.D., FAAP, Vice President of Clinical Development and Chief Medical Officer, GlycoMimetics. "We look forward to advancing GMI-1271 through clinical trials targeting E-selectin for multiple blood, or hematologic, cancers." The FDA's Orphan Drug designation program is designed to promote the development of promising therapeutics for the treatment of rare diseases affecting fewer than 200,000 people in the United States. Orphan Drug designation includes benefits such as a potential seven-year period of U.S. marketing exclusivity after approval. Other potential advantages include protocol assistance, the ability to apply for research funding, tax credits for certain research expenses, and regulatory fee waivers.