Currently, the company is enrolling patients in an open-label multicenter Phase I/II trial, which is designed to evaluate the safety, pharmacokinetics and efficacy of GMI-1271 in combination with chemotherapy in adult patients with AML.
The company has also completed a Phase I trial in healthy volunteers last year.
GlycoMimetics Clinical Development vice-president and chief medical officer Dr Helen Thackray said: "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.
"We look forward to advancing GMI-1271 through clinical trials targeting E-selectin for multiple blood, or hematologic, cancers."
AML is a cancer of the blood and bone marrow and is the most common type of acute leukemia in adults, with more than 18,000 new cases and over 10,000 deaths in 2014.
The company is focused on the discovery and development of new glycomimetic drugs to address unmet medical needs resulting from diseases in which carbohydrate biology plays a major role.