Rare Disease Report

GlycoMimetics' Drug Granted EU Orphan Drug Designation for AML Treatment

MAY 25, 2017
Mathew Shanley
GlycoMimetics, Inc. announced that orphan designation for the company’s drug candidate GMI-1271 was granted for the treatment of acute myeloid leukemia (AML).

While the European Commission, based on a recommendation from the European Medicines Agency (EMA) committee for Orphan Medicinal Products, granted the designation on Thursday, the U.S. Food and Drug Administration (FDA) had previously granted the same designation in May of 2015.

Most cases of AML develop from cells that turn into white blood cells, and can progress quickly if not treated. It is likely to become fatal if not properly diagnosed within the first few months. GMI-1271 is a specific E-selectin inhibitor and is being evaluated in an ongoing Phase 1/2 clinical trial. In the trial, clinicians are evaluating the use of the drug in addition to chemotherapy in patients with relapsed or refractory AML, as well as those with newly diagnosed AML.

“The European orphan designation will provide incentives for the commercialization and development of GMI-1271 in AML, where there are limited therapies available to patients,” said Helen Thackray, M.D., FAAP, Senior Vice President, Clinical Development and Chief Medical Officer of GlycoMimetics.

"We believe that GMI-1271, when combined with standard chemotherapy, has the potential to address an unmet therapeutic need for individuals living with AML, and we are encouraged by both our clinical results to date and achieving this designation from the European Commission."

Orphan designation for drugs intended to treat, prevent or diagnose life-threatening or chronically debilitating rare disorders, are granted by the European Commission to diseases with prevalence of no more than 5 in 10,000 in the EU, for which no satisfactory method of diagnosis, prevention or treatment yet exists. It was designed to provide benefits, such as commercialization incentives and protection of intellectual property, including 10 years of market exclusivity and protocol assistance through the EMA’s Scientific Advice program.

In April, Rare Disease Report recognized AML World Awareness Day in an effort to promote the history and causes of, as well as the treatment options for AML. Additionally, the day introduces patients and advocates to the available resources and support groups for the disease with help from the Know AML Coalition.

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