On May 25, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported that the European Commission, based on a favorable recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products, has granted orphan designation for the company’s drug candidate GMI-1271 for the treatment of acute myeloid leukemia (AML) (Press release, GlycoMimetics, MAY 25, 2017, View Source [SID1234519296]). The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation for GMI-1271 for the treatment of AML in May of 2015. Schedule your 30 min Free 1stOncology Demo! GMI-1271, a specific E-selectin inhibitor is being evaluated in the company’s ongoing Phase 1/2 clinical trial, in which clinicians are evaluating the use of GMI-1271 along with chemotherapy in patients with relapsed or refractory AML as well as those with newly diagnosed AML. Earlier this month, the company announced that GMI-1271 had been granted Breakthrough Therapy designation by the FDA. The company also announced that abstracts had been published by both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper) highlighting new data from the Phase 2 portion of the company’s ongoing Phase 1/2 trial that will be presented at their upcoming annual meetings in June.
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"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."
The European Commission grants orphan designation to drugs intended to treat, prevent or diagnose life-threatening or chronically debilitating rare disorders, defined as 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. Orphan designation provides benefits including commercialization incentives, protection of intellectual property, including 10 years of market exclusivity and protocol assistance through the EMA’s Scientific Advice program.
About AML
AML is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. The National Cancer Institute estimates that there will be over 21,000 new cases of AML diagnosed in 2017 in the United States, and over 10,000 people will die from all forms of the disease in 2017. AML is more commonly present in elderly patients. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of AML, including anemia (shortage of red blood cells resulting in tiredness and weakness), neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.