Cornerstone Pharmaceuticals has received Orphan Drug Designation from the FDA for CPI-613, the company’s lead Altered Energy Metabolism Directed (AEMD) drug candidate, for the treatment of myelodysplastic syndrome (MDS). MDS is a form of cancer that occurs when the blood-forming cells in the bone marrow are damaged, which leads to low numbers of one or more type of blood cells. In about one-third of MDS cases, the disease progresses to a rapidly growing cancer of bone marrow cells called ...