Hybrigenics is one step closer to enter the American market. The FDA granted Orphan Drug designation to the company’s Acute Myeloid Leukemia (AML) Treatment. Currently in preclinical stage, the therapy showed efficacy in combination with two AML-drugs that are already approved.
AML is the second most frequent form of leukemia and accounts for about 30% of all leukemic patients. It is characterized by a fast-increasing overproduction of immature white blood cells, called myeloblasts. These cells rapidly crowd the bone marrow, soon preventing it from making normal blood cells. They can also spill out into the blood stream and circulate around the body. Due to their immaturity, they are unable to function properly.
Current treatments include the two hypo-methylating anticancer drugs azacytidine or decitabine. Hybrigenics’ lead candidate is inecalcitol, a Vitamin D inhibitor that is currently tested in combination with those two. Preclinical trials showed that the combination improved efficacy to limit leukemia progression than the two approved drugs agents alone. Based on these, the FDA granted Orphan drug designation to the therapy.