FDA Orphan Drug Designation Granted To AG-120 To Treat IDH1-Mutant Positive AML

(RTTNews) - Agios Pharmaceuticals Inc. (AGIO) announced that the U.S. Food and Drug Administration or FDA has granted the company orphan drug designation for AG-120 for treatment of patients with acute myelogenous leukemia or AML.

AG-120 is an oral, first-in-class IDH1 mutant inhibitor being evaluated in a Phase 1 clinical trial in patients with advanced hematologic malignancies that carry an IDH1 mutation.

AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia in adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and according to the American Cancer Society the median age is 66. Less than 10 percent of U.S. patients are eligible for bone marrow transplant, and the vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases.