The FDA has granted Agios Pharmaceuticals’ Orphan Drug designation for AG-120 for treatment of patients with acute myelogenous leukemia (AML). AG-120 is an oral, first-in-class IDH1 mutant inhibitor being evaluated in a phase I clinical trial in patients with advanced hematologic malignancies that carry an IDH1 mutation.
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides to Agios certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
“Receiving Orphan Drug designation for AG-120 is an important milestone as we continue to move this program to late-stage development,” said Chris Bowden, M.D., chief medical officer of Agios. “We are pleased with the progress we are making in the clinic and look forward to presenting new data from our ongoing phase I study of AG-120 at the Congress of the European Hematology Association later this week. We believe that AG-120, which is on track to initiate multiple expansion cohorts in the next month, has the potential to play a significant role in shifting the treatment paradigm for IDH1-mutant positive hematologic cancers from the conventional chemotherapy approach.”
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% 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%. IDH1 mutations are present in about 6% to 10% of AML cases.