Agios Gets FDA Fast-Track Designation for its Experimental Leukemia Drug

Editor’s note: The U.S. Food and Drug Administration (FDA) has granted a “Fast-Track” designation to a new drug for certain patients with acute myelogenous leukemia (AML). The drug, called AG-221, is a targeted therapy that is meant to treat people with AML whose tumors have mutations in the isocitrate dehydrogenase-2 protein (IDH2), as detected by molecular testing. The Fast-Track designation means that the FDA will facilitate a faster approval process so that the drug can soon be prescribed by oncologists in the U.S.

“Agios Pharmaceuticals Inc., a Cambridge company seeking to develop new treatments for cancer, said Wednesday that the US Food and Drug Administration has granted a so-called “Fast Track” designation to its experimental treatment for a type of acute myelogenous leukemia, or AML.

“The FDA’s fast track program is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application, or NDA. The designation is given to medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. The program provides the opportunity to submit sections of a new drug application on a rolling basis as data become available. This permits the FDA to review portions of the new drug application as they are received instead of waiting for the entire application submission.

“Agios currently calls its drug candidate AG-221, and it is designed to the treat patients with AML who harbor an isocitrate dehydrogenase-2 mutation.”


Cancer Metabolism Drug AG-221 Shows Clinical Activity in Advanced Blood Cancers

“AG-221, a novel inhibitor of isocitrate dehydrogenase (IDH) 2-mutant metabolic enzyme, was well tolerated and showed early promise in patients with advanced and refractory blood cancers harboring IDH2 mutations, according to the initial results of a phase I study presented here at the AACR Annual Meeting 2014, April 5-9.

” ‘Mutations in the genes for the metabolic enzymes IDH1 and IDH2 are thought to be the drivers of distinct subsets of acute myeloid leukemias (AML),’ said Eytan M. Stein, M.D., assistant attending physician in the Leukemia Service at Memorial Sloan Kettering Cancer Center in New York. ‘They lead to the production of increased levels of an oncometabolite called 2-hydroxyglutarate (2-HG), which is hypothesized to prevent normal healthy bone marrow cells from maturing, leading to cancer.’ ”

Editor’s note: AG-221 is a targeted therapy drug. The goal of this phase I clinical trial was to test the safety of AG-221 for patients. The trial found that AG-221 is safe and shows real promise as a cancer-fighting treatment for patients whose blood cancers have mutations in the IDH2 gene, as detected by molecular testing.