Agios Pharmaceuticals Announces the U.S. FDA Grants Fast Track Designation to AG-221 for Treatment of Patients With Acute Myelogenous Leukemia That Harbor an IDH2 Mutation

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August 13, 2014

Agios Pharmaceuticals, Inc., a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AG-221 for the treatment of patients with acute myelogenous leukemia (AML) that harbor an isocitrate dehydrogenase-2 (IDH2) mutation. AG-221 is a first-in-class, oral, selective, potent IDH2 mutant inhibitor being evaluated in a Phase 1 clinical trial in patients with advanced hematologic malignancies.

“We believe this designation is an important recognition by the FDA of the nonclinical and clinical data reported to date and the potential for AG-221 to address a significant unmet need for patients diagnosed with AML,” said Chris Bowden, M.D., chief medical officer of Agios. “We remain on track to initiate the planned expansion cohorts for patients with IDH2 mutant positive AML and other IDH2 mutant positive hematologic malignancies in the second half of this year. We are committed to working with our partner Celgene Corporation to get this medicine to patients as soon as possible.”

The Fast Track Drug Development Program was established under the FDA Modernization Act of 1997. The program is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application (NDA) for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. Specifically, Fast Track designation facilitates meetings to discuss all aspects of development to support approval. It also provides the opportunity to submit sections of an NDA on a rolling basis as data become available. This permits the FDA to review portions of the NDA as they are received instead of waiting for the entire NDA submission.

AML is a cancer of blood and bone marrow characterized by rapid disease progression, and is the most common acute leukemia affecting adults. 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 or refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. AML prevalence is estimated to be approximately 115,000 to 160,000 patients worldwide, with approximately 20 percent of patients carrying an IDH mutation.

About Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals is focused on discovering and developing novel drugs to treat cancer and rare genetic disorders of metabolism through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class lead product candidates in cancer metabolism and rare genetic disorders of metabolism in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit our website at www.agios.com.

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