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FDA Approves Abbott CDx, Agios Drug for AML Patients With IDH1 Mutation

NEW YORK (GenomeWeb) – The US Food and Drug Administration said today that it has approved a drug developed by Agios Pharmaceuticals for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with IDH1 mutations, as well as companion diagnostic test developed by Abbott Laboratories.

The use of the first-in-its-class drug, called Tibsovo (ivosidenib), is associated with complete remission in some AML patients who have IDH1 mutations, and with a reduction in the need for both red cell and platelet transfusions, according to Richard Pazdur, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.

Tibsovo is an isocitrate dehydrogenase-1 (IDH-1) inhibitor that decreases abnormal production of the oncometabolite 2-hydroxyglutarate (2-HG), leading to differentiation of malignant cells. When mutations in the IDH1 gene are detected by Abbott's RealTime IDH1 Assay in blood or bone marrow samples, patients may be eligible for treatment with Tibsovo, the agency said.

The drug's efficacy was studied in a single-arm trial of 174 adult patients with relapsed or refractory AML with an IDH1 mutation. The trial measured the percentage of patients with no evidence of disease and full recovery of blood counts after treatment, as well as of patients with no evidence of disease and partial recovery of blood counts after treatment. After a median follow-up of 8.3 months, nearly 33 percent of patients experienced a complete remission or complete remission with partial hematologic recovery that lasted a median 8.2 months. Of the 110 patients who required transfusions of blood or platelets due to AML at the start of the study, 37 percent went at least 56 days without requiring a transfusion after treatment with Tibsovo, the FDA added.

"The FDA approval of Tibsovo … is an incredibly exciting milestone for our company and, importantly, for the approximately 6 percent to 10 percent of AML patients with an IDH1 mutation who have been waiting for new treatment options that work radically different than conventional chemotherapy," Agios CEO David Schenkein said in a statement.

Abbott had announced in October 2016 that it had inked deals with both Celgene and Agios to develop and commercialize PCR-based companion diagnostic tests to identify IDH mutations in patients with AML.

Celgene and Agios planned to run the companion diagnostic tests on Abbott's m2000 RealTime System, a PCR instrument designed to enable clinical laboratories to automate the PCR process and analysis of results.

At the time, Agios said it was developing a candidate drug it called AG-120, an IDH1 mutant inhibitor for the treatment of patients with relapsed or refractory AML who have an IDH1 mutation. Celgene was developing enasidenib, an IDH2 mutant inhibitor for the treatment of patients with relapsed or refractory AML who have an IDH2 mutation.