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FDA Approves First IDH1 Inhibitor for AML Treatment
The US Food and Drug Administration (FDA) has approved a first-in-class drug for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) who have a specific mutation.
Ivosidenib (Tibsovo; Agios Pharmaceuticals) is the first IDH1 inhibitor approved for the treatment of patients with AML who have the IDH1 mutation.
The FDA approved ivosidenib for use along with a companion diagnostic—the RealTime IDH1 Assay—which was approved by the agency on the same day and is specifically designed to detect mutations in the IDH1 gene in patients with AML. If the RealTime IDH1 Assay detects the IDH1 mutation in the patient’s blood or bone marrow, that patient may be eligible for treatment with ivosidenib.
This approval was based on results from a single-arm clinical trial of 174 adults with relapsed or refractory AML and IDH1 mutation. At a median follow-up of 8.3 months, complete remissions with or without partial hematologic recovery were reported in 32.8% of patients. Of the 110 patients who required blood or platelet transfusions at the start of the study, 37% did not require a transfusion for at least 56 days after receiving ivosidenib.
Common side effects seen with ivosidenib include fatigue, increased white blood cell count, joint pain, diarrhea, shortness of breath, swelling in the arms or legs, nausea, pain or sores in the mouth or throat, irregular heartbeat, rash, fever, cough, and constipation.
“Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation. The use of Tibsovo is associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, in a press release (July 20, 2018).—Janelle Bradley
