FDA Approves First Therapy for Relapsed, Refractory AML with IDH1 Mutation

JULY 20, 2018
Jennifer Barrett, Associate Editor
Officials with the FDA have approved ivosidenib (Tibsovo, Agios), the first targeted treatment for patients with relapsed or refractory acute myeloid leukemia (AML) who have an IDH1 genetic mutation, according to a press release.
The RealTime IDH1 Assay, a companion diagnostic that can be used to detect this mutation, was also granted FDA approval. RealTime IDH1 is manufactured by Abbot Laboratories.
According to the National Cancer Institute at the National Institutes of Health, it is estimated that approximately 19,520 individuals will be diagnosed with AML this year, and approximately 10,670 patients with AML will die of the disease in 2018.
Ivosidenib works by decreasing abnormal production of the oncometabolite 2-hydroxyglutarate (2-HG), leading to differentiation of malignant cells, according to the press release. Patients may be eligible for treatment with ivosidenib if an IDH1 mutation is detected in blood or bone marrow samples using the FDA-approved companion diagnostic.
The approval is based on data from a single-arm clinical trial of 174 adult patients with relapsed or refractory AML who have an IDH1 mutation. According to the data, 23.8% of patients experienced of complete remission or complete remission with partial hematologic recovery that lasted a median 8.2 months. Of the 110 patients who required blood or platelet transfusions due to AML at the start of the study, 37% went at least 56 days without requiring a transfusion after treatment with ivosidenib.
“Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation,” Richard Pazdur, MD, 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, said in the press release. “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.”
Common adverse effects associated with ivosidenib include fatigue, increase in white blood cells, 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. Additionally, the prescribing information includes a boxed warning that an adverse reaction known as differentiation syndrome can occur and can be fatal if not treated.

This article originally appeard on specialtypharmacytimes.com  


FDA approves first targeted treatment for patients with relapsed or refractory acute myeloid leukemia who have a certain genetic mutation [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm614115.htm?utm_campaign=07202018_PR_FDA%20approves%20new%20AML%20drug&utm_medium=email&utm_source=Eloqua. Accessed July 20, 2018.