FDA Approves Tibsovo for Individuals Newly Diagnosed With IDH1-mutated AML


The approval is supported by data from a global phase 3 trial assessing the use of the drug for individuals with previously untreated IDH1-mutated acute myeloid leukemia.

The FDA announced the approval of ivosidenib (Tibsovo, Servier) in combination with azacytidine for the treatment of individuals with newly diagnosed IDH1-mutated acute myeloid leukemia (AML) who are 75 years or older or who have comorbidities that preclude the use of intensive induction of chemotherapy. Ivosidenib is the first therapy that targets cancer metabolism approved in combination with azacytidine, and the AGILE trial was the only phase 3 trial designed for newly diagnosed individuals with IDH1-mutated AML who are ineligible for intensive chemotherapy.

"Today's approval builds on the established body of evidence for [ivosidenib], which is now approved across multiple IDH1-mutated cancer types," said David K. Lee, chief executive officer, Servier Pharmaceuticals, in a press release. "As a leader in oncology pioneering the science behind targeted IDH inhibition, we are proud to bring a new therapeutic option to the acute myeloid leukemia community and remain committed to pushing the boundaries of healthcare innovation in oncology and beyond."

The supplemental new drug application for ivosidenib received priority review and was reviewed by the FDA under the Real-Time Oncology Review pilot program. The FDA’s approval was assessed using data from the AGILE study, a global phase 3 trial of the drug in individuals with previously untreated IDH1-mutated AML.

The results of the AGILE study showed ivosidenib demonstrated a statistically significant improvement in event-free survival and in overall survival (OS). Additionally, the treatment combination results in a 3-fold improvement in median OS of 24 month compared to the placebo and azacytidine at 7.9 months as a first-line treatment for IDH1-mutated AML.

Furthermore, the combination demonstrated a safety profile consistent with the previously established safety profile in a prior study. The common adverse events experienced by the patient population in the AGILE study were nausea, vomiting, electrocardiogram QT prolonged, insomnia, differentiation syndrome, leukocytosis, hematoma, hypertension, arthralgia, dyspnea, and headache.

During the study, the investigators observed select laboratory abnormalities, including a decrease in leukocytes, platelets, hemoglobin, neutrophils, phosphate, aspartate, and magnesium, while they also observed an increase in lymphocytes, glucose, aminotransferase, alkaline phosphatase, and potassium.

"In addition to a favorable safety profile, TIBSOVO is the first therapy targeting cancer metabolism to demonstrate an impressive, significant benefit in event-free survival and overall survival in combination with azacitidine, underscoring its importance as part of a new combination regimen for patients with newly diagnosed IDH1-mutated acute myeloid leukemia who are not candidates for intensive induction chemotherapy,” said Eytan M. Stein, MD, director of Program for Drug Development in Leukemia for Leukemia Service in the Department of Medicine at Memorial Sloan Kettering Cancer Center, in the press release.

At the 2021 American Society of Hematology Annual Meeting and Exposition, the investigators presented their findings from the AGILE study and then subsequently published them in the New England Journal of Medicine. Based on their findings, the investigators wrote that the recommended dosage of ivosidenib for individuals who are newly diagnosed with IDH1-mutated AML is 500 mg once daily via oral administration.

Ivosidenib is also approved in the United States as a monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML.


Servier announces FDA approval of Tibsovo (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. PR Newswire. News release. May 25, 2022. Accessed May 26, 2022. Email.

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