FDA grants Tibsovo (ivosidenib) approval for the treatment of patients with relapsed/refractory acute myeloid leukemia with IDH1 mutation

On 20th July 2018, the U.S. Food and Drug Administration (FDA) granted approval to Tibsovo (ivosidenib) for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an Isocitrate Dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.1 The approval comes after the priority review designation granted to Tibsovo in February 2018, which was reported here.

This approval for Tibsovo, a first in class, oral, selective inhibitor of mutations in IDH1, was based on data from the phase I dose-escalation and expansion AG-120-C-001 study (NCT02074839), which is assessing the safety and efficacy of ivosidenib monotherapy in patients with IDH1 mutated (mIDH1) advanced hematologic malignancies. The results from the cohort of patients with R/R AML were presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting by Daniel Pollyea from the University of Colorado School of Medicine, Aurora, CO and reported by the AML Global Portal here.  

In this study, Tibsovo (500 mg daily dose) was evaluated in 174 patients (median age = 67 years) with mIDH1 R/R AML.  Complete Response (CR) or CR with partial hematologic recovery (CRh) rate was 32.8% with a median duration of 8.2 months and a median overall survival (OS) of 9.0 months. Median OS in responders was 18.8 months. 2

In an interview with the AGP, Courtney DiNardo from the MD Anderson Cancer Center, Houston, TX, commented on the findings of this study in which she stated that ivosidenib monotherapy was “well tolerated” in patients with mutated IDH1 AML. Additionally, in R/R AML patients with unmet medical needs, ivosidenib induced durable responses and improved patient outcomes. She concluded by noting that the findings of this phase I study support the role of ivosidenib as an “effective, oral targeted treatment for patients with mIDH1 AML”.

Tibsovo was approved alongside the Abbott RealTime™ IDH1 companion diagnostic test, which is FDA-approved as an aid in identifying IDH1 mutated AML patients for treatment with Tibsovo.

  1. Agios: FDA Grants Approval of TIBSOVO®, the First Oral, Targeted Therapy for Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and an IDH1 Mutation. 2018 Jul 20. [Accessed 2018 Jul 23].
  2. Pollyea D. A. et al. Ivosidenib (IVO; AG-120) in mutant IDH1 relapsed/refractory acute myeloid leukemia (R/R AML): Results of a phase 1 study. J Clin Oncol 36, 2018 (suppl; abstr #7000). 2018 American Society of Clinical Oncology (ASCO) Annual Meeting, 2018 June 1–5; Chicago, IL, USA.
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