FDA accepts New Drug Application and grants Priority Review status to ivosidenib for the treatment of patients with IDH1 mutated R/R AML

On 15 February 2018, the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for ivosidenib (AG-120), an Isocitrate Dehydrogenase 1 (IDH1) inhibitor, for the treatment of patients with Relapsed or Refractory (R/R)  Acute Myeloid Leukemia (AML) with an IDH1 mutation. In addition to the NDA acceptance, ivosidenib was also granted priority review status.1

The NDA submission 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 in patients with IDH1 mutated (mIDH1) advanced hematologic malignancies. The results of this study were presented at the 2017 American Society of Hematology (ASH) Annual Meeting by Courtney D. DiNardo, from The University Texas MD Anderson Cancer Center, Houston, TX, and reported by the AML Global Portal here. Treatment of mIDH1 R/R AML patients (n =125) with 500 mg daily dose of ivosidenib lead to a Complete Response (CR) or CR with partial hematologic recovery (CRh) rate of 30.4% with a median duration of 8.2 months and a median overall survival of 14.8 months. 2

In an interview with the AGP, Courtney DiNardo 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”.

Based on the Prescription Drug User Fee Act (PDUFA), the FDA would make a decision on the NDA priority review application for ivosidenib on 21 August 2018.1

  1. GlobeNewswire: FDA Accepts New Drug Application and Grants Priority Review for Ivosidenib in Relapsed or Refractory AML with an IDH1 Mutation. 2018 Feb 15. [Accessed 2018 Feb 15].
  2. DiNardo Cet al. Ivosidenib (AG-120) in Mutant IDH1 AML and Advanced Hematologic Malignancies: Results of a Phase 1 Dose Escalation and Expansion Study. Oral Abstract #725: 59th ASH Annual Meeting and Exposition, Atlanta, GA.