All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.
The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy
Mutation testing in AML:
What you need to know
with Charles Craddock, Ralph Hills, and Gail Roboz
Wednesday, April 23, 2025
17:30-18:30 BST
This independent educational activity is supported by Thermo Fisher Scientific. All content is developed independently by the faculty. The funder is allowed no influence on the content.
The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.
The AML Hub is an independent medical education platform, sponsored by Astellas, Daiichi Sankyo, Johnson & Johnson, Kura Oncology and Syndax, and has been supported through educational grants from Bristol Myers Squibb and the Hippocrate Conference Institute, an association of the Servier Group. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.
Withdrawal of European Marketing Association application for ivosidenib for patients with R/R IDH1-mutated AML
By Sumayya Khan
Bookmark this article
On October 16, 2020, the European Marketing Association application for ivosidenib for adult patients with relapsed/refractory (R/R) IDH1-mutated acute myeloid leukemia (AML) was withdrawn. This decision was based on the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) assessment that the data from the single-arm, uncontrolled phase I study in the R/R AML setting (NCT02074839) did not sufficiently support a positive benefit–risk balance.1
Complete remission or complete remission with partial hematologic recovery was 30.4%, with a median duration of response of 8.2 months. The overall response rate was 41.6%, with a median duration of response of 6.5 months. Treatment-related adverse events of Grade ≥ 3 included prolongation of the QT interval (7.8%), IDH differentiation syndrome (3.9%), anemia (2.2%), thrombocytopenia (3.4%), and leukocytosis (1.7%).2
Find out more about this trial and the FDA approval here. The phase III studies of ivosidenib treatment for newly diagnosed AML, AGILE (NCT03173248) and HOVON150/AMLSG29 (NCT03839771), remain ongoing.1
- Agios. Agios announces withdrawal of European Marketing Authorization application for TIBSOVO® as a treatment for relapsed or refractory IDH1-mutant acute myeloid leukemia. https://investor.agios.com/news-releases/news-release-details/agios-announces-withdrawal-european-marketing-authorization. Published Oct 16, 2020. Accessed Oct 19, 2020.
- DiNardio CD, Stein EM, de Botton S, et al. Durable remissions with ivosidenib in IDH1-mutated relapsed or refractory AML. N Engl J Med. 2018;378(25):2386-2398. DOI: 1056/NEJMoa1716984.
Your opinion matters
3 votes - 17 days left ...
Newsletter
Subscribe to get the best content related to AML delivered to your inbox