The US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for ivosidenib (Tibsovo®) tablets for the treatment of patients with isocitrate dehydrogenase 1-mutated relapsed or refractory myelodysplastic syndromes (MDS), Servier announced.

Ivosidenib tablets might become the first-in-class targeted therapy for patients with MDS who carry this particular mutation.  

“While the novel use of targeted isocitrate dehydrogenase inhibition has been proven across a number of difficult-to-treat cancers, there continues to be a significant unmet need for patients with MDS within this molecularly defined subset, especially for those who experience disease progression,” said Amir Fathi, MD, program director, Center for Leukemia at Massachusetts General Hospital and associate professor of medicine at Harvard Medical School.

“Today’s filing acceptance provides further support for the potential efficacy and acceptable safety profile of ivosidenib in relapsed or refractory MDS and reinforces the importance of mutational testing in this patient population,” he added.

Read more about MDS experimental therapies

According to the recent findings, 38.9% of the 18 patients treated with the experimental drug have experienced complete remission, and the objective response rate was 83.3%.

The median time to complete remission was 1.87 months (range, 1.0-5.6), yet the median duration of complete remission (range, 1.9-80.8) had not been reached at the time of data cutoff. Moreover, 6 of 9 (66.7%) patients who were transfusion dependent with red blood cells or platelets at baseline became transfusion independent at some point after 56 days postbaseline.

The median overall survival was 35.7 months (range, 3.7-88.7). All treatment-related adverse events reported during the study were aligned with the safety profile of ivosidenib, and no new safety concerns were detected.

Ivosidenib had previously received FDA Breakthrough Therapy designation for the treatment of adults with relapsed or refractory MDS and a susceptible isocitrate dehydrogenase 1 mutation indicated by an FDA-approved test.


Servier announces FDA filing acceptance and priority review for TIBSOVO® (ivosidenib tablets) in the treatment of IDH1-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). News release. Servier; August 15, 2023.