Therapeutic efficacy of Ivosidenib

A pivotal Phase 1 open-label study enrolled 18 patients with relapsed or refractory myelodysplastic syndromes (R/R MDS) harboring IDH1 mutations. The results demonstrated that among patients treated with Ivosidenib, the complete response (CR) rate was 38.9%, and the objective response rate (ORR) reached 83.3%. In addition, the median time to achieve complete response was 1.9 months (range: 1.0–5.6 months). As of the data cutoff date, the median duration of complete response had not been reached (range: 1.9–80.8+ months), and the median overall survival was 35.7 months (range: 3.7–88.7 months).

Furthermore, among the 9 patients who were dependent on red blood cell or platelet transfusions at baseline, 66.7% (n=6) achieved transfusion independence during any observation period of ≥56 days after treatment. Overall, treatment-related adverse events were consistent with the known safety profile of Ivosidenib.

from FDA，2023.10