MDS Study of Ivosidenib
Agios is investigating TIBSOVO® (ivosidenib tablets) in patients with IDH1-mutated relapsed or refractory myelodysplastic syndrome (R/R MDS) as a sub-study of the Phase 1 trial of TIBSOVO® in patients with advanced hematologic malignancies (NCT02074839).
In December 2019, TIBSOVO® was granted FDA Breakthrough Therapy Designation for the treatment of adult patients with R/R MDS with an IDH1 mutation. Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is under investigation to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints. The designation was based on data from the MDS arm of the Phase 1 study of TIBSOVO® in patients with advanced hematologic malignancies. Data from this cohort of patients was most recently presented at EHA 2020 (DiNardo C.D, European Hematology Association; 2020. Abstract #EP826).
The MDS sub-study will further evaluate the safety, tolerability and clinical activity of TIBSOVO® in adult patients with IDH1-mutant R/R MDS.
The effectiveness and safety of ivosidenib in R/R MDS have not been established. There is no guarantee that ivosidenib will receive health authority approval or become commercially available in any country for the use being investigated.