Ivosidenib Receives FDA Approval for New Indication in Relapsed or Refractory IDH1-Mutant Myelodysplastic Syndromes

On October 24, the FDA's official website showed that Servier's ivosidenib tablets (brand name: Tibsovo) were approved for the treatment of IDH1-mutated relapsed or refractory myelodysplastic syndromes (R/R MDS). Thus, ivosidenib has become the first targeted drug approved for the treatment of IDH1-mutated MDS. This is also the fourth indication for ivosidenib to be approved in the United States.

The FDA approval was primarily based on positive results from an open-label, dose-escalation, dose-expansion Phase I study (AG120-C-001). The study included 18 R/R MDS patients with IDH1 mutations and evaluated the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of ivosidenib (500mg, once daily) in this patient population.

The results showed that the proportion of patients achieving complete remission (CR) was 38.9%, and the proportion of patients achieving objective remission was 83.3%, with a median overall survival (OS) of 35.7 months. Among nine transfusion-dependent patients, six did not require transfusions for at least 56 days after receiving ivosidenib treatment. The safety profile of ivosidenib was consistent with previous studies.

Ivosidenib is a small molecule inhibitor targeting IDH1 mutations developed by Agios Pharmaceuticals. Later, its rights were acquired by Servier and CStone Pharmaceuticals. CStone Pharmaceuticals owns the rights to ivosidenib in China, while Servier holds all rights to the product outside of China.

It is estimated that 16,000 patients are diagnosed with MDS annually in the United States. Approximately 3.6% of MDS patients carry IDH1 mutations, which are considered early drivers of MDS. MDS patients with IDH1 mutations typically have a poor prognosis and an increased risk of progression to acute myeloid leukemia (AML) at later stages.

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