CStone Pharmaceuticals announced that the National Medical Products Administration (NMPA) of China has approved the new drug application (NDA) of TIBSOVO® (ivosidenib tablets) for the treatment of adult patients with relapsed/refractory acute myeloid leukemia (R/R AML) who have a susceptible IDH1 mutation, providing a new precision therapy for this patient population. Acute myeloid leukemia (AML) is the most common type of leukemia in adults. The disease progresses rapidly, and the vast majority of patients are the elderly.

In the US, there are about 20,000 new cases of AML each year, and the 5-year survival rate is about 29%. With the aging of the population, the incidence of AML in China has been rising, and particularly the elderly and relapsed/refractory patients have a poorer prognosis. In China, there are about 75.3 thousand new cases of Leukemia each year and approximately 59% are AML patients, among whom about 6-10% have IDH1 mutations.

In China, TIBSOVO® was selected in the list of the third batch of Overseas New Drugs Urgently Needed in Clinical Settings by the Center for Drug Evaluation, NMPA in China, and granted fast-track designation. As a potent and highly selective first-in-class oral IDH1 inhibitor, TIBSOVO® was also recommended by the 2020 edition of the CSCO Guidelines for Diagnosis and Treatment of Hematological Malignancies due to its proven clinical advantages. The approval of TIBSOVO® was based on the China registrational bridging study CS3010-101, which aimed to evaluate the pharmacokinetic (PK), pharmacodynamics (PD), safety, and clinical efficacy of orally administered TIBSOVO® in Chinese adult patients with R/R AML who have a susceptible IDH1 mutation.

TIBSOVO® demonstrated robust clinical efficacy and a well-tolerated, manageable safety profile in the treatment of Chinese adults with R/R AML with a susceptible IDH1 mutation. Among 30 evaluable patients, the primary efficacy endpoint of complete remission plus complete remission with partial hematologic recovery (CR+CRh) rate was 36.7% (11/30) with all 11 patients achieving CR. The median duration of CR+CRh is not reached, and the estimated 12-month CR+CRh duration rate is 90.9%.

The data were presented in a proffered paper presentation at the 2021 European Society for Medical Oncology (ESMO) Congress.