New drug for treating leukemia: Is Enasidenib effective?

Ensidipine (Enasidenib) is the first of its kind, an oral, selective, mutant IDH2 enzyme inhibitor. This first-in-human Phase 1/2 study evaluated the maximum tolerated dose (MTD), pharmacokinetic and pharmacodynamic characteristics, safety and clinical activity of ensidipine in patients with advanced acute myeloid leukemia (AML) with mutant IDH2.

During the dose escalation phase, doses of 50 to 650 mg daily did not reach the maximum tolerated dose. Based on pharmacokinetic and pharmacodynamic characteristics and proven efficacy, ensidipine 100 mg once daily was selected for the expansion phase. Grade 3-4 ensidipinerelated adverse events include indirect hyperbilirubinemia and IDH inhibitor-associated differentiation syndrome. Among patients with relapsed or refractory AML, the overall response rate was 40.3% and the median duration of response was 5.8 months. The response is associated with cellular differentiation and maturation, and there is usually no evidence of aplastic anemia. The median overall survival of relapsed/refractory patients was 9.3 months, 34 patients (19.3%) achieved complete remission, and the overall survival was 19.7 months. In patients who have failed prior AML therapy, daily continuous ensidipine therapy is generally well tolerated and induces hematologic responses.

Encidipine research medicine is not currently on the market in China, so it cannot be included in medical insurance. The price of ensidipine plain medicine when it is launched overseas is very high, and the price of each box may be around 30,000 yuan (the price may fluctuate due to the exchange rate). Currently, there are cheaper generic ensidipine drugs produced by other pharmaceutical companies overseas. For example, the price of Specification50mg*30 tablets produced by a Laos pharmaceutical factory may be more than 2,000 yuan per box (the price may fluctuate due to the exchange rate). Its pharmaceutical ingredients are basically the same as those of the original drugs sold abroad. For more information on overseas drug prices and specific information, please consult Yaode Medical Consultant.