Detailed explanation of the efficacy and role of Enasidenib and how long it usually takes to be effective

Enasidenib (Enasidenib) is an oral small molecule selective targeted inhibitor of the mutant IDH2 enzyme. It was approved by the U.S. Food and Drug Administration in August 2017. FDA (FDA) approved for the treatment of patients with relapsed or refractory (R/R) IDH2 mutant acute myeloid leukemia (AML). Preclinical studies have shown that ensidipine can effectively inhibit the production of highly efficient 2-hydroxyglutarate and alleviate the differentiation block induced by mutant IDH2.

Recurrent mutations affecting cellular metabolism and epigenetic regulation have been implicated in the pathogenesis of acute myeloid leukemia (AML). Twelve percent of patients with AML and 5% of patients with myelodysplastic syndromes have mutations in the isocitrate dehydrogenase 2 (IDH2) gene. IDH2 enzyme participates in the Krebs cycle and catalyzes the production of α-ketoglutarate from isocitrate. The mutated IDH2 enzyme acquires a new form of enzyme activity, which can produce 2-hydroxyglutarate from α-ketoglutarate and inhibit multiple α-ketoglutarate-dependent dioxygenase reactions; leading to abnormal DNA methylation and differentiation block in bone marrow precursors, and ultimately promoting leukemia.

In the initial Phase I/II trial of AG221-001, patients with R/R AML received ensidipine monotherapy with dose escalation to 650 mg daily, with the 100 mg dose level determined to be safe and effective and available for further evaluation. Overall, 113 patients received dose-escalation therapy and 126 patients received dose-expansion therapy. The overall response rate in R/R patients was 40%, including 19% complete responses. At a median follow-up of 7.7 months, median overall survival was 9.3 months, with responders reaching 19.7 months. Although adverse events of clinical concern include indirect hyperbilirubinemia and IDH inhibitor-induced differentiation syndrome, which can be life-threatening if not recognized and treated promptly, ensidipine was well tolerated.

However, ensidipine is not a drug suitable for all patients with leukemia. For example, women who are pregnant or breastfeeding should not take ensidipine because the drug may harm a developing fetus or newborn baby. In addition, drugs should be used under the guidance of a physician and self-medication should be avoided. In summary, ensidipine is a new drug for the treatment of acute myeloid leukemia that can help restore patients' normal hematopoietic ability. However, the specific efficacy and scope of application need to be determined based on the patient's specific situation and the doctor's advice.

Ensiidi plain investigational medicine is not currently on the market in the country, 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, such as those produced by Laos Pharmaceutical Factory.Specifications50mg* The price of 30 tablets per box may be more than 2,000 yuan (the price may fluctuate due to the exchange rate), and its pharmaceutical ingredients are basically the same as those of original drugs sold abroad.