原产地英文商品名：IDHIFA 50mg/tablets 30tablets/bottles
中文参考商品译名：IDHIFA 50毫克/片 30片/瓶
作用机制：Enasidenib是一种异柠檬酸脱氢酶2(IDH2)酶的小分子抑制剂。Enasidenib靶向突变体IDH2 变异体R140Q，R172S，和R172K在体外比野生型酶在浓度较低约40-倍。突变体IDH2酶被enasidenib的抑制作用导致减低2-羟戊二酸[2-hydroxyglutarate(2-HG)]水平和诱导骨髓分化在体外和在体内在IDH2突变AML的小鼠移植物模型，在来自患者有突变的IDH2AML血样品中，enasidenib减低2-HG水平，减低母细胞计数[blast counts]和增加成熟骨髓细胞百分率。
Approved for Relapsed/Refractory AML with IDH2 MutationThe Food and Drug Administration (FDA) has approved Idhifa (enasidenib; Celgene) for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) with a specific genetic mutation. Idhifa is approved for use with the Real Time IDH2 Assay companion diagnostic (Abbott), intended to identify specific mutations in the IDH2 gene in these patients.“AML is a complex, heterogeneous disease, which is particularly difficult to treat in the relapsed or refractory setting,” said Martin Tallman, M.D., Hematologic Oncologist and Chief, Leukemia Service at Memorial Sloan Kettering Cancer Center. “IDH2 mutations inhibit the normal maturation of myeloid cells, so having a treatment that targets this mechanism is promising for patients and encouraging to us as physicians who have it as our goal to provide options for every patient.”Idhifa, an isocitrate dehydrogenase-2 inhibitor, was eva luated in a single-arm trial (n=199) of patients with relapsed or refractory AML who had IDH2 mutations. The study assessed the percentage of patients with complete remission (no evidence of disease and full recovery of blood counts post-treatment) as well as patients with complete remission with partial hematologic recoveryAfter a minimum 6 months of treatment, complete remission was seen in 19% of patients for a median of 8.2 months. Complete remission with partial hematologic recovery was seen in 4% of patients with a median of 9.6 months. Following treatment with Idhifa, 34% of patients who required blood or platelet transfusions at the start of study no longer required them.Nausea, vomiting, increased bilirubin, and reduced appetite were reported as common adverse effects of Idhifa. The drug carries a Boxed Warning regarding the risk of differentiation syndrome, which can be fatal if untreated. Symptoms may include fever, dyspnea, acute respiratory distress, radiographic pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain, peripheral edema, or hepatic/renal or multi-organ dysfunction.Idhifa was previously granted FDA Priority Review and Orphan Drug designations. The product will be available as 50mg and 100mg strength tablets in 30-count bottles.