2017年8月3日,美国食品和药品监管局今天批准Vyxeos为治疗成年有两种类型急性髓性白血病(AML)；新诊断的治疗相关AML(t-AML)或AML与骨髓增生异常有关的变化(AML-MRC). Vyxeos是一种化疗药物柔红霉素[daunorubicin]和阿糖胞苷[cytarabine]的固定-组合。
FDA的药品评价和研究中心中血液学和肿瘤产品代理主任FDA的卓越的肿瘤中心主任Richard Pazdur，M.D.说：“这是第一个被批准的治疗特异性地为患者高风险AML某些类型,” “Vyxeos组合两种常用化疗至一个单个制剂可能帮助有些患者活得更长如果他们是分开地接受两种治疗。”
AML是在骨髓中形成的一种迅速地进展癌症和导致在血流中白血细胞数量增加。在美国卫生院的国立癌症研究所估算在今年约21,380人将被诊断有AML；在2017约10,590有AML患者将死于该疾病。T-AML出现作为一种化疗或辐射的合并症在约8至10%的所有被治疗患者对癌症在治疗后平均五年内。AML-MRC的特征是某些血液疾病史和癌症细胞内其他显著突变。Patients有t-AML或AML-MRC患者有非常低生命期望。
在309有新诊断t-AML或AML-MRC 患者被研究Vyxeos的安全性和疗效患者被随机化接受Vyxeos或分开地给予柔红霉素和阿糖胞苷的治疗。试验测量患者从他们开始试验日期活得如何长(总体生存)。接受Vyxeos患者活得比分开地接受柔红霉素和阿糖胞苷治疗患者较长(中位总体活存9.56月相比5.95月)。
Vyxeos的常见副作用包括包括出血事件，发热与低白血，组织肿胀，恶心，黏膜炎，腹泻，便秘，肌肉骨骼疼痛，疲乏，腹痛，气短呼吸困难，头痛，咳嗽，食欲减低，心律不齐，肺炎，菌血症，发冷，睡眠障碍和呕吐。
对柔红霉素，阿糖胞苷或制剂任何组分严重超敏性患者史不应使用Vyxeos。患者用Vyxeos 应被监视对超敏性反应和减低心脏功能。Vyxeos曽被伴随有严重或致命性出血事件。柔红霉素曽被伴随有严重损伤其中药物从静脉输注渗漏至皮肤和皮下组织。妊娠或哺乳喂养妇女不应用，因为它可能致危害至一位发育胎儿或一个新生婴儿。
对Vyxeos处方资料包括一个黑框警告不应互换Vyxeos 与其他含柔红霉素- 和/或阿糖胞苷产品。
FDA授权这个申请优先审评和突破性治疗指定。Vyxeos还接受孤儿药物指定，它提供激励帮助和鼓励对罕见疾病药物开发。.
FDA授权Vyxeos的批准给予Jazz Pharmaceuticals
Jazz announced that the Food and Drug Administration (FDA) has approved Vyxeos (daunorubicin and cytarabine) liposome for injection to treat adults with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).
The approval was supported by data from a study in 309 patients (60–75 years of age) with newly diagnosed t-AML or AML-MRC who were randomized to either Vyxeos or cytarabine and daunorubicin separately. The data showed patients treated with Vyxeos had longer overall survival vs. patients who received separate treatments of cytarabine and daunorubicin (median 9.56 months vs. 5.95 months).
Hemorrhage, febrile neutropenia, rash, edema, nausea, mucositis, diarrhea, among others, were reported as common adverse effects of Vyxeos. The drug carries a Boxed Warning stating it is not interchangeable with other daunorubicin- or cytarabine-containing products.
Vyxeos utilizes the proprietary CombiPlex technology, which uses liposomes to control the release and distribution of amphipathic drugs, and nanoparticles to control the release and distribution of hydrophobic drugs. The resulting liposomal formulation provides a fixed-ratio of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, to the bone marrow. Both in vitro and animal models have demonstrated their synergistic effects at killing leukemia cells.
"Vyxeos is the first chemotherapy to demonstrate an overall survival advantage over the standard of care in a Phase 3 randomized study of older adults with newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes," said Jeffrey E. Lancet, MD, from the Moffitt Cancer Center.
Vyxeos injection will be available as a 44mg/100mg strength lyophilized cake in single-dose vials in 2- and 5-count cartons. It was previously granted Priority Review status, as well as Breakthrough Therapy, Orphan Drug, and Fast Track designations by the FDA. Vyxeos is anticipated to be available within one week.