European Medicines Agency, London, United Kingdom
Medicines and Healthcare Products Regulatory Agency Licensing, London, United Kingdom.
Oncologist. 2019 May;24(5):e171-e179. doi: 10.1634/theoncologist.2019-0025. Epub 2019 Mar 21.
On February 22, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product gemtuzumab ozogamicin (Mylotarg; Pfizer, New York City, NY), intended for the treatment of acute myeloid leukemia. Mylotarg was designated as an orphan medicinal product on October 18, 2000. The applicant for this medicinal product was Pfizer Limited (marketing authorization now held by Pfizer Europe MA EEIG).The demonstrated benefit with Mylotarg is improvement in event-free survival. This has been shown in the pivotal ALFA-0701 (MF-3) study. In addition, an individual patient data meta-analysis from five randomized controlled trials (3,325 patients) showed that the addition of Mylotarg significantly reduced the risk of relapse (odds ratio [OR] 0.81; 95% CI: 0.73-0.90; = .0001), and improved overall survival at 5 years (OR 0.90; 95% CI: 0.82-0.98; = .01) [Lancet Oncol 2014;15:986-996]. The most common (>30%) side effects of Mylotarg when used together with daunorubicin and cytarabine are hemorrhage and infection.The full indication is as follows: "Mylotarg is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33-positive acute myeloid leukemia (AML), except acute promyelocytic leukemia (APL)."The objective of this article is to summarize the scientific review done by the CHMP of the application leading to regulatory approval in the European Union. The full scientific assessment report and product information, including the Summary of Product Characteristics, are available on the European Medicines Agency website (www.ema.europa.eu). IMPLICATIONS FOR PRACTICE: This article reflects the scientific assessment of Mylotarg (gemtuzumab ozogamicin; Pfizer, New York City, NY) use for the treatment of acute myeloid leukemia based on important contributions from the rapporteur and co-rapporteur assessment teams, Committee for Medicinal Products for Human Use members, and additional experts following the application for a marketing authorization from the company. It's a unique opportunity to look at the data from a regulatory point of view and the importance of assessing the benefit-risk.
2018 年 2 月 22 日，人用药品委员会（CHMP）采纳了一项积极意见，建议授予吉妥珠单抗奥佐米星（商品名：Mylotarg；辉瑞，纽约）上市许可，用于治疗急性髓细胞白血病。Mylotarg 于 2000 年 10 月 18 日被指定为孤儿药。该药品的申请人是辉瑞有限公司（现由辉瑞欧洲 MA EEIG 持有上市许可）。Mylotarg 的临床获益为无事件生存的改善。这在关键性 ALFA-0701（MF-3）研究中得到了证实。此外，来自五项随机对照试验（3325 例患者）的个体患者数据汇总分析显示，Mylotarg 的加入显著降低了复发风险（比值比[OR]0.81；95%置信区间[CI]：0.73-0.90；=0.0001），并提高了 5 年总生存率（OR 0.90；95%CI：0.82-0.98；=0.01）[柳叶刀肿瘤学 2014；15：986-996]。当与柔红霉素和阿糖胞苷联合使用时，Mylotarg 的最常见（>30%）副作用是出血和感染。完整的适应证如下：“Mylotarg 联合柔红霉素（DNR）和阿糖胞苷（AraC）用于治疗年龄在 15 岁及以上、未经治疗、初诊 CD33 阳性急性髓细胞白血病（AML）的患者，不包括急性早幼粒细胞白血病（APL）。”本文的目的是总结人用药品委员会对导致欧盟监管批准的申请进行的科学审查。完整的科学评估报告和产品信息，包括产品特性摘要，可在欧洲药品管理局网站（www.ema.europa.eu）上查阅。对实践的意义：本文反映了辉瑞公司（纽约辉瑞公司）对 Mylotarg（吉妥珠单抗奥佐米星）用于治疗急性髓细胞白血病的科学评估，该评估基于报告员和共同报告员评估团队、人用药品委员会成员以及公司申请上市许可后其他专家的重要贡献。这是从监管角度看待数据和评估获益-风险的重要机会。
