FDA approves new treatment for refractory multiple myeloma

美国FDA批准Karyopharm公司Xpovio片剂用于联合治疗多发性骨髓瘤

2019-07-04 10:28:00 CISION

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Today, the U.S. Food and Drug Administration granted accelerated approval to Xpovio tablets in combination with the corticosteroid dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody. "While there is no cure for multiple myeloma, there are FDA-approved treatments to target the cancer and slow down the spread of the disease. Sadly, often over time, patients can exhaust all available treatments and still see their disease progress," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "Today we approved a treatment under our accelerated approval program that provides a treatment option for patients with multiple myeloma with no available therapy." Multiple myeloma is cancer that begins in plasma cells (white blood cells that produce antibodies) and may also be referred to as plasma cell myeloma. Abnormal plasma cells build up in the bone marrow, forming tumors in many bones of the body. As more antibodies are made, it can cause blood to thicken and keep the bone marrow from making enough healthy blood cells. The exact causes of multiple myeloma are unknown, but it is more common in older people and African Americans. Efficacy was evaluated in 83 patients with RRMM who were treated with Xpovio in combination with dexamethasone. At the end of the study, the overall response rate was measured at 25.3%. The median time to first response was four weeks, with a range of one to ten weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma. Common side effects of patients taking Xpovio in combination with dexamethasone include a low white blood cell count (leukopenia), a low count of neutrophils, a type of white blood cell (neutropenia), low count of platelets (thrombocytopenia) and low amount of red blood cells (anemia). Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections and low blood sodium levels (hyponatremia). Health care professionals are advised to monitor patients for low blood counts, platelets and sodium levels. Patients should avoid taking Xpovio with other medications that may cause dizziness or confusion and avoid situations where dizziness may be a problem. Health care professionals are advised to optimize the patient's hydration status, blood counts and other medications to avoid dizziness or confusion. The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio because it may cause harm to a developing fetus or newborn baby. Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug's uses and risks. Xpovio in combination with dexamethasone was granted accelerated approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on an endpoint that is reasonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Xpovio's clinical benefit. The FDA granted this application Fast Track designation. Xpovio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Xpovio to Karyopharm Therapeutics.
今天,美国食品药品监督管理局(Food and Drug Administration)批准 Xpovio 片剂与地塞米松皮质激素联合应用于治疗成人复发难治多发性骨髓瘤患者的加速审批,这些患者已经接受了至少四种先前的治疗,并且其疾病对其他几种形式的治疗具有耐药性。包括至少两种蛋白酶体抑制剂、至少两种免疫调节剂和抗 CD38单克隆抗体。 “虽然目前还没有治愈多发性骨髓瘤的方法,但仍有 FDA 批准的针对癌症的治疗方法,以减缓这种疾病的传播。不幸的是,通常随着时间的推移,患者可以用尽所有可用的治疗方法,仍然看到他们的疾病进展,”理查德·帕兹杜尔说,医学博士(M.D.)博士, FDA 肿瘤学卓越中心主任和 FDA 药物评价和研究中心血液和肿瘤产品办公室的代理主任。“今天,我们批准了我们的加速审批程序下的治疗方案,为没有可用治疗的多发性骨髓瘤患者提供治疗选择。” 多发性骨髓瘤是始于血浆细胞(产生抗体的白细胞)的癌症,也可称为浆细胞骨髓瘤。异常的血浆细胞在骨髓中积聚,在身体的许多骨骼中形成肿瘤。当产生更多的抗体时,它会导致血液变厚,使骨髓不能产生足够健康的血液细胞。多发性骨髓瘤的确切病因尚不清楚,但在老年人和非洲裔美国人中更常见。 应用 Xpovio 联合地塞米松治疗 RRMM 83例疗效评价。在研究结束时,总体反应率为25.3%。第一次反应的中位时间为四周,范围为一至十周。平均持续时间为3.8个月。疗效评价由正在进行的多发性骨髓瘤患者随机试验的附加信息支持。 服用 Xpovio 联合地塞米松的患者常见的副作用包括白细胞计数低(白细胞减少)、中性粒细胞计数低、白细胞类型(中性粒细胞减少)、血小板计数低(血小板减少)和红细胞数量低(贫血)。患者还报告呕吐、恶心、疲劳、腹泻、发烧、食欲和体重下降、便秘、上呼吸道感染和低血钠水平(低钠血症)。 卫生保健专业人员建议监测病人的低血计数,血小板和钠水平。患者应避免服用 Xpovio 与其他药物可能导致头晕或混乱,并避免情况下头晕可能是一个问题。卫生保健专业人员建议优化病人的水化状态,血液计数和其他药物,以避免头晕或混乱。FDA 建议卫生保健专业人员告诉生育年龄的女性和有生育潜力的女性伴侣的男性在使用 Xpovio 治疗期间使用有效的避孕方法。怀孕或母乳喂养的妇女不应该服用 Xpovio ,因为它可能会对发育中的胎儿或新生儿造成伤害。Xpovio 必须配有一个病人用药指南,描述关于药物的使用和风险的重要信息。 Xpovio 联合地塞米松获得了加速审批,这使得 FDA 能够根据合理可能预测患者临床受益的终点,批准条件严重的药物以满足未满足的医疗需求。需要进一步的临床试验来验证和描述 Xpovio 的临床疗效。 美国食品和药物管理局批准了这一申请快速跟踪指定。Xpovio 还获得了孤儿药物的指定,这为帮助和鼓励罕见疾病药物的开发提供了激励。FDA 批准 Xpovio 用于 Karyopharm Therapeutics 。

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