Celgene公司用于治疗由骨髓异常综合症（MDS）导致的贫血药物Lenalidomide通过了FDA的批准。Celgene公司计划将该药商品名定为Revlimid。
MDS患者体内的骨髓未产生足够的正常血细胞时，会导致身体多方面出现异常。MDS患者可以通过输血或输注血小板进行治疗，同时使用抗生素以防感染。在临床试验中，患者使用Revlimid进行平均44周的治疗之后，不再需要输血或输注血小板。
该药与会导致胎儿畸形的Thalidomide相似。FDA称，这种药在销售时应在标签中应加入警示信息，女性患者在使用这种药之前应进行怀孕检测并征得医生同意，开这种药的医生应进行相应登记。目前，相关人员正在通过动物试验确定怀孕期间服用Revlimid是否会导致胎儿畸形。
Celgene公司近日宣布，revlimid（曾用名：revimid）已被欧洲药品评审局(EMEA)定义为罕用药。Revlimid是新型的免疫调节剂，适应证范围广泛，包括多发性骨髓瘤、恶性血液疾病（譬如：骨髓增生异常综合征和转移性黑色素瘤）。
Revlimid作为罕用药享有EMEA授予的优惠措施。一旦revlimid获批上市，将在多发性骨髓瘤治疗领域享有10年市场独占期。EMEA将为revlimid在欧洲的集中申请程序提供便利，减少申请费用。
多发性骨髓瘤在常见的血癌中排名第二位，全球影响到15万人，每年新增患者大约7.4万。2002年5.7万人死于该病。2003年，revlimid被FDA认定为罕用药，进入快速审批通道，用于治疗骨髓瘤和骨髓增生异常综合征。骨髓增生异常综合征是一种血细胞生成障碍性疾病，最终将导致急性白血病，全球大约30万人患有该病。
Revlimid的Ⅱ期临床纳入70例患者。患者平均年龄为62岁，有23例已经接受骨髓干细胞移植，23例接受了沙利度胺治疗。研究中有57例患者参加了药物毒性评估，46例患者参加了对疗效的评估。
FDA Approves New Treatment for Myelodysplastic Syndrome (MDS)
The Food and Drug Administration (FDA) has approved the drug Revlimid (lenalidomide) for the treatment of patients with a subtype of Myelodysplastic Syndrome (MDS).
The subtype is MDS patients with deletion 5q cytogenetic abnormality.
MDS is a collection of disorders in which the bone marrow does not function normally and the body does not make enough normal blood cells. Patients with MDS may need blood and platelet transfusions and antibiotic therapy for infections. In clinical trials, patients treated with Revlimid no longer needed transfusions, with most patients becoming independent of transfusion by three months.
The transfusion-free period lasted for an average of 44 weeks.
"This new product will offer a much needed treatment option for patients suffering from this rare illness that, in some cases, has been found to progress to fatal forms of leukemia," said Dr.
Steven Galson, M.D., Director of FDA''s Center for Drug eva luation and Research (CDER).
MDS can develop following treatment with drugs or radiation therapy for other diseases, or it can develop without any known cause. Some forms of MDS can progress to acute myeloid leukemia (AML), a type of cancer in which too many white blood cells are made.
An estimated 7,000 to12,000 new cases of MDS are diagnosed yearly in the United States. Although MDS occurs in all age groups, the highest preva lence is in people over 60 years of age.
Typical symptoms include weakness, fatigue, infections, easy bruising, bleeding, and feverRevlimid is structurally similarto thalidomide, a drug known to cause severe birth defects. Additional studies are ongoing in animals to address whether there is a risk that Revlimid will also cause birth defects when taken during pregnancy.
While these studies are under way, the company is marketing Revlimid under a risk management plan called RevAssist, designed to prevent fetal exposure.
Under RevAssist, only pharmacists and prescribers registered with the program will prescribe and dispense Revlimid.
The program requires patients, including female patients undergoing mandatory pregnancy testing, to give informed consent before starting Revlimid.
Physicians are to check pregnancy tests, limit prescriptions to a one-month mail supply, and report any pregnancies to FDA.
FDA and the manufacturer will re-eva luate the risk management plan when results of further animal testing for birth defects are completed.
The labeling for Revlimid will include a Black Box Warning and a Medication Guide regarding the prevention of fetal exposure.
Additional Black Box Warnings include the potential need to lower the dose due to suppressed blood counts and increased risk of blood clots. Common side effects reported with Revlimid include thrombocytopenia (low platelet count), neutropenia (low white blood cell count), diarrhea, pruritis (itch), rash, and fatigue.
Revlimid is distributed by Celgene Corporation of Summit, N.J. |
