美国FDA批准Nplate（romiplostim）作为首个并且是唯一的血小板生成药物，用于治疗脾切除及非脾切除的成人免疫性血小板减少症。Nplate为首个FDA批准的肽体蛋白,通过升高与维持血小板数而发挥作用,是长期治疗该慢性疾病的新方法。 慢性ITP是1种严重的自身免疫性疾病，其特征为血液中血小板计数低(血小板减少症)，可能导致严重出血。 本品的批准是基于2项双盲临床研究，纳入患者约125例，这些患者先前至少接受过1次ITP治疗。其中1项研究纳入的患者未进行脾切除，另1项中的患者则无脾。在为期6个月的研究中，接受本品治疗的患者，其血小板计数明显增高，并能维持。未进行脾切除的患者对本品的反应要高于脾切患者。 在临床研究中，与本品相关的严重不良反应有骨髓网硬蛋白沉积、停用后血小板减少症恶化。其他用药风险包括由于血小板过度升高而引起血凝块。另外，如果患者骨髓发育不良而服用本品，可能引起急性白血病。针对本品的治疗风险，已开展“风险评估和减缓策略”(REMS)。 安进公司（Amgen）用于治疗罕见血凝异常疾病的药物——血小板生成素拟肽romiplostim（Nplate）已在欧洲通过批准，这使得该公司抢得先机，比GSK/Ligand两家公司的同类药品Promacta更早进入欧洲市场。 欧盟委员会批准Nplate用于治疗那些对目前常用药无应答的免疫性血小板减少性紫癜（ITP），但它仅获准作为二线药物用于那些未进行脾脏切除，且不适宜接受手术治疗的成人ITP患者。 这次Nplate在欧洲获准基于2项III期临床研究。研究结果证实进行过或未进行过脾脏切除手术的患者用药治疗之后，他们体内的血小板读数均升高83%，并且保持稳定。有医务人员表示，Nplate是第一种在欧洲获准特别针对提高患者体内血小板数量，并且能长期使用的ITP治疗药。 虽然患有ITP的人数量不多，据估计在欧洲有5万例慢性ITP患者，但这种病症会极大地影响他们的生活，而Nplate可以为他们带来福音。 安进公司负责人表示，此次Nplate在欧洲获准是该公司15年来辛勤努力的结果，同时作为第一种获准的肽体类药，其在该公司生物制剂的开发上也具有里程碑意义。2008年8月，本品已在美国获准。 --------------------------------------------------------------- 产地国家: 法国 原产地英文商品名: NPLATE PDR Solution Injection FL 500MCG/ML Vial. 原产地英文药品名: ROMIPLOSTIM 中文参考商品译名: NPLATE PDR 500微克/毫升/瓶 中文参考药品译名: 罗米司亭 生产厂家中文参考译名: 安进公司 生产厂家英文名: Amgen --------------------------------------------------------------- 产地国家: 法国 原产地英文商品名: NPLATE PDR Solution Injection FL 250MCG/ML Vial. 原产地英文药品名: ROMIPLOSTIM 中文参考商品译名: NPLATE PDR 250微克/毫升/瓶 中文参考药品译名: 罗米司亭 生产厂家中文参考译名: 安进公司 生产厂家英文名: Amgen ------------------------------------------------------------------- Important Safety Information What is the most important information I should know about Nplate? Nplate can cause uncommon but serious side effects: Bone marrow changes (increased reticulin and possible bone marrow fibrosis). Long-term use of Nplate® may cause changes in your bone marrow. These changes may lead to abnormal blood cells or your body making less blood cells. The mild form of these bone marrow changes is called "increased reticulin." It is not known if this may progress to a more severe form called "fibrosis." The mild form may cause no problems while the severe form may cause life-threatening blood problems. Signs of bone marrow changes may show up as abnormalities in your blood tests. Your healthcare provider will decide if abnormal blood tests mean that you should have bone marrow tests or if you should stop taking Nplate. Worsening low blood platelet count (thrombocytopenia) and risk of bleeding shortly after stopping Nplate®. When you stop receiving Nplate, your low blood platelet count (thrombocytopenia) may become worse than before you started receiving Nplate. These effects are most likely to happen shortly after stopping Nplate and may last about 2 weeks. The lower platelet counts during this time period may increase your risk of bleeding, especially if you are taking a blood thinner or other medicine that affects platelets. Your healthcare provider will check your blood platelet counts for at least two weeks after you stop taking Nplate. Call your healthcare provider right away to report any bruising or bleeding. High platelet counts and higher chance for blood clots. You have a higher chance of getting a blood clot if your platelet count is too high during treatment with Nplat. You may have severe complications or die from some forms of blood clots, such as clots that spread to the lungs or that cause heart attacks or strokes. Your healthcare provider will check your blood platelet counts and change your dose or stop Nplate if your platelet counts get too high. Worsening of blood cancers. Nplate is not for use in patients with blood cancer or a precancerous condition called myelodysplastic syndrome (MDS). If you have one of these conditions, Nplate may worsen your cancer or condition and may cause you to die sooner. Lack or loss of response: If you do not experience results from Nplate your body may have created cells that are counteractive to Nplate. Your healthcare provider will monitor your platelet counts and test your blood regularly to determine if this is an issue. Your healthcare provider will check your platelet count every week and change your dose of Nplate as needed. This will continue until your healthcare provider decides that your dose of Nplate can stay the same. After that, you will need to have blood tests every month. When you stop receiving Nplate, you will need blood tests for at least 2 weeks to check if your platelet count drops too low. Nplate is only: Prescribed by healthcare providers who are enrolled in the Nplate NEXUS Program. Given to patients who are enrolled in the Nplate NEXUS Program.Given by the enrolled healthcare provider or a provider under their direction. You may not give Nplate® injections to yourself. Nplate is for treatment of certain people with low blood platelet counts caused by chronic ITP, not low platelet counts caused by other conditions or diseases. What are the possible side effects of Nplate? Nplate may cause serious side effects. See "What is the most important information I should know about Nplate?" The most common side effects of Nplate are: Headache Joint pain Dizziness Trouble sleeping Muscle tenderness or weakness Pain in arms and legs Abdominal pain Shoulder pain Indigestion Tingling or numbness in hands and feet These are not all the possible side effects of Nplate. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. For more information, ask your healthcare provider or pharmacist. -------------------------------------------------------- 附件： 201021920423214.pdf