2023年ITP新药新消息.doc

ITP新药新消息 　 ITP新药新消息——奥赛康申报一罕见病药物 该品国内市场尚属空白 国家食药监总局〔CFDA〕网站显示，奥赛康申报临床的3.1类造血新药艾曲泊帕原料药和片剂获受理。 　目前，艾曲泊帕原研药尚未进入中国市场，也无国内企业生产该产品。 　根据临床研究结果，艾曲泊帕片可刺激巨核细胞的增殖与分化，用于治疗血小板减少性紫癜〔ITP〕，该产品最早由英国葛兰素史克公司研发，并于2023年5月获FDA指定为治疗罕见病的药物，在当年11月获得加速批准用来治疗慢性ITP。资料显示，我国治疗血小板减少性紫癜药品种类有限，主要有羟基脲片、复方皂矾丸、血康胶囊等产品，艾曲泊帕片的国内市场尚属空白。目前申报该艾曲泊帕片的国内企业除了奥赛康外，仅有南京华威医药，该公司于今年3月申报临床。奥赛康主要从事消化类、抗肿瘤类及其他药品的生产和销售，拳头产品为消化药“奥西康〞〔注射用奥美拉唑钠〕。奥西康占公司营业收入和毛利的比例均超过50%。———————————————————————————————————————英文药名：PROMACTA〔Eltrombopag〕中文药名:艾曲泊帕片 艾曲波帕生产商：Ligand制药公司和葛兰素史克药品介绍通用名为Eltrombopag的Promacta〔SB-497115〕是口服的小分子血小板生成素受体冲动剂，由美国Ligand制药公司和英国制药巨头葛兰素史克共同开发，并于2023年被美国食品药品监管局批准上市，用于治疗经糖皮质激素类药物、免疫球蛋白治疗无效或脾切除术后慢性特发性血小板减少性紫癜(ITP)患者的血小板减少。在美国和欧洲大约有140,000该类患者。ITP是一种罕见的血液疾病，表现为血小板损害或血小板数量缺乏，使患者出现淤伤或出血的风险增加。Eltrombopag可与人体跨膜区的血小板生成素受体作用，产生信号放大，从而诱导骨髓巨核细胞的增殖和分化。该药主要用于对糖皮质激素类、球蛋白药物或接受脾切除术后效果不理想的慢性血小板减少性紫癜患者。Promacta在2023年的临床实验中对治疗丙型肝炎血液相关的并发症显示良好疗效。PROMACTAGeneric Name for PROMACTAEltrombopag (as olamine) 25mg, 50mg; tabs.Legal Classification:RxPharmacological Class for PROMACTAThrombopoietin receptor agonist.Manufacturer of PROMACTAGlaxoSmithKline PharmaceuticalsIndications for PROMACTAThrombocytopenia due to chronic immune (idiopathic) thrombocytopenic purpura (ITP) in adults who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.Adult dose for PROMACTATake on empty stomach. Initially 50mg once daily. Moderate to severe hepatic impairment or East Asian ancestry: initially 25mg once daily. Titrate to maintain platelet count ≥50x109/L; max 75mg once daily. Adjust dose based on platelet count: see literature.Children's dosing for PROMACTANot recommended.Warnings/Precautions for PROMACTAMonitor CBC, platelet count, and peripheral blood smears for cytopenias and abnormal morphologies; discontinue if no increase in platelet count occurs after 4 weeks at max dose, or if excessive increase in platelet count occurs (eg, >400x109/L), or if evidence of bone marrow fibrosis occurs (eg, cytopenias, nucleated RBCs). Monitor liver function closely before, during, and after treatment (see literature); discontinue if ALT >3xULN and is progressive or persistent for ≥4 weeks, or if it occurs with evidence of hepatic injury; reinitiation of therapy: not recommended; if restarted, use lower dose and monitor carefully. Do baseline eye exam; monitor for cataracts. Thromboembolism risk factors. Myelodysplastic syndromes. Renal impairment. Pregnancy (Cat.C). Nursing mothers: not recommended.Interactions for PROMACTADo not take within 4 hours of food/drugs containing polyvalent cations (eg, Fe+2, Ca+2, Al+3, Mg+2, Se+2, Zn+2). May potentiate substrates of organic anion transporter polypeptide 1B1 (eg, benzylpenicillin, most statins, methotrexate, nateglinide, repaglinide, rifampin); monitor and consider reducing their doses. May be potentiated by strong inhibitors of CYP1A2 (eg, ciprofloxacin, fluvoxamine) or CYP2C8 (eg, gemfibrozil, trimethoprim), and with moderate or strong inhibitors of UGT1A1 or UGT1A3.Adverse Reactions for PROMACTANausea, vomiting, menorrhagia, myalgia, paresthesia, cataract, ecchymosis, thrombocytopenia, increased ALT/AST, conjunctival hemorrhage, increased risk of hematologic malignancies; thrombotic events with excessive increases in platelet counts; worsened thrombocytopenia after discontinuation.Notes for PROMACTANote: Physicians, pharmacies, and patients must enroll in Promacta Cares program. Register pregnant patients taking eltrombopag by calling (888) 825-5249.How is PROMACTA suppliedTabs—30Related Disease:Thrombocytopenia PROMACTA is indicated for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.Limitations of use:PROMACTA should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increases the risk for bleeding. PROMACTA should not be used in an attempt to normalize platelet counts.Important Safety InformationBOXED WARNINGPROMACTA may cause hepatotoxicity. Patients receiving therapy with PROMACTA must have regular monitoring of serum liver tests (see Laboratory Monitoring). Discontinue PROMACTA if ALT levels increase to ≥3X upper limit of normal (ULN) and are: progressive; or persistent for ≥4 weeks; or accompanied by increased direct bilirubin; or accompanied by clinical symptoms of liver injury or evidence of hepatic decompensation. Reinitiating treatment with PROMACTA is not recommended and should be considered only with close medical supervision and under exceptional circumstances where the potential benefit outweighs the risk.Additional Safety Information Regarding Risk of Hepatotoxicity:Reinitiating treatment with PROMACTA is not recommended. If the potential benefit for reinitiating treatment with PROMACTA is considered to outweigh the risk for hepatotoxicity, then cautiously reintroduce PROMACTA and measu