2018年4月17日，Rigel制药宣布，美国FDA已批准TAVALISSE(fostamatinib disodium hexahydrate)用于对之前治疗缓解不佳的成年慢性免疫性血小板减少症（ITP）患者血小板减少的治疗。
  TAVALISSE是一种口服的脾脏酪氨酸激酶(SYK)抑制剂，通过阻止血小板的破坏来应对疾病的潜在自身免疫原因，为成年慢性ITP患者提供了一个重要的新的治疗方案。
  康奈尔医学院儿科教授、FIT 3期试验的主要研究人员、Rigel制药咨询委员会的顾问和付费成员James Bussel博士表示：“慢性ITP的治疗是有挑战性的，因为疾病的异质性使得很难预测个体患者如何对可用的治疗作出反应，并不是所有的患者都能找到治疗效果良好的治疗方法。TAVALISSE获得FDA的批准让临床医生拥有了一种具有新作用机制的治疗药物。”
  TAVALISSE的批准依赖于FIT临床研究项目的数据，FIT包含两项随机安慰剂对照的临床3期研究047和048、一项开标扩展试验049以及最初的概念验证试验。新药上市申请包括了163例ITP患者的数据，并得到了一项安全数据集的支持，该数据集包含4600多名涉及其它适应症的已接受TAVALISSE评估的受试者。
  血小板紊乱支持协会执行主任Caroline Kruse表示：“患有慢性ITP的人常常感到自己有一种无形的疾病，这种疾病不仅影响生活质量，而且威胁生命。这就是为什么我们鼓励我们的社区成员了解他们的疾病，了解治疗策略，并寻求支持，以让他们能够拥有最好的照顾。
  本次新药的获批为ITP医疗界提供了新选择。”
  血小板在凝血和愈合中起着积极的作用，但是在ITP患者中，免疫系统会攻击并破坏自身的血小板。ITP的常见症状包括过度瘀伤、出血和疲劳。患有慢性ITP的人可能会增加严重出血事件的风险，这可能导致严重的医学并发症甚至死亡。
  目前治疗ITP的方法包括类固醇、血小板生成促进剂和脾切除术。然而，并不是所有的病人都对现有疗法有足够的治疗反应。因此，对于ITP患者来说，仍有需要额外治疗方案的很大医疗需求
药品名称：Tavalisse
活性物质：Fostamatinib
剂       量：100mg；150mg
剂型/服用方式：Table；Oral
适应症：Idiopathic (Immune) Thrombocytopenic Purpura
持证商：Rigel Pharmaceuticals，Inc
持证商地址：1180 Veterans B1vd.South San Francisco,CA 94080 USA
生产商：Patheon Whitby
生产商地址：111 Consumers Drive Whitby，Ontario L1N 525 Canada
批准时间：2018.04.17
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TAVALISSE™(fostamatinib disodium hexahydrate) Tablets, for Oral Use
DESCRIPTION
Fostamatinib is a tyrosine kinase inhibitor. TAVALISSE is formulated with the disodium hexahydrate salt of fostamatinib, a phosphate prodrug that converts to its pharmacologically active metabolite, R406, in vivo.
The chemical name for fostamatinib disodium hexahydrate is disodium (6-[[5-fluoro-2-(3,4,5trimethoxyanilino) pyrimidin-4-yl]amino]-2,2-dimethyl-3-oxo-pyrido[3,2-b][1,4]oxazin-4-yl)methyl phosphate hexahydrate. The molecular formula is C23H24FN6Na2O9P·6H2O, and the molecular weight is 732.52. The structural formula is:

Fostamatinib disodium is a white to off-white powder that is practically insoluble in pH 1.2 aqueous buffer, slightly soluble in water, and soluble in methanol.
Each TAVALISSE oral tablet contains 100 mg or 150 mg fostamatinib, equivalent to 126.2 mg or 189.3 mg fostamatinib disodium hexahydrate, respectively.
The inactive ingredients in the tablet core are mannitol, sodium bicarbonate, sodium starch glycolate, povidone, and magnesium stearate. The inactive ingredients in the film coating are polyvinyl alcohol, titanium dioxide, polyethylene glycol 3350, talc, iron oxide yellow, and iron oxide red.
INDICATIONS
TAVALISSE is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
DOSAGE AND ADMINISTRATION
Recommended Dosage
Initiate TAVALISSE at a dose of 100 mg taken orally twice daily. After a month, if platelet count has not increased to at least 50 x 109/L, increase TAVALISSE dose to 150 mg twice daily.
Use the lowest dose of TAVALISSE to achieve and maintain a platelet count at least 50 x 109/L as necessary to reduce the risk of bleeding.
TAVALISSE may be taken with or without food. In the case of a missed dose of TAVALISSE, instruct patients to take their next dose at its regularly scheduled time.
Monitoring
After obtaining baseline assessments:
•Monitor CBCs, including platelet counts, monthly until a stable platelet count (at least 50 x 109/L) is achieved. Thereafter, continue to monitor CBCs, including neutrophils, regularly.
•Monitor liver function tests (LFTs) (e.g., ALT, AST, and bilirubin) monthly.
•Monitor blood pressure every 2 weeks until establishment of a stable dose, then monthly thereafter.
Dose Modification For Adverse Reactions
TAVALISSE dose modification is recommended based on individual safety and tolerability. Management of some adverse reactions may require dose -interruption, reduction, or discontinuation.
A dose reduction schedule is provided in Table 1, based on daily dose. For example, if a patient is on the maximum dose at the time of an adverse reaction, the first dose reduction would be from 300 mg/day to 200 mg/day.
Dose Modification For Drug Interactions
Concomitant use with a strong CYP3A4 inhibitor increases exposure to R406 (the major active metabolite). Monitor for toxicities of TAVALISSE that may require TAVALISSE dose modifications (see Table 1) when given concurrently with a strong CYP3A4 inhibitor [see DRUG INTERACTIONS].
Discontinuation
Discontinue TAVALISSE after 12 weeks of treatment if the platelet count does not increase to a level sufficient to avoid clinically important bleeding [see Clinical Studies].
HOW SUPPLIED
Storage And Handling
TAVALISSE 100 mg tablets are round, biconvex, orange, film-coated tablets debossed with “100” on one side and “R” on the reverse side.
TAVALISSE 150 mg tablets are oval, biconvex, orange, film-coated tablets debossed with “150” on one side and “R” on the reverse side.
100 mg tablets: Available in bottle of 60 with 2 desiccant canisters     NDC 71332-001-01
150 mg tablets: Available in bottle of 60 with 2 desiccant canisters     NDC 71332-002-01
Store at room temperature, 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature]. Do not remove desiccants.
Dosage Forms And Strengths
TAVALISSE is available as:
•100 mg tablet: orange, film-coated, round, biconvex tablets debossed with “100” on one side and “R” on the reverse side.
•150 mg tablet: orange, film-coated, oval, biconvex tablets debossed with “150” on one side and “R” on the reverse side.
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