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近日，美国FDA批准Doptelet（avatrombopag）片用于治疗血小板计数降低（血小板减少症），适用于拟进行医疗或牙科手术的慢性肝病成年患者。这是FDA批准用于该适应症的首个药物。
FDA药物评价与研究中心血液学及肿瘤产品办公室代理主任兼FDA肿瘤优化中心主任Pazdur博士称：血小板计数降低及需要手术的慢性肝病患者的出血风险增加，Doptelet证明可以安全地增加血小板计数。这款药物可以降低或消除血小板输注的需求，从而避免相关的感染风险及其它副作用。
批准日期：2018年5月23日 公司：AkaRx Inc.
DOPTELET（avatrombopag）片剂，用于口服
美国最初批准：2018年
作用机制
Avatrombopag是一种口服生物可利用的小分子TPO受体激动剂，可刺激骨髓祖细胞中巨核细胞的增殖和分化，从而增加血小板的产生。Avatrombopag不与TPO竞争结合TPO受体，并且与TPO在血小板生成上具有累加效应。
适应症和用法
DOPTELET（avatrombopag）是一种血小板生成素受体激动剂，用于治疗预定接受手术的成人慢性肝病患者的血小板减少症。
剂量和给药
在预定程序之前10至13天开始给药DOPTELET。
患者应在最后一次给药后5至8天内接受手术。
DOPTELET应每天口服一次食物，连续5天。
推荐剂量的DOPTELET基于患者在预定程序之前的血小板计数。
推荐剂量和持续时间：
血小板计数（x109/L） 每日剂量一次 持续时间
不到40 60毫克（3片） 5天
40到不到50 40毫克（2片） 5天
剂量形式和强度
片剂：20mg
禁忌症
没有。
警告和注意事项
血栓形成/血栓栓塞并发症：DOPTELET是一种血小板生成素（TPO）受体激动剂，TPO受体激动剂与慢性肝病患者的血栓形成和血栓栓塞并发症有关。监测血小板计数和血栓栓塞事件并立即进行治疗。
不良反应
最常见的不良反应（≥3％）是：发热，腹痛，恶心，头痛，疲劳和外周水肿。
要报告疑似不良反应，请致电1-844-506-3682联系Dova制药公司或1-800-FDA-1088或www.fda.gov/medwatch联系FDA。
用于特定人群
怀孕：根据动物研究，可能会导致胎儿伤害
哺乳期：治疗期间不推荐母乳喂养.
包装提供/存储和处理
DOPTELET 20mg片剂以圆形，双凸面，黄色，薄膜包衣片剂形式提供，并且在一侧用“AVA”压印，在另一侧用“20”压印。
NDC 71369-020-10：带有10个20毫克片剂的一个吸塑卡的纸箱
NDC 71369-020-11：一个含有10个20毫克片剂的泡罩卡
NDC 71369-020-15：带有15个20毫克片剂的一个吸塑卡的纸箱
NDC 71369-020-16：一个15个20毫克片剂的吸塑卡
储存温度为20°C至25°C（68°F至77°F），允许偏移温度为15°C至30°C（59°F至86°F）。 将药片保存在原包装中。
完整资料附件：
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e2d5960d-6c18-46cc-86bd-089222b09852
Avatrombopag Tablet Approved by FDA for Patients with Chronic Liver Disease
The FDA has announced its approval of avatrombopag (Doptelet, AkaRx) tablets to treat low blood platelet count in adults with chronic liver disease (CLD) who are scheduled to undergo a medical or dental procedure. This is the first drug approved for this use by the FDA.
According to Richard Pazdur, MD, 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, patients with chronic liver disease who have low platelet counts—thrombocytopenia—and who require a medical or dental procedure are at increased risk of bleeding.
“Doptelet was demonstrated to safely increase the platelet count. This drug may decrease or eliminate the need for platelet transfusions, which are associated with risk of infection and other adverse reactions,” Pazdur said, in a statement.
The drug is expected to be made available in June, according to Dova Pharmaceuticals, the parent company of AkaRx.2 “Doptelet is the first orally administered treatment option for patients with CLD, allowing a majority of patients to avoid a platelet transfusion prior to a procedure by increasing platelet counts to the target level of greater or equal to 50,000 per microliter,” said Alex Sapir, president and CEO of Dova Pharmaceuticals, in a statement.
When patients have moderately to severely reduced platelet counts, serious or life-threatening bleeding can occur, especially during invasive procedures.
Patients with significant thrombocytopenia typically receive platelet transfusions immediately prior to a procedure to increase the platelet count.
The safety and efficacy of avatrombopag was studied in a pair of Phase 3 trials (ADAPT-1 and ADAPT-2) involving 435 patients with chronic liver disease and severe thrombocytopenia, who were scheduled to undergo a procedure that would typically require platelet transfusion. The trials investigated 2 dose levels of avatrombopag administered orally over 5 days, as compared to placebo. The trial results showed that for both dose levels of avatrombopag, a higher proportion of patients had increased platelet counts, and did not require platelet transfusion or any rescue therapy on the day of the procedure, and up to 7 days following the procedure, as compared to those treated with placebo.
“Given the need for patients with CLD to routinely undergo multiple, invasive procedures, the availability of an oral agent that can lead to a measured increase in platelets, to minimize the need for platelet transfusions and risk of bleeding, will facilitate the clinical management of these patients,” said Norah Terrault, MD, MPH, principal investigator for the pivotal pair of Phase 3 avatrombopag trials, and a professor of medicine at the University of California San Francisco’s Division of Gastrointerology, in a statement.
The most common adverse effects reported by clinical trial participants who received avatrombopag were fever, stomach pain, nausea, headache, fatigue, and swelling in the hands or feet. People with CLD, and people with certain blood clotting conditions may have an increased risk of developing blood clots when taking avatrombopag.
This product was granted Priority Review, under which the FDA’s goal is to take action on an application within 6 months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.
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