镰状细胞疾病新药L-glutamine（商品名 Endari）为近20年中首个被批准治疗罕见血液疾病的口服粉药
近日，美国食品和药品局今天批准Endari (L-谷氨酰胺[glutamine]口服粉)为患者年龄5岁和以上有镰状细胞疾病减少伴随血液疾病严重并发症。 FDA的药品评价和研究中心血液学和肿瘤学办公室代理主任和卓越的FDA肿瘤学中心主任Richard Pazdur，M.D.说："Endari是在几乎20年中为有镰状细胞疾病患者首个被批准的治疗，""迄今，仅有一个为生活在有这个严重的，衰弱性状态患者其他药物被批准。"
批准日期：2017年7月7日 公司：Emmaus Life Sciences Inc
ENDARI (L-谷氨酰胺[glutamine])粉剂 供口服使用
美国初次批准：2017
作用机制
氨基酸L-谷氨酰胺在治疗镰刀细胞病(SCD)的作用机制是未完全了解。在SCD的病理生理学涉及氧化应急现象。镰刀红细胞(RBCs)是比正常RBCs对氧化损伤更易感，它可能对慢性溶血和伴随SCD血管-阻塞事件有贡献。吡啶核苷酸，NAD+和其还原型NADH，在RBCs中在调节和预防氧化损伤中起作用。在镰刀RBCs通过增加还原型谷胱甘可利用性L-谷氨酰胺可能改善NAD氧化还原电位势。
适应证和用途
ENDARI是一种氨基酸适用减低在成年和5岁和以上儿童患者镰刀细胞病急性并发症。
剂量和给药方法
● 5克至15克口服地，每天2次根据体重。
● 在摄取前Endari的每剂应被8 oz. (240mL)的冷或室温饮料或4 oz.至6 oz.的食物混合。
剂型和规格
口服粉：5克的L-谷氨酰胺粉每个纸箔层压塑料包装。
禁忌证
无。
不良反应
最常见不良反应(发生率>10%)是便秘，恶心，头痛，腹痛，咳嗽，肢体痛，背痛，和胸痛。
包装规格/贮存和处置
Endari是在纸箔层压塑料包中含 5克的L-谷氨酰胺白色结晶粉中供应。
● 60包装纸盒：NDC 42457-420-60
贮存在20ºC至25ºC (68ºF至77ºF)离开直接阳光
完整说明书附件：https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d5a783f4-12ef-4326-8faa-40018e45ba3b
Endari Approval History
•FDA approved: Yes (First approved July 7th, 2017)
•Brand name: Endari
•Generic name: L-glutamine
•Dosage form: Oral Powder
•Company: Emmaus Life Sciences Inc.
•Treatment for: Anemia, Sickle Cell
Endari (L-glutamine) is orally-administered pharmaceutical grade L-glutamine (PGLG), an amino acid formulation to relieve pain, swelling and other complications of sickle cell anemia.
Emmaus Life Sciences announced that the Food and Drug Administration (FDA)'s Oncologic Drugs Advisory Committee voted 10–3 that the overall benefit-risk profile of Endari (L-glutamine) for the treatment of sickle cell disease was favorable.
If approved, Endari will be the first treatment for pediatric patients with sickle cell disease as well as the first treatment for adults in nearly two decades. Endari has previously received Orphan Drug and Fast Track designations from the FDA.
An efficacy study of L-glutamine (Study 09-01) showed that the treatment resulted in a lower frequency of sickle cell painful crises, longer time to first and second crisis, fewer ACS (acute chest syndrome), fewer hospitalizations, fewer days hospitalized, and fewer transfusions compared to placebo. Overall, L-glutamine was well tolerated with a safety profile similar to placebo.
The most common side effects for Latuda versus placebo were nausea (16% vs. 5.85%), somnolence (9.1% vs. 4.7%), weight gain (6.9% vs. 1.7%), vomiting (6.3% vs. 3.5%), dizziness (5.7% vs. 4.7%) and insomnia (5.1% vs 2.3%).
The full data from this study will be presented at the 2017 Annual Meeting of the American Psychiatric Association, it has also been sent to the Food and Drug Administration to support a supplemental New Drug Application (sNDA).
Latuda, an atypical antipsychotic, is already approved for the treatment of major depressive episodes associated with bipolar I disorder as monotherapy and as adjunctive therapy with lithium or valproate in adults and for the treatment of schizophrenia in patients ≥13 years of age. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm566084.htm https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/208587s000lbl.pdf
