近日,美国食品和药物管理局(FDA)批准Revcovi(elapegademase-lvlr)在美国的注射。脱氨酶严重联合免疫缺陷(ADA SCID)在儿科和成人患者中的应用。 ReVCOVI是一种PEG化重组腺苷脱氨酶(RADA)酶,由LedioAddioStudioS开发来治疗AAD-SCID。作为重组技术的产物,Revcovi消除了从动物中提取酶的需要,并且通过补充一种叫做腺苷脱氨酶(ADA)的基本酶的水平来工作。 批准日期:2018年10月5日 公司:Leadiant Biosciences, Inc ReVCOVI(EAPGEADMEAS-LVLR)注射液,肌肉注射用 首次美国批准:2018 作用机理 与艾达酶缺乏相关的SCID是一种罕见的遗传性疾病,通常是致命的疾病。ADA酶参与嘌呤代谢,催化腺苷或脱氧腺苷的不可逆水解脱氨,分别为肌苷或脱氧肌苷,以及几种天然存在的甲基化腺苷化合物。维持低水平的2’-脱氧腺苷和腺苷对于免疫细胞的数量和功能以及减少机会性感染的发生率至关重要。腺苷水平升高,如发生在ADA缺乏症,有助于细胞凋亡和阻断胸腺细胞的分化,导致严重T淋巴细胞减少。 Elapegademase-lvlr提供了外源ADA酶,该酶与腺苷酸酶在有毒腺苷和脱氧腺苷核苷酸水平以及淋巴细胞数量的增加有关[见临床研究]。 适应症及用法 REVCOVI是一种重组腺苷脱氨酶,用于治疗儿童和成人的严重联合免疫缺陷(ADA-SCID)。 剂量与给药 患者从ADGEN过渡到ReVCOVI:开始剂量ReVCOVI每周0.2mg/kg,肌肉注射。查看完整处方信息(FPI)转换公式从AdgEnto RevCOVI。 ●青少年患者:REVCOVI的起始剂量是0.4mg/kg,基于理想的体重,每周分成两个剂量(0.2mg/kg,每周两次),肌肉注射。 对于完整的信息、维护剂量和治疗监测,请参见FPI。 RVCOVI仅用于肌肉注射。参见行政指示的FPI。 剂型和强度 注射:在单剂量小瓶中注射2.4mg/1.5毫升(1.6mg/ml)。 禁忌症 没有 警告和注意事项 ·血小板减少症患者注射部位出血:血小板减少症患者局部出血风险增加;如果血小板减少症严重,则不应使用。 延缓免疫功能的改善:保护免疫缺陷患者免受感染直至免疫功能的改善。 不良反应 最常见的不良反应为咳嗽(50%)和呕吐(33%)。 包装供应/储存和搬运 REVCOVI(elapegademase-lvlr)注射液,2.4mg/1.5mL(1.6mg/mL),是一种无菌、无防腐剂、透明、无色的肌肉内用溶液,可作为每个纸箱的单剂量小瓶使用(NDC 57665-002-01)。 瓶塞不是用天然胶乳制成的。 单剂量药瓶;不要重复使用小瓶。丢弃未使用的部分。 将ReVCOVI存放在冰箱中2°C至8°C(36°F至46°F)之间,在原纸箱中起到保护光的作用。不要冷冻或摇晃。如果有任何迹象表明它可能已被冻结,则不应使用ReVCOVI。 完整说明书附件: http://revcovi.com/wp-content/uploads/2018/10/revcovi-final-labeling-text-10-05-2018.pdf
Revcovi Approved for Adenosine Deaminase Severe Combined Immune Deficiency Leadiant Biosciences announced that the Food and Drug Administration (FDA) has approved Revcovi (elapegademase-lvlr) injection, a new enzyme replacement therapy (ERT) for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID). ADA-SCID is an ultra-rare, inherited genetic disorder caused by an ADA enzyme deficiency, that if left untreated, can be fatal. The condition mainly affects infants and young children and is diagnosed within the first few months of life. Revcovi, a PEGylated recombinant adenosine deaminase, works by supplementing levels of the ADA enzyme, eliminating the need to source the enzyme from animals. The FDA approval was supported by data from 2 multicenter, open-label clinical trials (Study 1 [N=6] and Study 2 [N=4]). The data showed treatment with Revcovi increased ADA activity, decreased concentrations of toxic metabolites, and improved total lymphocyte counts. Cough and vomiting were the most common adverse reactions associated with Revcovi therapy. “Individuals with ADA-SCID are at an increased risk of severe and recurrent infections and often fail to thrive," said Morna Dorsey, MD, MMSc, Professor of Pediatrics at the University of California, San Francisco. "By providing specific and direct replacement of the adenosine deaminase enzyme, Revcovi can reduce patients' risk of potentially serious, life-threatening infections and their debilitating complications." Revcovi is expected to be available by the end of 2018 in single-dose vials containing 2.4mg/1.5mL (1.6mg/mL) sterile, preservative-free solution for intramuscular (IM) injection.
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