近日，FDA批准ARIKAYCE（amikacin liposome inhalation suspension）的雾化系统。ARIKAYCE是第一种也是唯一一种专门用于治疗成人肺部分枝杆菌Avium复合体(MAC)疾病的治疗方法，其治疗方案有限，或者没有其他选择。 批准日期：2018年10月3日 公司：PARI制药公司 ARIKAYCE(阿米卡星脂质体[amikacin liposome])悬液,口服吸入 首次美国批准：2018 有限人口警告：增加呼吸不良的风险反应见完整处方信息完整盒装警告。 ARIKAYCE与呼吸系统不良反应增加的风险相关，包括过敏性肺炎、咯血、支气管痉挛，以及导致某些病例住院的潜在肺部疾病的恶化。 作用机理 ARIKAYCE是一种抗菌药物[参见微生物学]。 适应症及用法 有限人口：ARIKAYCE是一种氨基糖苷类抗生素，用于治疗禽分枝杆菌复合体（MAC）肺部疾病，用于治疗痰菌阴性的患者。M培养后至少6个月的多药背景治疗方案。由于目前只有有限的ARIKAYCE的临床安全性和有效性数据可用，因此保留ARIKAYCE用于那些选择有限或没有替代疗法的成年人。这种药物是在有限和特定人群中使用的。 患者。这个适应症是在加速批准下批准的，基于实现粪便培养转化（定义为连续3个月阴性痰）文化6个月。 临床效益尚未确定。 使用限制： ARICKYCE仅在难治性MAC肺部疾病患者中被定义为在至少6个月的多药背景治疗方案后没有达到痰培养阴性的患者。不适用于非难治性肺疾病的患者不宜使用ARIKAYCE。 剂量与给药 仅用于口服吸入。 ·使用LAMIRACE小瓶仅与LAMRAA雾化器系统。 成人推荐剂量为每日一次口服吸入590毫克/8.4毫升ARIKAYCE小瓶的内容物。 应考虑吸入性支气管扩张剂的预治疗在有高反应性气道疾病史的患者中的应用。 剂型和强度 ARIKAYCE作为一种无菌的水性脂质体悬浮液，在含有阿米卡星590mg/8.4毫升的单位剂量玻璃瓶内进行口服吸入。 禁忌症 ARIKAYCE在已知的氨基糖苷类药物过敏患者中是禁忌的。 警告和注意事项 过敏性肺炎：用ARIKAYCE治疗；超敏性肺炎发生，停药和管理者在医学上适当。 咯血：用ARIKAYCE治疗咯血的频率更高。如果咯血发生，应适当地管理患者。 支气管痉挛：支气管痉挛的发生率较高。如果在ARIKAYCE治疗期间发生这种情况，就医治疗。 肺内疾病恶化：肺动脉硬化症加重的发生率已报告阿里克伊斯治疗。如果在ARIKAYCE治疗期间发生这种情况，就医治疗。 耳毒性：用ARIKAYCE治疗耳毒性的频率更高。密切监测已知或可疑的前庭功能障碍或前庭功能障碍患者。如果患者发生耳鸣，这可能是耳毒性的早期症状。 肾毒性：氨基糖苷类药物可引起肾毒性。对已知或疑似肾功能不全患者的闭锁可能在开处方时受益。 •神经肌肉阻滞：氨基糖苷可加重肌肉无力，因为潜在的荨麻疹样作用对神经肌肉功能。如果发生神经肌肉阻滞，可能通过施用钙盐来逆转，但是可能需要机械辅助。 •胚胎-胎儿毒性：氨基糖苷可导致暴露在子宫中的儿科患者的完全、不可逆的双侧先天性耳聋。 不良反应 难治性MAC肺病患者最常见的不良反应(发生率≥10%,高于对照组)有:发音困难、咳嗽、支气管痉挛、咯血、耳毒性、上呼吸道刺激、肌肉骨骼痛、疲劳/乏力及肺部疾病加重、腹泻。恐吓和恶心。 包装供应/储存和搬运 供应 ARIKAYCE（阿米卡星脂质体吸入悬浮液），590毫克/毫升8.4毫升，提供在无菌，单位-剂量10-ml玻璃瓶。该产品被分配为28瓶成套工具。 每个纸箱包含28天的药物供应（28瓶）。除了卡里顿的ARIKAYCE小瓶外，还提供了一个拉米拉雾化器手机和四个拉米拉喷雾头。 NDC 71558－590-28 LAMRAA雾化器系统包含控制器、备用气雾剂头、备用手机、电源线和附件。 储运 储存在2°C至8°C（36°F至46°F）的AikayCE瓶，直到瓶上的到期日期。不要冻僵。一旦过期，丢弃任何未使用的药物。 ARIKYCE可以在室温下储存到25°C（77°F），持续4周。一旦在室温下，任何未使用的药物必须在4周结束时被丢弃。 完整说明书附件：https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/207356s000lbl.pdf ARIKAYCE(amikacin liposome inhalation suspension) U.S. Food and Drug Administration (FDA) has granted accelerated approval of ARIKAYCE® (amikacin liposome inhalation suspension) for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients who have limited or no alternative treatment options. ARIKAYCE is the first and only therapy approved in the U.S. specifically for patients with MAC lung disease, a chronic and debilitating condition that can significantly increase patient morbidity and mortality. ARIKAYCE is the first product approved via the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD). LPAD, which was enacted as part of the 21st Century Cures Act, serves to advance the development of new antibacterial drugs to treat serious or life-threatening infections in limited populations of patients with unmet needs.   About ARIKAYCE®(amikacin liposome inhalation suspension) ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed’s proprietary PULMOVANCE™ liposomal technology enables the delivery of amikacin directly to the lungs, where it is taken up by lung macrophages where the infection resides. This approach prolongs the release of amikacin in the lungs while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira™ Nebulizer System manufactured by PARI Pharma GmbH. About PARI Pharma and the Lamira™ Nebulizer System ARIKAYCE® (amikacin liposome inhalation suspension) is delivered by a novel inhalation device, the Lamira™ Nebulizer System, developed by PARI. Lamira™ is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ARIKAYCE, via a vibrating, perforated membrane. Based on PARI's 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care. About CONVERT (INS-212) and INS-312 CONVERT is a randomized, open-label, global Phase 3 trial designed to confirm the sputum culture conversion results seen in Insmed's Phase 2 clinical trial of ARIKAYCE in patients with refractory NTM lung disease caused by MAC. CONVERT is being conducted in 18 countries at more than 125 sites. The primary efficacy endpoint is the proportion of patients who achieved sputum culture conversion at Month 6 in the ARIKAYCE plus GBT arm compared to the GBT-only arm. Patients who achieved sputum culture conversion by Month 6 are continuing in the CONVERT study for an additional 12 months of treatment following the first monthly negative sputum culture. Patients who did not culture convert may have been eligible to enroll in our INS-312 study. INS-312 is a single-arm open-label extension study for patients who completed six months of treatment in the INS-212 study but did not demonstrate culture conversion by Month 6. Under the study protocol, non-converting patients in the ARIKAYCE plus GBT arm of the INS-212 study will receive an additional 12 months of ARIKAYCE plus GBT. Patients who crossed over from the GBT-only arm of the INS-212 study will receive 12 months of treatment of ARIKAYCE plus GBT.