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近日，美国食品和药物管理局今天批准了Gamifant(emapalumab)用于治疗患有原发性噬血细胞性淋巴组织细胞增多症(HLH)的儿童(新生儿及以上)和成年患者，这些患者患有难治性、复发性或进行性疾病或不耐受症并伴有转归。NH-HLH治疗获FDA批准为HLH专用药物。
“原发性HLH是一种罕见的危及生命的疾病，通常影响儿童，这种批准填补了这些患者未满足的医学需求，”FDA肿瘤学中心主任、血液学和肿瘤学专业办公室代理主任Richard Pazdur医学博士说。FDA药物评价和研究中心的导管。“我们致力于继续加快开发和审查为罕见疾病患者提供有意义的治疗方案的疗法。” HLH是机体免疫细胞不能正常工作的一种状态。细胞变得过度活跃释放分子，从而导致炎症。免疫细胞开始损害身体自身的器官，包括肝脏、大脑和骨髓。它可以被继承，这被称为原发性或家族性HLH。它也可以有非遗传的原因。原发性HLH患者通常在生命的第一个月或几年内出现症状。症状可能包括发烧、肝脾肿大和血细胞减少。
批准日期：2018年11月20日 公司：Novimmune SA
GAMIFANTTM(emapalumab-lzsg)注射液，用于静脉注射
美国初步批准:2018年
作用机制
Emapalumab-lzsg是一个结合的单克隆抗体和中和干扰素γ(IFNγ)。临床前数据表明IFNγ的发病机制中起着举足轻重的作用通过可是hypersecreted。
适应症和用法
GAMIFANT是一种干扰素γ(IFNγ)抑制性抗体表示为治疗成人和儿童患者(新生儿和以上)初选食血淋巴组织细胞增生症(HLH)，伴有顽固性、复发性或进行性疾病或传统HLH治疗不耐受。
剂量和管理
只适用于静脉输液:
建议起始剂量:1mg/kg，每周两次，每次1小时以上。
与GAMIFANT一起使用地塞米松。
剂型及强度
注:
10mg/2mL (5mg/mL)单剂量瓶溶液。
50mg/10mL (5mg/mL)单剂量瓶溶液。
禁忌症
没有。
警告和预防措施
感染:监测患者的体征和症状并及时治疗。
检测潜伏肺结核。对带状疱疹、肺囊虫病和真菌感染进行预防性治疗。
活疫苗:不要给服用GAMIFANT的患者注射活疫苗或减毒活疫苗。
与注射相关的反应:监测患者是否发生与注射相关的反应。对严重的输液反应进行中断输液和适当的医疗管理。
不良反应
最常见的不良反应(≥20%):感染、高血压、输注相关反应和发热。
要报告可疑的不良反应，请联系1-866-773-5274或FDA(电话:1-800-FDA-1088)或www.fda.gov/medwatch。
包装提供/存储和处理
GAMIFANT (emapalumab-lzsg)注射液是一种无菌、透明至微乳白色、无色至微黄色的溶液，包装配置如下:
NDC 72171-501-01 -含1支10毫克/2毫升(5毫克/毫升)单剂量瓶
NDC 72171-505-01 -含50mg/10ml (5mg/mL)单剂量瓶
GAMIFANT存储在冰箱2ºC - 8ºC(36ºF 46ºF)在原来的纸箱保护fromlight。不要冻僵或摇晃。这种产品不含防腐剂。
完整资料附件：https://gamifant.com/pdf/Full-Prescribing-Information.pdf
FDA Approves Gamifant(emapalumab-lzsg), the First and Only Treatment Indicated for Primary Hemophagocytic Lymphohistiocytosis (HLH)
Indication and Usage
Gamifant® (emapalumab-lzsg) is an interferon gamma (IFNγ)–blocking antibody indicated for the treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.
Important Safety Information
Before initiating Gamifant, patients should be evaluated for infection, including latent tuberculosis (TB). Prophylaxis for TB should be administered to patients who are at risk for TB or known to have a positive purified protein derivative (PPD) test result or positive IFNγ release assay.
During Gamifant treatment, patients should be monitored for TB, adenovirus, Epstein-Barr virus (EBV), and cytomegalovirus (CMV) every 2 weeks and as clinically indicated.
Patients should be administered prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infections prior to Gamifant administration.
Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.
Infusion-Related Reactions
Infusion-related reactions, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.
Adverse Reactions
In the pivotal trial, the most commonly reported adverse reactions (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).
Additional selected adverse reactions (all grades) that were reported in less than 10% of patients treated with Gamifant included vomiting, acute kidney injury, asthenia, bradycardia, dyspnea, gastrointestinal hemorrhage, epistaxis, and peripheral edema.
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