2018年8月13日，美国FDA批准RNAi治疗药物patisiran（商品名ONPATTRO，Alnylam制药公司）输注上市，用于由遗传性转甲状腺素蛋白淀粉样变性（hATTR）引起的周围神经疾病（多发性神经病，polyneuropathy）成人患者。值得一提的是，这是FDA批准的首款用于治疗由hATTR引起的多发性神经病患者的疗法，也是FDA批准的首款小干扰RNA（siRNA）药物。   hATTR影响了全球约50000人，是一种罕见、使人衰弱且常常致命的遗传性疾病。它的主要特征是在体内器官和组织中形成称为淀粉样蛋白的蛋白质纤维异常沉积物，干扰器官和组织的正常功能。这些蛋白质沉积最常发生在周围神经系统中，会导致手臂、腿、手和脚的感觉丧失、疼痛或不能移动。淀粉样蛋白沉积物也会影响心脏、肾脏、眼睛和胃肠道的功能。目前的治疗方法通常侧重于症状管理，这一领域还有医疗需求亟待满足。   由Alnylam开发的Onpattro是一种靶向转甲状腺素蛋白（transthyretin，TTR）的siRNA疗法，这类药物通过`审评资格、快速通道资格和孤儿药资格。今日它的获批，对患者和医生来说，都具有里程碑的意义。   Onpattro的疗效在临床试验中也得到了证实。在一项包含225名患者的研究中，148名患者被随机分配接受Onpattro输注，每三周一次，共18个月，另外77名患者被随机分配接受相同频率的安慰剂输注。   结果显示，与接受安慰剂输注的患者相比，接受Onpattro治疗的患者在多发性神经病变测量方面有更好的结果，包括肌肉力量、感觉（疼痛、体温、麻木）、反射和自主神经症状（血压、心率、消化）。接受Onpattro治疗的患者在行走、营养状况和进行日常活动能力的评估中也有更高的得分。 完整资料附件： 1）：https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 2）：http://www.alnylam.com/wp-content/uploads/2018/09/RNAi-Roundtable_TTR_Slides_FINAL.pdf Onpattro(Patisiran Lipid Complex Injection) ONPATTRO Rx Generic Name and Formulations: Patisiran 10mg/5mL (2mg/mL); per vial; lipid complex for IV infusion after filtration and dilution; preservative-free. Company: Alnylam Pharmaceuticals Indications for ONPATTRO: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Adult: See full labeling. Premedicate with IV corticosteroid, oral acetaminophen, IV H1- and H2-blocker on the day of Onpattro infusion at least 60mins prior to initiation; may reduce corticosteroid as needed, if related adverse reactions occur. Give as IV infusion over ~80mins at an initial rate of 1mL/min for first 15mins, then increase to ~3mL/min for the remainder. <100kg: 0.3mg/kg once every 3 weeks. ≥100kg: 30mg once every 3 weeks. Children: Not established. Warnings/Precautions: Monitor for infusion-related reactions during infusion; consider slowing or interrupting infusion as clinically indicated; discontinue and do not resume if serious or life-threatening reaction occurs. Monitor infusion site for possible infiltration during infusion; manage suspected extravasation accordingly. Reduced serum Vit. A levels may develop; give supplementation at the recommended daily allowance of Vit. A. Refer patients for eye exam if ocular symptoms suggestive of Vit. A deficiency (eg, night blindness). Moderate or severe hepatic impairment: not studied. Severe renal impairment or ESRD: not studied. Pregnancy. Nursing mothers. Pharmacological Class: Small interfering RNA (siRNA). Adverse Reactions: Upper respiratory tract infections, infusion-related reactions, dyspepsia, dyspnea, muscle spasms, arthralgia, erythema, bronchitis, vertigo. Generic Availability: NO How Supplied: Single-dose vial—1