Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand.
Drugs. 2021 May;81(7):841-848. doi: 10.1007/s40265-021-01511-3. Epub 2021 Apr 19.
Givosiran (Givlaari) is an δ-aminolevulinic acid synthase 1 (ALAS1)-directed small interfering RNA (siRNA) approved for the treatment of acute hepatic porphyria (AHP). In the phase 3 ENVISION trial, givosiran significantly reduced the annualized rate of composite porphyria attacks (i.e. attacks requiring hospitalization, urgent healthcare visit or intravenous hemin administration at home) compared with placebo in patients with recurrent acute intermittent porphyria (the most common type of AHP) attacks. Givosiran also improved several other outcomes, including hemin use and pain (the cardinal symptom of AHP). While generally well tolerated with an acceptable safety profile, the drug may increase the risk of hepatic and kidney adverse events. Givosiran offers the convenience of once-monthly subcutaneous administration. Available evidence indicates that givosiran is an important newer therapeutic option for patients with AHP and severe recurrent attacks.
吉伏沙兰（Givlaari）是一种δ-氨基酮戊酸合酶 1（ALAS1）靶向的小干扰 RNA（siRNA），已被批准用于治疗急性肝卟啉症（AHP）。在 3 期 ENVISION 试验中，与安慰剂相比，吉伏沙兰显著降低了复发性急性间歇性卟啉症（最常见的 AHP 类型）患者的复合卟啉症发作的年化发生率（即需要住院、紧急医疗就诊或在家中静脉内给予血红素治疗的发作）。吉伏沙兰还改善了其他几项结局，包括血红素的使用和疼痛（AHP 的主要症状）。虽然该药总体耐受性良好，安全性可接受，但可能会增加肝脏和肾脏不良事件的风险。吉伏沙兰提供了每月一次皮下给药的便利。现有证据表明，吉伏沙兰是 AHP 患者和严重复发性发作患者的一种重要的新型治疗选择。
