UCB重症肌无力药ZILBRYSQ自动注射笔获FDA批准 预计2027年一季度上市
布鲁塞尔讯——优时比（UCB，布鲁塞尔泛欧交易所：UCB）10月16日宣布，美国食品药品监督管理局（FDA）已批准其ZILBRYSQ（zilucoplan）自动注射笔，用于抗乙酰胆碱受体（AChR）抗体阳性的成人全身性重症肌无力（gMG）患者自行给药。该药是首个也是唯一获批可由患者自行每日一次皮下注射的大环肽类C5抑制剂；此前该药已以可自行给药的预充式注射器形式在美国上市，2023年首次获得批准。
批准依据包括：一项在14名健康受试者中进行的一期研究显示自动注射笔与预充式注射器具有生物等效性；一项三期b研究中，449支自动注射装置的给药99.8%实现了完整剂量递送；一项100名参与者（患者、护理者和医护人员）的人体工学研究显示，90%的参与者首次尝试即在未经培训的情况下完成注射。最常见的治疗期不良事件为注射部位疼痛，发生率9.7%。自动注射笔预计于2027年第一季度在美国上市。
UCB美国罕见病负责人Kimberly Moran在公告中表示，自动注射笔为全身性重症肌无力患者提供了一种自行给药的新选择。UCB总部位于比利时布鲁塞尔，专注于免疫学和神经学疾病领域。
FDA Approves UCB's ZILBRYSQ Autoinjector for Generalized Myasthenia Gravis
BRUSSELS — UCB (Euronext Brussels: UCB) announced on October 16 that the U.S. Food and Drug Administration has approved the ZILBRYSQ (zilucoplan) autoinjector for self-administration by adults with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody positive. Zilbrysq is the first and only macrocyclic peptide C5 inhibitor approved in the United States as a self-administered, once-daily subcutaneous autoinjector for gMG; it was previously available as a self-administered prefilled syringe following its initial U.S. approval in 2023.
The approval was supported by a phase 1 study in 14 healthy volunteers showing bioequivalence between the autoinjector and the prefilled syringe; a phase 3b study in which 99.8% of 449 autoinjector administrations achieved complete dose delivery; and a human-factors study of 100 participants — patients, caregivers and health-care professionals — in which 90% completed the injection on the first attempt without training. The most common treatment-emergent adverse event was injection-site pain, at 9.7%. Commercial availability of the autoinjector is expected in the first quarter of 2027.
Kimberly Moran, UCB's senior vice president and head of U.S. rare diseases, said in the release that the autoinjector gives people living with gMG a new self-administration option. UCB is headquartered in Brussels, Belgium, and focuses on immunology and neurology.
发布：GSM·美国（发布链接）
图片来源：ZILBRYSQ产品图（UCB官网） / ZILBRYSQ product image (UCB official site)
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