FDA批准Enspryng新适应证：首款可居家注射的甲状腺眼病药物获批
美国食品药品管理局（FDA）已批准基因泰克公司（Genentech）的药物 Enspryng（通用名：萨特利珠单抗，satralizumab-mwge）用于治疗成人甲状腺眼病。基因泰克10月15日在加州南旧金山总部宣布，这是首个、也是目前唯一一个可在家中皮下注射的甲状腺眼病治疗方案，患者本人或照护者在接受医护人员培训后即可自行注射。
甲状腺眼病又称格雷夫斯眼病，是一种累及眼部及眼周组织的自身免疫性炎症疾病，常见表现包括眼球突出、复视、眼睑退缩、眼部红肿与疼痛，严重时可威胁视力。据基因泰克介绍，该病发病率约为每10万人155人，最常见于甲状腺功能亢进人群，约半数甲亢患者会出现至少轻度的眼部病变。
此次批准经过FDA优先审评，依据是两项三期临床试验 SatraGO-1 与 SatraGO-2，两项试验共纳入19个国家的258名患者。在 SatraGO-2 中，活动性甲状腺眼病患者治疗24周后，眼球突出改善达到预设标准者占52.9%，安慰剂组为23.4%；复视减轻者占60.7%，安慰剂组为25.8%。试验期间未发现新的安全性信号。基因泰克同时说明，SatraGO-1 未达到其预设的主要疗效终点，本次批准基于两项试验的总体数据。
这是 Enspryng 在美国获批的第二个适应证，该药2020年已获批用于治疗视神经脊髓炎谱系疾病（NMOSD）。临床试验研究者、宾夕法尼亚大学谢伊眼科研究所副教授塞萨尔·布里塞尼奥（César A. Briceño）表示，皮下注射方案可为患者与医生提供更大灵活性，减少往返医院接受输注治疗的负担。基因泰克首席医疗官利瓦伊·加拉韦（Levi Garraway）表示，此次批准为长期以来治疗选择有限的甲状腺眼病患者提供了一个重要新选项。
FDA Approves Enspryng for Thyroid Eye Disease, the First At-Home Subcutaneous Treatment
The U.S. Food and Drug Administration (FDA) has approved Genentech's Enspryng (satralizumab-mwge) for the treatment of adults with thyroid eye disease (TED), the company announced on October 15 at its South San Francisco headquarters. Genentech, a member of the Roche Group, said Enspryng is the first and only at-home subcutaneous treatment option for the disease, and can be administered by the patient or a caregiver after training from a healthcare provider.
Thyroid eye disease, also known as Graves' ophthalmopathy, is an autoimmune inflammatory disorder of the eye and surrounding tissues. Common symptoms include bulging eyes (proptosis), double vision, eyelid retraction, redness, swelling and pain, and the disease can threaten vision. Genentech said TED affects approximately 155 out of every 100,000 people in the United States and occurs most commonly in people with hyperthyroidism.
The approval, granted following a priority review, was based on two Phase III studies, SatraGO-1 and SatraGO-2, which enrolled a total of 258 patients from 19 countries. In SatraGO-2, 52.9% of patients with active TED receiving Enspryng achieved the proptosis response endpoint at week 24, compared with 23.4% on placebo, and 60.7% achieved a reduction in double vision, compared with 25.8% on placebo. No new safety signals were identified. Genentech noted that SatraGO-1 did not meet its pre-specified primary efficacy endpoint, and that the approval rested on the totality of the data.
This is the second U.S. indication for Enspryng, following its 2020 approval for neuromyelitis optica spectrum disorder (NMOSD). Dr. César A. Briceño, a clinical trial investigator and associate professor of ophthalmology at the Scheie Eye Institute of the University of Pennsylvania, said a subcutaneous option could offer greater flexibility for patients and physicians while reducing the logistical burden of infusion-based therapies. Dr. Levi Garraway, Genentech's chief medical officer, said the approval provides an important new option for people with TED, who have long faced limited treatment choices.
来源：GSM·美国（原文链接）
图片来源：甲状腺眼病（格雷夫斯眼病）患者眼球突出与眼睑退缩临床照片（资料图）。照片：Jonathan Trobe 医学博士／密歇根大学 Kellogg 眼科中心，CC BY 3.0，经 Wikimedia Commons
GSM Global · 本篇英文版 English edition of this story
- 2026-10-16 00:45FDA批准Enspryng新适应证：首款可居家注射的甲状腺眼病药物获批本篇
- 2026-10-17 20:17食品药品监管局批准Enspryng治疗甲状腺眼病 成首款可居家皮下注射的该病疗法
