Amgen announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for TEPEZZA® (teprotumumab) as the first therapy specifically licensed for the treatment of adult patients with moderate-to-severe Thyroid Eye Disease (TED).

安进公司宣布，英国药品和健康产品管理局 (MHRA) 已授予 TEPEZZA®（替普鲁单抗）上市许可，这是首个专门用于治疗中度至重度甲状腺眼病 (TED) 成人患者的疗法。

Affecting approximately 50,000 people in the UK,1 TED is a progressive and potentially vision-threatening condition, which can cause eye bulging, double vision, eye pain, redness and swelling.2 People living with TED often experience anxiety and depression, concerns about their facial appearance and a loss of confidence.3 This can impact their ability to work, socialise and maintain relationships.3,6.

影响英国约50,000人，TED是一种进行性的且可能威胁视力的疾病，可导致眼球突出、复视、眼痛、发红和肿胀。患有TED的人常常经历焦虑和抑郁，对面部外观的担忧以及自信心的丧失。这会影响他们的工作能力、社交和维持人际关系的能力。

“The marketing authorisation for teprotumumab as the first therapy specifically licensed for Thyroid Eye Disease (TED) in the UK marks a step forward for the patient community,” said Dr Tony Patrikios, Executive Medical Director, Amgen UK & Ireland. “TED can negatively affect patients’ lives impacting vision, causing eye pain, making everyday tasks difficult and causing a loss of self-confidence.

“特普妥单抗作为首个在英国获得甲状腺眼病 (TED) 特许治疗的药物，其营销授权标志着患者群体向前迈进了一步，”安进英国及爱尔兰执行医疗总监托尼·帕特里基奥斯博士表示。“TED 可能对患者的视力产生负面影响，导致眼部疼痛，使日常任务变得困难，并引发自信心丧失。”

This authorisation introduces a new alternative treatment option and reinforces Amgen’s commitment to supporting eligible patients with serious, underserved conditions.”.

该授权引入了一种新的替代治疗方案，并加强了安进公司对支持符合条件的患有严重且未满足需求疾病的患者的承诺。

Teprotumumab’s marketing authorisation in the UK is supported by multiple clinical studies,5,7,8,9 including the Phase 3 clinical trial, OPTIC (n=83).5 Other clinical studies that supported the marketing authorisation in the UK include the TED01RV Phase 2 clinical study (n=88),7 the HZNP-TEP-403 Phase 4 clinical study (n=62),8 and the OPTIC-J Phase 3 study in Japan (n=54).9 In the OPTIC trial, teprotumumab demonstrated a statistically significant improvement in the primary endpoint, proptosis (eye bulging) responder rate at 24 weeks, with 83% (34/41) of patients demonstrating a reduction of at least 2mm compared to baseline, versus 10% (4/42) of patients treated with placebo (difference of 73%, 95% confidence interval, 59 to 88; P=<0.001]).5.

特普妥单抗在英国的上市许可得到了多项临床研究的支持，5,7,8,9，其中包括3期临床试验OPTIC（n=83）。5 其他支持在英国上市许可的临床研究包括TED01RV 2期临床研究（n=88），7 HZNP-TEP-403 4期临床研究（n=62），8 以及在日本进行的OPTIC-J 3期研究（n=54）。9 在OPTIC试验中，特普妥单抗在主要终点——眼球突出（眼球鼓胀）反应率上显示出统计学显著改善，在24周时，83%（34/41）的患者眼球突出较基线减少至少2毫米，而安慰剂组仅有10%（4/42）的患者达到这一效果（差异为73%，95%置信区间为59至88；P=<0.001）。5

“For adult patients living with Thyroid Eye Disease (TED), it can be challenging to receive a diagnosis and referral to specialist centres. TED is often mistaken for other more common conditions, which can be frustrating and distressing for patients. This may also lead to delays in receiving treatment.

“对于患有甲状腺眼病 (TED) 的成年患者来说，获得诊断并被转诊到专科中心可能会面临挑战。TED 常常被误认为是其他更为常见的疾病，这对患者而言可能会带来沮丧和痛苦。这也可能导致治疗的延误。

When patients are diagnosed with TED, they are currently faced with limited treatment options targeting ‘generalised inflammation’. These treatments do not specifically target the underlying cause and drivers of TED,” said Dr Jimmy Uddin, Consultant Ophthalmologist, Oculoplastic & Orbital Surgeon, Moorfields Eye Hospital and St George’s Hospital Medical School.

当患者被诊断出患有TED时，他们目前面临的治疗选择有限，这些治疗针对的是“全身性炎症”。 这些治疗并未专门针对TED的根本原因和驱动因素，”摩尔菲尔德眼科医院和圣乔治医院医学院的顾问眼科医生、眼整形和眼眶外科医生吉米·乌丁博士说道。

“Teprotumumab offers eligible TED patients in the UK an important new treatment option.”.

“Teprotumumab 为英国符合条件的 TED 患者提供了一个重要的新治疗选择。”

The safety data for teprotumumab is based on multiple clinical studies.5,7,8,9 The most common side effects observed in clinical trials are muscle spasms (27.6%), diarrhoea (14.5%), hearing impairment (13.8%), alopecia (13.2%), hyperglycaemia (13.2%), fatigue (12.5%), nausea (10.5%), headache (10.5%), dry skin (9.9%), dysgeusia (8.6%), COVID-19 (6.6%), ear discomfort (6.6%) and nail disorder (5.9%).4.

特普他单抗的安全性数据基于多项临床研究。最常见的副作用包括肌肉痉挛 (27.6%)、腹泻 (14.5%)、听力障碍 (13.8%)、脱发 (13.2%)、高血糖 (13.2%)、疲劳 (12.5%)、恶心 (10.5%)、头痛 (10.5%)、皮肤干燥 (9.9%)、味觉障碍 (8.6%)、COVID-19 (6.6%)、耳部不适 (6.6%) 和指甲疾病 (5.9%)。

Amgen will work with the National Institute for Health and Care Excellence (NICE) to seek reimbursement of teprotumumab for all eligible patients.

安进将与英国国家卫生与临床优化研究所（NICE）合作，为所有符合条件的患者寻求替普罗单抗的报销。

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.

▼ 该药品受到额外监控。这将有助于快速识别新的安全信息。医疗专业人员被要求报告任何可疑的不良反应。

Notes to Editors

编辑须知

About Thyroid Eye Disease (TED)

关于甲状腺眼病（TED）

TED is a serious, progressive and potentially vision-threatening autoimmune disease.10 It often occurs in people living with Graves’ disease, but is a distinct disease that is caused by autoantibodies activating an insulin-like growth factor-1 receptor (IGF-1R)-mediated signalling complex on cells within the retro-orbital space.11,12 This leads to a cascade of negative effects, which may cause long-term, irreversible damage, including blindness.13,14 Early signs and symptoms of TED may include dry eyes and grittiness; redness, swelling and excessive tearing; eyelid retraction; proptosis; pressure and/or pain behind the eyes; and double vision.2.

TED 是一种严重的、进行性的且可能威胁视力的自身免疫性疾病。它常发生在患有格雷夫斯病的人群中，但是一种由自身抗体激活眼眶后空间内细胞上的胰岛素样生长因子-1 受体（IGF-1R）介导的信号复合物所引起的独立疾病。这会导致一系列负面效应，可能造成包括失明在内的长期不可逆损伤。TED 的早期症状可能包括眼睛干涩和异物感；红肿、过度流泪；眼睑退缩；眼球突出；眼后压力或疼痛；以及复视。

About teprotumumab

关于替妥木单抗

Teprotumumab is a monoclonal antibody that specifically targets and blocks the insulin-like growth factor-1 receptor (IGF-1R), which is thought to play a central role in the development of TED.15 It binds to the IGF-1R, preventing it from being activated and sending signals that lead to muscle and tissue expansion, as well as inflammation behind the eye.15 This can help reduce TED symptoms, such as proptosis (eye bulging).4,5.

特普妥单抗是一种单克隆抗体，专门靶向并阻断胰岛素样生长因子-1受体（IGF-1R），该受体被认为在甲状腺眼病（TED）的发展中起核心作用。它与IGF-1R结合，阻止其被激活并向导致肌肉和组织扩张以及眼后炎症的信号传导。这有助于减轻TED症状，如眼球突出（眼球鼓出）。

For further product information, please see the Summary of Product Characteristics, which will be available at: https://www.medicines.org.uk/emc

有关更多产品信息，请参阅产品特性摘要，该摘要将在以下网址提供：https://www.medicines.org.uk/emc

About the Phase 3 OPTIC clinical trial

关于第3期OPTIC临床试验

A prospective, multi-centre randomised, double-blind, placebo-controlled, Phase 3 trial of the IGF-1R inhibitor teprotumumab or placebo in adults with Graves’ disease and active moderate-to-severe thyroid eye disease.5

一项针对IGF-1R抑制剂替普鲁单抗或安慰剂在患有Graves病和活动性中重度甲状腺眼病成人中的前瞻性、多中心随机、双盲、安慰剂对照的3期临床试验。

83 patients between the ages of 18-80 were recruited and randomised 1:1 to receive 8 intravenous infusions of either teprotumumab or placebo. Infusions were administered every 3 weeks over 21 weeks, with a final clinical evaluation at week 24. The initial dose of teprotumumab was 10mg/kg, with each subsequent infusion dosed at 20mg/kg.5.

招募了83名18-80岁的患者，并以1:1的比例随机分配接受8次静脉注射，药物为替普罗单抗或安慰剂。每3周进行一次输注，持续21周，第24周进行最终临床评估。替普罗单抗的初始剂量为10mg/kg，随后每次输注剂量为20mg/kg。

The primary endpoint of the trial was proptosis response, defined as a reduction of at least 2mm in proptosis from baseline in the study eye without a corresponding increase of at least 2mm in the fellow (contra-lateral) eye at week 24.5

试验的主要终点是眼球突出反应，定义为在第24.5周时，研究眼的眼球突出度较基线减少至少2毫米，而对侧眼没有相应增加至少2毫米。

About Amgen UK

关于安进英国

Amgen’s mission is to serve patients. A biotechnology innovator since 1980, our science- based heritage is at the heart of everything we do. We remain on the cutting edge of innovation, using technology and human genetic data to push beyond what is known today. In the UK, Amgen is advancing a broad and deep pipeline that builds on its existing portfolio of medicines to treat cancer, heart disease, osteoporosis, inflammatory diseases and rare diseases.

Amgen的使命是服务患者。作为自1980年以来的生物技术先驱，我们以科学为基础的传统是我们一切工作的核心。我们始终站在创新的前沿，利用技术和人类基因数据突破当今已知的界限。在英国，Amgen正在推进广泛而深入的研发管线，依托其现有的药物组合，治疗癌症、心脏病、骨质疏松症、炎症性疾病和罕见疾病。

As a regional hub, we employ around 650 people in the UK and Ireland across our commercial, R&D and corporate functions. Committed to driving sustainable solutions that can adapt to an ever-evolving health system, we are proud to serve patients every day..

作为区域中心，我们在英国和爱尔兰的商业、研发和企业职能部门雇用了大约650人。我们致力于推动能够适应不断发展的医疗体系的可持续解决方案，我们为每天服务患者感到自豪。