WILMINGTON, Del.--(BUSINESS WIRE)--Positive results from the 24-week and long-term extension (LTE) period of the pivotal ALPHA Phase III trial showed danicopan as add-on to standard of care C5 inhibitor therapy ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) continued to demonstrate clinical benefit for patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH).1.

威尔明顿，Del。--（商业新闻）-关键的ALPHA III期试验的24周和长期延长（LTE）期的阳性结果显示，达尼科潘作为标准护理C5抑制剂治疗ULTOMIRIS®（ravulizumab cwvz）或SOLIRIS®（依库利珠单抗）的附加品，继续证明对阵发性夜间血红蛋白尿（PNH）患者具有临床意义血管外溶血（EVH）。

Results from the trial were presented today at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, California. Danicopan is an investigational, first-in-class, oral, Factor D inhibitor.

该试验的结果于今天在加利福尼亚州圣地亚哥举行的第65届美国血液学会（ASH）年会和博览会上公布。Danicopan是一种研究性的，一流的口服D因子抑制剂。

Data showed that improvements in mean hemoglobin levels and absolute reticulocyte count (ARC) levels, which were demonstrated at 12 weeks, were maintained through 48 weeks.1

数据显示，在12周时证实的平均血红蛋白水平和绝对网织红细胞计数（ARC）水平的改善维持了48周。1

PNH is a rare and severe blood disorder characterized by the destruction of red blood cells within blood vessels, known as intravascular hemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.2-4 Immediate, complete and sustained terminal complement inhibition by blocking the C5 protein with ULTOMIRIS or SOLIRIS helps reduce symptoms and complications, resulting in improved survival for patients with PNH.4-7 Approximately 10-20% of people living with PNH who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anemia and require blood transfusions.2,8-12.

PNH是一种罕见且严重的血液疾病，其特征是破坏血管内的红细胞，称为血管内溶血（IVH），白细胞和血小板活化，可引起血栓形成（血块），并导致器官损伤和潜在的过早死亡。2-4立即，通过用ULTOMIRIS或SOLIRIS阻断C5蛋白来完全和持续地抑制末端补体，有助于减轻症状和并发症，从而提高PNH患者的生存率。4-7大约10-20%接受C5抑制剂治疗的PNH患者经历了临床上显着的EVH，这可能导致贫血的持续症状，需要输血。2,8-12。

Austin Kulasekararaj, MD, Consultant Hematologist at King's College Hospital, London and investigator in the ALPHA trial, said: “These new data further demonstrate the potential of danicopan as add-on to ULTOMIRIS or SOLIRIS to address the needs of the small subset of patients with PNH who experience clinically significant EVH.

伦敦国王学院医院顾问血液学家、ALPHA试验研究者Austin Kulasekararaj医学博士说：“这些新数据进一步证明了达尼科班作为ULTOMIRIS或SOLIRIS的附加品的潜力，可以满足一小部分经历临床显着EVH的PNH患者的需求。

Expanding on positive 12-week results, the findings demonstrate sustained improvements in hemoglobin levels for up to 48 weeks, while also maintaining disease control, as measured by lactate dehydrogenase levels.”.

根据12周的阳性结果，研究结果表明血红蛋白水平持续改善长达48周，同时还可以通过乳酸脱氢酶水平来维持疾病控制。”。

Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: “Unlike IVH, EVH is not life-threatening, but its manifestations can be burdensome for people living with this condition, which is why we continue to explore the potential of the complement system to advance patient care.

亚历克赛（Alexion）高级副总裁、发展、监管和安全主管吉安卢卡·皮罗齐（Gianluca Pirozzi）表示：“与IVH不同，EVH不会危及生命，但其表现可能会给患有这种疾病的人带来负担，这就是为什么我们继续探索补体系统在推进患者护理方面的潜力。

The pivotal ALPHA results suggest that dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for the 10-20% of patients with PNH who experience clinically significant EVH. Importantly, C5 inhibition maintains effective IVH control, which is critical for patients, and the addition of Factor D inhibition addresses signs and symptoms of EVH.”.

关键的α结果表明，因子D和C5的双重补体途径抑制可能是10-20%经历临床显着EVH的PNH患者的最佳治疗方法。重要的是，C5抑制可维持有效的IVH控制，这对患者至关重要，而D因子抑制的加入可解决EVH的体征和症状。”。

The pivotal ALPHA Phase III trial is designed as a superiority study to evaluate the efficacy and safety of danicopan as an add-on to C5 inhibitor therapy ULTOMIRIS or SOLIRIS in patients with PNH who experience clinically significant EVH. A total of 86 patients were randomized. The prespecified interim analysis (primary analysis) occurred after 63 participants either completed or discontinued from the primary treatment period of 12 weeks.

关键的ALPHA III期临床试验旨在作为一项优越性研究，评估达尼科班作为C5抑制剂治疗ULTOMIRIS或SOLIRIS的附加药物对经历临床显着EVH的PNH患者的疗效和安全性。共有86名患者被随机分组。预先指定的中期分析（主要分析）发生在63名参与者完成或停止12周的主要治疗期后。

Following the 12-week randomized control period, patients were eligible to enroll in an open-label treatment period for an additional 12 weeks. During the open-label period, participants receiving placebo plus ULTOMIRIS or SOLIRIS switched to danicopan plus ULTOMIRIS or SOLIRIS (placebo-danicopan), and participants receiving add-on therapy with danicopan continued treatment with danicopan add-on therapy (danicopan-danicopan).

在12周的随机对照期后，患者有资格再参加12周的开放标签治疗期。在开放标签期间，接受安慰剂加ULTOMIRIS或SOLIRIS的参与者改用达尼科班加ULTOMIRIS或SOLIRIS（安慰剂-达尼科班），接受达尼科班附加治疗的参与者继续接受达尼科班附加治疗（达尼科班-达尼科班）。

The open-label treatment period was followed by the option to join a two-year LTE period during which all participants received danicopan add-on therapy. At the time of data cut-off on September 20, 2022, 60 of the 63 patients who were included in the primary analysis had reached 24 weeks and entered the LTE.1.

开放标签治疗期之后，可以选择加入为期两年的LTE期，在此期间所有参与者都接受了达尼科潘附加治疗。在2022年9月20日数据截止时，纳入初步分析的63名患者中有60名已达到24周并进入LTE。

Data showed that the significant improvements in hemoglobin levels observed at 12 weeks [LSM (SEM) change 2.94 (0.21) g/dL] continued at 24 weeks [LSM (SEM) change 3.17 (0.30) g/dL] among patients treated with danicopan plus ULTOMIRIS or SOLIRIS and were sustained through 48 weeks.1

数据显示，在接受达尼科潘联合ULTOMIRIS或SOLIRIS治疗的患者中，12周时观察到的血红蛋白水平显着改善[LSM（SEM）变化2.94（0.21）g/dL]在24周时持续[LSM（SEM）变化3.17（0.30）g/dL]，并持续48周

Secondary endpoints measured at 24 weeks include change from baseline in hemoglobin, ARC, and lactate dehydrogenase (LDH) levels; the percentage of patients with hemoglobin increase of ≥2 g/dL in the absence of transfusion; and the percentage of patients with transfusion avoidance.1

在24周时测量的次要终点包括血红蛋白，ARC和乳酸脱氢酶（LDH）水平与基线的变化；在没有输血的情况下，血红蛋白增加≥2 g/dL的患者百分比；以及避免输血的患者比例。1

All key secondary endpoints met superiority in favor of danicopan plus ULTOMIRIS or SOLIRIS compared to placebo plus ULTOMIRIS or SOLIRIS at 12 weeks, and data showed benefits were maintained at 24 weeks in the danicopan-danicopan arm.1

与安慰剂加ULTOMIRIS或SOLIRIS相比，所有关键的次要终点在12周时均优于安慰剂加ULTOMIRIS或SOLIRIS，数据显示，达尼科潘-达尼科潘组的益处维持在24周

Further, all key secondary endpoints showed meaningful improvement at 24 weeks in patients who switched from placebo to add-on treatment with danicopan at 12 weeks, including ARC levels and percentage of patients with transfusion avoidance, two indicators of potential EVH.1

此外，所有关键的次要终点在12周时从安慰剂转换为达尼科潘附加治疗的患者在24周时均显示出有意义的改善，包括ARC水平和避免输血患者的百分比，这是潜在EVH的两个指标

Additionally, mean (SD) LDH levels were maintained from baseline through 48 weeks in both treatment arms, demonstrating effective control of terminal complement activity and IVH with ULTOMIRIS or SOLIRIS.1

此外，两个治疗组的平均（SD）LDH水平从基线维持到48周，表明ULTOMIRIS或SOLIRIS有效控制了终末补体活性和IVH

Summary of efficacy resultsi

疗效结果总结i

Hemoglobin and ARC levels improved with danicopan at 12 weeks and were maintained through 48 weeks (please see hemoglobin and ARC level graphs included in the image carousel).

danicopan在12周时血红蛋白和ARC水平有所改善，并维持了48周（请参阅图像转盘中包含的血红蛋白和ARC水平图）。

Endpoints

端点

Statistic

统计

Danicopan-Danicopan

达尼科潘

Placebo-Danicopan

丹麦安慰剂

Change at

更改时间

12 weeks

12周

Change at

更改时间

24 weeks

24周

Change at

更改时间

12 weeks

12周

Change at

更改时间

24 weeks

24周

Change from baseline in hemoglobin (g/dL)

血红蛋白与基线的变化（g/dL）

LSM (SEM)

LSM（SEM）

2.94 (0.21)

2.94 (0.21)

3.17 (0.30)

3.17 (0.30)

0.50 (0.31)

0.50 (0.31)

2.26 (0.34)

2.26 (0.34)

Change from baseline in ARC levels (×109/L)

ARC水平与基线的变化（×109/L）

LSM (SEM)

LSM（SEM）

–83.8 (8.93)

–83.8 (8.93)

–80.2 (8.75)

–80.2 (8.75)

3.5 (12.68)

3.5 (12.68)

–65.2 (12.74)

–65.2 (12.74)

Change from baseline in LDH levels (U/L)

LDH水平与基线的变化（U/L）

LSM (SEM)

LSM（SEM）

–23.49 (8.29)

–23.49 (8.29)

–17.79 (13.73)

–17.79 (13.73)

–2.92 (11.91)

–2.92 (11.91)

–6.03 (18.77)

–6.03 (18.77)

Endpoints

端点

Statistic

统计

Danicopan-Danicopan

达尼科潘

Placebo-Danicopan

丹麦安慰剂

Percent at

百分比

12 weeks

12周

Percent at

百分比

24 weeks

24周

Percent at

百分比

12 weeks

12周

Percent at

百分比

24 weeks

24周

Proportion of participants with transfusion avoidance (%)

避免输血的参与者比例（%）

Percent (%)

百分比（%）

83

83

78

78

38

38

90

90

i. LDH, lactate dehydrogenase; ARC, absolute reticulocyte count; LSM, least squares mean; SEM, standard error of the mean; ULN, upper limit of normal.

i、 LDH，乳酸脱氢酶；ARC，绝对网织红细胞计数；LSM，最小二乘均值；SEM，平均值的标准误差；ULN，正常上限。

Results from the ALPHA Phase III trial and LTE showed danicopan is generally well tolerated, and no new safety concerns were identified. The safety analysis was performed using data from all participants who took at least one dose of danicopan (n=80). The most common treatment-emergent adverse events (TEAEs) (≥10%) were COVID-19 (21.3%), diarrhea (15%), headache (15%), pyrexia (13.8%), nausea (12.5%) and fatigue (10%).1.

阿尔法III期试验和LTE的结果显示，达尼科潘的耐受性通常良好，并且没有发现新的安全问题。使用来自服用至少一剂达尼科班（n=80）的所有参与者的数据进行安全性分析。最常见的治疗紧急不良事件（TEAE）（≥10%）为COVID-19（21.3%），腹泻（15%），头痛（15%），发热（13.8%），恶心（12.5%）和疲劳（10%）。

Additionally, an analysis of patient-reported outcomes from the ALPHA Phase III trial at 24 weeks was also presented at ASH, suggesting danicopan plus ULTOMIRIS or SOLIRIS has the potential to improve quality of life compared to C5 inhibitor therapy alone for the 10-20% of patients with PNH who experience clinically significant EVH.13 Findings showed clinically relevant patient-reported outcomes were observed in patients treated with danicopan as add-on to ULTOMIRIS or SOLIRIS during the first 12 weeks of treatment, compared to placebo plus C5 inhibition.

此外，ASH还对患者报告的24周ALPHA III期试验结果进行了分析，提示对于10-20%经历临床显着EVH的PNH患者，与单独使用C5抑制剂治疗相比，达尼科潘联合ULTOMIRIS或SOLIRIS有可能改善生活质量。13研究结果显示，在治疗的前12周内，达尼科潘作为ULTOMIRIS或SOLIRIS的附加品治疗的患者观察到了临床相关的患者报告结果，与安慰剂加C5抑制相比。

Additionally, data showed improvements in Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30) scores were maintained during the open-label period to 24 weeks in the danicopan-danicopan arm and improved at 24 weeks in the placebo-danicopan arm.13.

此外，数据显示，慢性疾病治疗疲劳（FACIT疲劳）的功能评估有所改善，欧洲癌症研究与治疗组织生活质量问卷核心30量表（EORTC-QLQ-C30）评分在开放标签期间维持至24周在danicopan-danicopan组中，在24周时在安慰剂danicopan组中得到改善。

ALPHA Phase III trial results from the primary prespecified interim analysis at 12 weeks were presented at the European Hematology Association (EHA) 2023 Hybrid Congress and published in The Lancet Haematology.

12周时主要预先指定的中期分析的ALPHA III期试验结果在欧洲血液学协会（EHA）2023年杂交大会上发表，并发表在《柳叶刀血液学》上。

Regulatory submissions for danicopan are currently under review with multiple global health authorities.

目前，多家全球卫生部门正在审查danicopan的监管提交。

INDICATION(S) & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab-cwvz)

ULTOMIRIS®（ravulizumab cwvz）的适应症和重要安全信息

What is ULTOMIRIS?

什么是ULTOMIRIS？

ULTOMIRIS is a prescription medicine used to treat:

ULTOMIRIS是一种处方药，用于治疗：

adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).

患有阵发性夜间血红蛋白尿（PNH）疾病的1个月及以上的成年人和儿童。

adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

患有非典型溶血性尿毒症综合征（aHUS）的成人和1个月及以上的儿童。ULTOMIRIS不用于治疗志贺毒素大肠杆菌相关溶血性尿毒症综合征（STEC-HUS）患者。

adults with a disease called generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

患有抗乙酰胆碱受体（AChR）抗体阳性的全身性重症肌无力（gMG）的成年人。

adults with PNH or aHUS when administered subcutaneously (under your skin).

当皮下（皮下）给药时，患有PNH或aHUS的成年人。

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

目前尚不清楚ULTOMIRIS对1个月以下的儿童是否安全有效。

It is not known if ULTOMIRIS is safe and effective for the treatment of gMG in children.

目前尚不清楚ULTOMIRIS是否安全有效地治疗儿童gMG。

Subcutaneous administration of ULTOMIRIS has not been evaluated and is not approved for use in children.

ULTOMIRIS的皮下给药尚未评估，也未批准用于儿童。

IMPORTANT SAFETY INFORMATION

重要安全信息

What is the most important information I should know about ULTOMIRIS?

关于ULTOMIRIS，我应该知道的最重要的信息是什么？

ULTOMIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.

ULTOMIRIS是一种影响免疫系统的药物，可以降低免疫系统抵抗感染的能力。

ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.

ULTOMIRIS会增加你患严重且危及生命的脑膜炎球菌感染的机会，如果不及早发现和治疗，这种感染可能会迅速危及生命并导致死亡。

You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.

如果您没有接种疫苗，您必须在第一剂ULTOMIRIS之前至少2周接种脑膜炎球菌疫苗。

If your healthcare provider decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.

如果您的医疗保健提供者决定需要紧急治疗ULTOMIRIS，您应该尽快接种脑膜炎球菌疫苗。

If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.

如果您没有接种疫苗，必须立即开始ULTOMIRIS治疗，您还应该在接种疫苗的同时接受2周的抗生素治疗。

If you had a meningococcal vaccine in the past, you might need additional vaccination. Your healthcare provider will decide if you need additional vaccination.

如果你过去接种过脑膜炎球菌疫苗，你可能需要额外接种疫苗。您的医疗保健提供者将决定您是否需要额外接种疫苗。

Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light..

脑膜炎球菌疫苗可以减少但不能预防所有脑膜炎球菌感染。如果你有脑膜炎球菌感染的任何体征和症状，请立即致电你的医疗保健提供者或获得紧急医疗护理：头痛伴恶心或呕吐，头痛伴发烧，头痛伴颈部僵硬或背部僵硬，发烧伴皮疹，精神错乱，肌肉酸痛伴流感样症状，眼睛对光敏感。。

Your healthcare provider will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any healthcare provider or nurse to help them diagnose and treat you quickly..

您的医疗保健提供者会给您一张关于脑膜炎球菌感染风险的患者安全卡。在治疗期间以及最后一次服用ULTOMIRIS后的8个月内，始终随身携带。向任何医疗保健提供者或护士出示此卡，以帮助他们快速诊断和治疗您，这一点很重要。。

ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your healthcare provider must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine.

ULTOMIRIS只能通过名为ULTOMIRIS REMS的程序获得。在您接受ULTOMIRIS之前，您的医疗保健提供者必须：注册ULTOMIRIS REMS计划；向您咨询脑膜炎球菌感染的风险；向您提供有关脑膜炎球菌感染症状和风险的信息和患者安全卡（如上所述）；并确保您接种了脑膜炎球菌疫苗，如果需要，请重新接种脑膜炎球菌疫苗。

Ask your healthcare provider if you are not sure if you need to be revaccinated..

如果您不确定是否需要重新接种疫苗，请咨询您的医疗保健提供者。。

ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS. Call your healthcare provider right away if you have any new signs or symptoms of infection..

ULTOMIRIS也可能增加其他类型严重感染的风险。如果用ULTOMIRIS治疗，确保您的孩子接种肺炎链球菌和b型流感嗜血杆菌（Hib）疫苗。如果您有任何新的感染迹象或症状，请立即致电您的医疗保健提供者。。

Who should not receive ULTOMIRIS?

谁不应该接受ULTOMIRIS？

Do not receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your healthcare provider decides that urgent treatment with ULTOMIRIS is needed.

如果您患有脑膜炎球菌感染或未接种脑膜炎球菌感染疫苗，请不要服用ULTOMIRIS，除非您的医疗保健提供者决定需要紧急治疗ULTOMIRIS。

Before you receive ULTOMIRIS, tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS will harm your unborn baby or if it passes into your breast milk.

在您接受ULTOMIRIS之前，请告知您的医疗保健提供者您的所有医疗状况，包括您是否感染或发烧，是否怀孕或计划怀孕，是否正在母乳喂养或计划母乳喂养。目前尚不清楚ULTOMIRIS是否会伤害您未出生的婴儿或是否会进入您的母乳。

You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS..

在治疗期间以及服用ULTOMIRIS后的8个月内，您不应进行母乳喂养。。

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

告诉你的医疗保健提供者你接种的所有疫苗和服用的药物，包括处方药和非处方药、维生素和草药补充剂，这些都可能影响你的治疗。

If you have PNH and you stop receiving ULTOMIRIS, your healthcare provider will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in your red blood cell count, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males..

如果您患有PNH并且停止服用ULTOMIRIS，您停止服用ULTOMIRIS后，您的医疗保健提供者将需要对您进行至少16周的密切监测。停止ULTOMIRIS可能会导致PNH导致红细胞分解。红细胞分解可能产生的症状或问题包括：男性红细胞计数下降、疲劳、尿液中有血、腹部（腹部）疼痛、呼吸急促、血块、吞咽困难和勃起功能障碍（ED）。。

If you have aHUS, your healthcare provider will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include: confusion or loss of consciousness, seizures, chest pain (angina), difficulty breathing and blood clots or stroke..

如果您患有aHUS，在停止治疗aHUS恶化的迹象或与一种异常凝血和红细胞分解（称为血栓性微血管病（TMA））相关的问题后，您的医疗保健提供者将需要对您进行至少12个月的密切监测。TMA可能出现的症状或问题可能包括：意识混乱或丧失，癫痫发作，胸痛（心绞痛），呼吸困难，血栓或中风。。

ULTOMIRIS can cause serious side effects including allergic reactions to acrylic adhesive. Allergic reactions to the acrylic adhesive may happen with your subcutaneous ULTOMIRIS treatment. If you have an allergic reaction during the delivery of subcutaneous ULTOMIRIS, remove the on-body injector and get medical help right away.

ULTOMIRIS会引起严重的副作用，包括对丙烯酸粘合剂的过敏反应。皮下ULTOMIRIS治疗可能会对丙烯酸粘合剂产生过敏反应。如果您在皮下注射ULTOMIRIS期间出现过敏反应，请取出体内注射器并立即寻求医疗帮助。

Your healthcare provider may treat you with medicines to help prevent or treat allergic reaction symptoms as needed..

您的医疗保健提供者可能会根据需要为您提供药物，以帮助预防或治疗过敏反应症状。。

What are the possible side effects of ULTOMIRIS?

ULTOMIRIS可能有哪些副作用？

ULTOMIRIS can cause serious side effects including infusion-related reactions. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, tiredness, feeling faint, discomfort in your arms or legs, bad taste, or drowsiness. Stop treatment of ULTOMIRIS and tell your healthcare provider or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out..

ULTOMIRIS可引起严重的副作用，包括输液相关反应。ULTOMIRIS输液相关反应的症状可能包括腰痛、疲倦、头晕、手臂或腿部不适、味觉不佳或嗜睡。停止ULTOMIRIS的治疗，如果您在ULTOMIRIS输注过程中出现这些症状或任何其他症状，可能意味着您有严重的输注反应，包括：胸痛，呼吸困难或呼吸急促，面部，舌头或喉咙肿胀，感觉昏厥或昏厥，请立即告诉您的医疗保健提供者或护士。。

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory tract infection and headache.

ULTOMIRIS在PNH患者中最常见的副作用是上呼吸道感染和头痛。

The most common side effects of ULTOMIRIS in people treated for aHUS are upper respiratory tract infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.

在接受aHUS治疗的人群中，ULTOMIRIS最常见的副作用是上呼吸道感染，腹泻，恶心，呕吐，头痛，高血压和发烧。

The most common side effects of ULTOMIRIS in people with gMG are diarrhea and upper respiratory tract infections.

ULTOMIRIS在gMG患者中最常见的副作用是腹泻和上呼吸道感染。

The most common side effects of subcutaneous administration of ULTOMIRIS in adults treated for PNH and aHUS are local injection site reactions.

在接受PNH和aHUS治疗的成年人中，皮下注射ULTOMIRIS最常见的副作用是局部注射部位反应。

Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider right away if you miss an ULTOMIRIS infusion or for medical advice about side effects.

告诉你的医疗保健提供者任何困扰你或不会消失的副作用。这些并不是ULTOMIRIS可能产生的所有副作用。有关更多信息，请咨询您的医疗保健提供者或药剂师。如果您错过了ULTOMIRIS输液或有关副作用的医疗建议，请立即致电您的医疗保健提供者。

You may report side effects to FDA at 1-800-FDA-1088..

您可以通过1-800-FDA-1088向FDA报告副作用。。

Read the Instructions for Use that comes with subcutaneous ULTOMIRIS for instructions about the right way to prepare and give your subcutaneous ULTOMIRIS injections through an on-body injector.

阅读皮下注射ULTOMIRIS的使用说明，了解如何正确准备和通过体内注射器注射皮下注射ULTOMIRIS。

Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis. Please see the accompanying Instructions for Use for the ULTOMIRIS On Body Delivery System.

请参阅随附的ULTOMIRIS完整处方信息和药物指南，包括关于严重和危及生命的脑膜炎球菌感染/败血症的盒装警告。请参阅随附的ULTOMIRIS On Body传送系统使用说明。

INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab) [injection for intravenous use 300mg/30mL vial]

SOLIRIS®（依库丽珠单抗）[静脉注射用300mg/30mL小瓶]的适应症和重要安全信息

What is SOLIRIS?

什么是SOLIRIS？

SOLIRIS is a prescription medicine used to treat:

SOLIRIS是一种处方药，用于治疗：

patients with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).

患有阵发性夜间血红蛋白尿症（PNH）的患者。

adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

患有非典型溶血性尿毒症综合征（aHUS）的成人和儿童。SOLIRIS不用于治疗志贺毒素大肠杆菌相关溶血性尿毒症综合征（STEC-HUS）患者。

adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

患有抗乙酰胆碱受体（AChR）抗体阳性的全身性重症肌无力（gMG）的成年人。

adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

患有抗水通道蛋白4（AQP4）抗体阳性的视神经脊髓炎谱系障碍（NMOSD）疾病的成年人。

It is not known if SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.

目前尚不清楚SOLIRIS对PNH，gMG或NMOSD儿童是否安全有效。

IMPORTANT SAFETY INFORMATION

重要安全信息

What is the most important information I should know about SOLIRIS?

关于SOLIRIS，我应该知道的最重要的信息是什么？

SOLIRIS is a medicine that affects your immune system and can lower the ability of your immune system to fight infections.

SOLIRIS是一种影响免疫系统的药物，可以降低免疫系统抵抗感染的能力。

SOLIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.

如果不及早发现和治疗，SOLIRIS会增加你患严重且危及生命的脑膜炎球菌感染的机会，这种感染可能会迅速危及生命并导致死亡。

You must receive meningococcal vaccines at least 2 weeks before your first dose of SOLIRIS if you are not vaccinated.

如果您没有接种疫苗，您必须在第一剂SOLIRIS之前至少2周接种脑膜炎球菌疫苗。

If your doctor decided that urgent treatment with SOLIRIS is needed, you should receive meningococcal vaccination as soon as possible.

如果你的医生决定需要紧急治疗，你应该尽快接种脑膜炎球菌疫苗。

If you have not been vaccinated and SOLIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.

如果您没有接种疫苗，必须立即开始SOLIRIS治疗，您还应该在接种疫苗的同时接受2周的抗生素治疗。

If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.

如果你过去接种过脑膜炎球菌疫苗，你可能需要额外接种疫苗。你的医生将决定你是否需要额外接种疫苗。

Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms, and eyes sensitive to light..

脑膜炎球菌疫苗可以减少但不能预防所有脑膜炎球菌感染。如果你有脑膜炎球菌感染的任何体征和症状，请立即致电医生或接受紧急医疗护理：头痛伴恶心或呕吐，头痛伴发烧，头痛伴颈部僵硬或背部僵硬，发烧伴皮疹，精神错乱，肌肉酸痛伴流感样症状，眼睛对光线敏感。。

Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 3 months after your last SOLIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

你的医生会给你一张关于脑膜炎球菌感染风险的患者安全卡。在治疗期间以及最后一次服用SOLIRIS后的3个月内，始终随身携带。向任何医生或护士出示这张卡片都很重要，以帮助他们快速诊断和治疗您。

SOLIRIS is only available through a program called the SOLIRIS REMS. Before you can receive SOLIRIS, your doctor must enroll in the SOLIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with the meningococcal vaccine and, if needed, get revaccinated with the meningococcal vaccine.

SOLIRIS只能通过名为SOLIRIS REMS的程序获得。在您接受SOLIRIS之前，您的医生必须注册SOLIRIS REMS计划；向您咨询脑膜炎球菌感染的风险；向您提供有关脑膜炎球菌感染症状和风险的信息和患者安全卡（如上所述）；并确保您接种了脑膜炎球菌疫苗，如果需要，请重新接种脑膜炎球菌疫苗。

Ask your doctor if you are not sure if you need to be revaccinated..

如果不确定是否需要重新接种疫苗，请咨询医生。。

SOLIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with SOLIRIS. Certain people may be at risk of serious infections with gonorrhea. Certain fungal infections (Aspergillus) may occur if you take SOLIRIS and have a weak immune system or a low white blood cell count..

SOLIRIS还可能增加其他类型严重感染的风险。如果用SOLIRIS治疗，确保您的孩子接种肺炎链球菌和b型流感嗜血杆菌（Hib）疫苗。某些人可能有严重感染淋病的风险。如果服用SOLIRIS且免疫系统较弱或白细胞计数低，可能会发生某些真菌感染（曲霉）。。

Who should not receive SOLIRIS?

谁不应该接收SOLIRIS？

Do not receive SOLIRIS if you have a meningococcal infection or have not been vaccinated against meningitis infection unless your doctor decides that urgent treatment with SOLIRIS is needed.

如果您患有脑膜炎球菌感染或未接种脑膜炎疫苗，请不要服用SOLIRIS，除非您的医生决定需要紧急治疗SOLIRIS。

Before you receive SOLIRIS, tell your doctor about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed. It is not known if SOLIRIS will harm your unborn baby or if it passes into your breast milk..

在您接受SOLIRIS之前，请告诉您的医生您的所有医疗状况，包括您是否感染或发烧，是否怀孕或计划怀孕，是否正在母乳喂养或计划母乳喂养。目前尚不清楚SOLIRIS是否会伤害您未出生的婴儿或是否会进入您的母乳。。

Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment. It is important that you have all recommended vaccinations before you start SOLIRIS, receive 2 weeks of antibiotics if you immediately start SOLIRIS, and stay up-to-date with all recommended vaccinations during treatment with SOLIRIS..

告诉你的医生你接种的所有疫苗和服用的药物，包括处方药和非处方药、维生素和草药补充剂，这些都可能影响你的治疗。重要的是，在开始使用SOLIRIS之前，您必须接种所有推荐的疫苗，如果您立即开始使用SOLIRIS，请接受2周的抗生素治疗，并在使用SOLIRIS治疗期间随时了解所有推荐的疫苗接种情况。。

If you have PNH, your doctor will need to monitor you closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of your red blood cell count, drop in your platelet count, confusion, kidney problems, blood clots, difficulty breathing, and chest pain..

如果您患有PNH，停止SOLIRIS后，您的医生需要对您进行至少8周的密切监测。停止使用SOLIRIS治疗可能会导致PNH导致红细胞分解。红细胞分解可能产生的症状或问题包括：红细胞计数下降、血小板计数下降、精神错乱、肾脏问题、血块、呼吸困难和胸痛。。

If you have aHUS, your doctor will need to monitor you closely during and for at least 12 weeks after stopping treatment for signs of worsening aHUS symptoms or problems related to abnormal clotting (thrombotic microangiopathy). Symptoms or problems that can happen with abnormal clotting may include: stroke, confusion, seizure, chest pain (angina), difficulty breathing, kidney problems, swelling in arms or legs, and a drop in your platelet count..

如果您患有aHUS，您的医生需要在停止治疗后至少12周内密切监测您的aHUS症状恶化的迹象或与异常凝血（血栓性微血管病）相关的问题。异常凝血可能出现的症状或问题可能包括：中风、精神错乱、癫痫发作、胸痛（心绞痛）、呼吸困难、肾脏问题、手臂或腿部肿胀以及血小板计数下降。。

What are the possible side effects of SOLIRIS?

SOLIRIS可能有哪些副作用？

SOLIRIS can cause serious side effects including serious infusion-related reactions. Tell your doctor or nurse right away if you get any of these symptoms during your SOLIRIS infusion: chest pain; trouble breathing or shortness of breath; swelling of your face, tongue, or throat; and feel faint or pass out.

SOLIRIS会引起严重的副作用，包括严重的输液相关反应。如果您在输注SOLIRIS期间出现以下任何症状，请立即告诉您的医生或护士：胸痛；呼吸困难或呼吸急促；面部、舌头或喉咙肿胀；感觉昏倒或昏倒。

If you have an infusion-related reaction to SOLIRIS, your doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS..

如果您对SOLIRIS有输注相关反应，您的医生可能需要更缓慢地输注SOLIRIS，或停止SOLIRIS。。

The most common side effects in people with PNH treated with SOLIRIS include: headache, pain or swelling of your nose or throat (nasopharyngitis), back pain, and nausea.

用SOLIRIS治疗的PNH患者最常见的副作用包括：头痛，鼻子或喉咙疼痛或肿胀（鼻咽炎），背痛和恶心。

The most common side effects in people with aHUS treated with SOLIRIS include: headache, diarrhea, high blood pressure (hypertension), common cold (upper respiratory infection), stomach-area (abdominal) pain, vomiting, pain or swelling of your nose or throat (nasopharyngitis), low red blood cell count (anemia), cough, swelling of legs or feet (peripheral edema), nausea, urinary tract infections, and fever..

使用SOLIRIS治疗的aHUS患者最常见的副作用包括：头痛，腹泻，高血压（高血压），普通感冒（上呼吸道感染），胃区（腹部）疼痛，呕吐，鼻子或喉咙疼痛或肿胀（鼻咽炎），红细胞计数低（贫血），咳嗽，腿或脚肿胀（外周水肿），恶心，尿路感染，还有发烧。。

The most common side effects in people with gMG treated with SOLIRIS include: muscle and joint (musculoskeletal) pain.

用SOLIRIS治疗的gMG患者最常见的副作用包括：肌肉和关节（肌肉骨骼）疼痛。

The most common side effects in people with NMOSD treated with SOLIRIS include: common cold (upper respiratory infection); pain or swelling of your nose or throat (nasopharyngitis); diarrhea; back pain; dizziness; flu-like symptoms (influenza), including fever, headache, tiredness, cough, sore throat, and body aches; joint pain (arthralgia); throat irritation (pharyngitis); and bruising (contusion)..

用SOLIRIS治疗的NMOSD患者最常见的副作用包括：普通感冒（上呼吸道感染）；鼻子或喉咙疼痛或肿胀（鼻咽炎）；腹泻；背痛；头晕；流感样症状（流感），包括发烧，头痛，疲倦，咳嗽，喉咙痛和身体疼痛；关节痛（关节痛）；咽喉刺激（咽炎）；和瘀伤。。

Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of SOLIRIS. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA.

告诉你的医生任何困扰你或不会消失的副作用。这些并不是SOLIRIS所有可能的副作用。有关更多信息，请咨询您的医生或药剂师。打电话给你的医生，询问有关副作用的医疗建议。鼓励您向FDA报告处方药的负面副作用。

Visit MedWatch, or call 1-800-FDA-1088..

访问MedWatch，或致电1-800-FDA-1088。。

Please see the full Prescribing Information and Medication Guide for SOLIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections.

请参阅SOLIRIS的完整处方信息和药物指南，包括关于严重和危及生命的脑膜炎球菌感染的盒装警告。

Notes

注意事项

PNH

PNH

PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterized by red blood cell destruction within blood vessels (also known as intravascular hemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots).2-4

PNH是一种罕见的，慢性的，进行性的，可能危及生命的血液疾病。它的特征是血管内的红细胞破坏（也称为血管内溶血）和白细胞和血小板活化，这可能导致血栓形成（血块）。2-4

PNH is caused by an acquired genetic mutation that may happen any time after birth and results in the production of abnormal blood cells that are missing important protective blood cell surface proteins. These missing proteins enable the complement system, which is part of the immune system and is essential to the body’s defense against infection, to ‘attack’ and destroy or activate these abnormal blood cells.2 Living with PNH can be debilitating, and signs and symptoms may include blood clots, abdominal pain, difficulty swallowing, erectile dysfunction, shortness of breath, excessive fatigue, anemia and dark-colored urine.2,10,14.

PNH是由获得性基因突变引起的，这种突变可能在出生后的任何时间发生，并导致产生异常的血细胞，而这些血细胞缺少重要的保护性血细胞表面蛋白。这些缺失的蛋白质使补体系统（免疫系统的一部分，对身体抵抗感染至关重要）能够“攻击”并破坏或激活这些异常血细胞。2患有PNH可能会使人衰弱，体征和症状可能包括血栓，腹痛，吞咽困难，勃起功能障碍，呼吸急促，过度疲劳，贫血和深色尿液。2,10,14。

Clinically Significant EVH

具有临床意义的EVH

EVH, the removal of red blood cells outside of the blood vessels, can sometimes occur in PNH patients who are treated with C5 inhibitors.15,16 Since C5 inhibition enables PNH red blood cells to survive and circulate, EVH may occur when these now surviving PNH red blood cells are marked by proteins in the complement system for removal by the spleen and liver.2,4,6 Patients with PNH with EVH may continue to experience anemia, which can have various causes, and may require blood transfusions.15-18 A small subset of people living with PNH who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anemia and require blood transfusions.2,10-12.

EVH，即去除血管外的红细胞，有时可能发生在接受C5抑制剂治疗的PNH患者中。15,16由于C5抑制使PNH红细胞能够存活和循环，当这些现在存活的PNH红细胞被补体系统中的蛋白质标记以被脾脏和肝脏去除时，可能会发生EVH。2,4,6患有EVH的PNH患者可能会继续出现贫血，这可能有多种原因，可能需要输血。15-18一小部分接受C5抑制剂治疗的PNH患者会出现临床上显着的EVH，这可能导致贫血的持续症状，需要输血。2,10-12。

ALPHA

阿尔法

ALPHA is a pivotal, global Phase III trial designed as a superiority study to evaluate the efficacy and safety of danicopan as an add-on to C5 inhibitor therapy eculizumab or ravulizumab-cwvz in patients with PNH who experience clinically significant EVH. In the double-blind, placebo-controlled, multiple-dose trial, patients were enrolled and randomized to receive danicopan or placebo (2:1) in addition to their ongoing eculizumab or ravulizumab-cwvz therapy for 12 weeks.

ALPHA是一项关键的全球III期临床试验，旨在作为一项优越性研究，评估达尼科班作为C5抑制剂治疗依库利珠单抗或ravulizumab cwvz的附加药物在经历临床显着EVH的PNH患者中的疗效和安全性。在双盲，安慰剂对照，多剂量试验中，除了持续的依库利珠单抗或ravulizumab cwvz治疗12周外，患者被纳入并随机接受达尼科班或安慰剂（2:1）。

A prespecified interim analysis was performed once 63 randomized patients had completed 12 weeks of the primary evaluation period or discontinued treatment as of June 28, 2022. At 12 weeks, patients on placebo plus a C5 inhibitor were switched to danicopan plus eculizumab or ravulizumab-cwvz, and patients on danicopan plus eculizumab or ravulizumab-cwvz remained on treatment for an additional 12 weeks.

截至2022年6月28日，63名随机患者完成了12周的主要评估期或停止治疗后，进行了预先指定的中期分析。在12周时，安慰剂加C5抑制剂的患者改用达尼科班加依库珠单抗或ravulizumab cwvz，达尼科班加依库珠单抗或ravulizumab cwvz的患者继续治疗12周。

Patients who completed both treatment periods (24 weeks) had the option to participate in a two-year long-term extension period and continue to receive danicopan in addition to eculizumab or ravulizumab-cwvz. The open-label period of the study is still ongoing.5,19.

完成两个治疗期（24周）的患者可以选择参加为期两年的长期延长期，除依库利珠单抗或ravulizumab cwvz外，还可以继续接受达尼科班治疗。该研究的开放标签期仍在进行中[5,19]。

Danicopan

达尼科潘

Danicopan is an investigational oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab-cwvz for patients with PNH who experience clinically significant EVH. It is designed to selectively inhibit Factor D, a complement system protein that plays a key role in the amplification of the complement system response.

Danicopan是一种正在开发的研究性口服药物，作为C5抑制剂治疗依库利珠单抗或ravulizumab cwvz的附加药物，用于经历临床显着EVH的PNH患者。它被设计用于选择性抑制因子D，这是一种补体系统蛋白，在补体系统反应的扩增中起关键作用。

Danicopan has been granted Breakthrough Therapy designation by the US Food and Drug Administration and PRIority MEdicines (PRIME) status by the European Medicines Agency. Danicopan has also been granted Orphan Drug Designation in the US, EU and Japan for the treatment of PNH. Alexion is also evaluating danicopan as a potential monotherapy for geographic atrophy in a Phase II clinical trial..

Danicopan已被美国食品和药物管理局授予突破性治疗称号，并被欧洲药品管理局授予优先药物（PRIME）地位。Danicopan还被美国、欧盟和日本授予孤儿药称号，用于治疗PNH。Alexion还在一项II期临床试验中评估了达尼科班作为治疗地理萎缩的潜在单一疗法。。

ULTOMIRIS® (ravulizumab-cwvz)

到期®（ravulizumab cwvz）

ULTOMIRIS® (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells.

ULTOMIRIS®（ravulizumab cwvz）是第一种也是唯一一种长效C5补体抑制剂，可立即，完全和持续地抑制补体。该药物通过抑制末端补体级联（人体免疫系统的一部分）中的C5蛋白起作用。当以不受控制的方式激活时，补体级联反应过度，导致身体攻击自己的健康细胞。

ULTOMIRIS is administered intravenously every eight weeks in adult patients, following a loading dose..

ULTOMIRIS在成年患者中每八周静脉注射一次，服用负荷剂量。。

ULTOMIRIS is approved in the US, EU and Japan for the treatment of certain adults with generalized myasthenia gravis (gMG).

ULTOMIRIS在美国、欧盟和日本被批准用于治疗某些患有全身性重症肌无力（gMG）的成年人。

ULTOMIRIS is also approved in the US, EU and Japan for the treatment of certain adults with PNH and for certain children with PNH in the US and EU.

ULTOMIRIS在美国，欧盟和日本也被批准用于治疗美国和欧盟的某些PNH成人和某些PNH儿童。

Additionally, ULTOMIRIS is approved in the US, EU and Japan for certain adults and children with atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy (aHUS).

此外，ULTOMIRIS在美国，欧盟和日本被批准用于某些患有非典型溶血性尿毒症综合征的成人和儿童，以抑制补体介导的血栓性微血管病（aHUS）。

Further, ULTOMIRIS is approved in the EU and Japan for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

此外，ULTOMIRIS在欧盟和日本被批准用于治疗某些患有视神经脊髓炎谱系障碍（NMOSD）的成年人。

As part of a broad development program, ULTOMIRIS is being assessed for the treatment of additional hematology and neurology indications.

作为广泛发展计划的一部分，ULTOMIRIS正在评估其他血液学和神经学适应症的治疗。

SOLIRIS® (eculizumab)

SOLIRIS®（eculizumab）

SOLIRIS® (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells.

SOLIRIS®（依库丽单抗）是一流的C5补体抑制剂。该药物通过抑制末端补体级联（人体免疫系统的一部分）中的C5蛋白起作用。当以不受控制的方式激活时，末端补体级联反应过度，导致身体攻击自己的健康细胞。

SOLIRIS is administered intravenously every two weeks, following an introductory dosing period..

在开始给药后，每两周静脉注射一次SOLIRIS。。

SOLIRIS is approved in the US, EU, Japan and China for the treatment of patients with PNH and aHUS.

SOLIRIS在美国、欧盟、日本和中国被批准用于治疗PNH和aHUS患者。

Additionally, SOLIRIS is approved in Japan and the EU for the treatment of certain adult and pediatric patients with gMG and in the US and China for certain adults with gMG.

此外，SOLIRIS在日本和欧盟被批准用于治疗某些成人和儿科gMG患者，在美国和中国被批准用于治疗某些成人gMG患者。

Further, SOLIRIS is approved in the US, EU, Japan and China for the treatment of certain adults with NMOSD.

此外，SOLIRIS在美国，欧盟，日本和中国被批准用于治疗某些患有NMOSD的成年人。

SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome.

SOLIRIS不适用于治疗志贺毒素大肠杆菌相关溶血性尿毒症综合征患者。

Alexion

亚历克西斯

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for more than 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines.

亚力兄（AstraZeneca Rare Disease）是阿斯利康旗下专注于罕见疾病的集团，于2021年收购亚力兄制药公司（Alexion Pharmaceuticals，Inc.）后成立。亚力兄作为罕见疾病的领导者已有30多年的历史，通过这一发现，亚力兄专注于为受罕见疾病和破坏性疾病影响的患者和家庭提供服务，改变生命的药物的开发和商业化。

Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.

亚力兄的研究重点是补体级联反应中的新分子和靶标，以及血液学、肾脏病、神经病学、代谢紊乱、心脏病学和眼科的发展努力。Alexion总部位于马萨诸塞州波士顿，在全球设有办事处，为50多个国家的患者提供服务。

For more information, please visit www.alexion.com..

欲了解更多信息，请访问www.alexion.com。。

AstraZeneca

阿斯利康

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

阿斯利康（LSE/STO/Nasdaq:AZN）是一家全球科学领先的生物制药公司，专注于肿瘤学，罕见病和生物制药（包括心血管，肾脏和代谢以及呼吸和免疫学）处方药的发现，开发和商业化。

Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit www.astrazeneca-us.com and follow the Company on social media @AstraZeneca..

阿斯利康总部位于英国剑桥，在100多个国家运营，其创新药物被全球数百万患者使用。请访问www.astrazeneca-us.com并在社交媒体@astrazeneca上关注公司。。

References

参考文献

Kulasekararaj, AG, et al. Danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: phase 3 long-term data. Presented at: American Society of Hematology (ASH) Congress; December 9-12, 2023; San Diego, California.

Kulasekaraj，AG，et al。Danicopan作为ravulizumab或依库利珠单抗与安慰剂的附加疗法治疗阵发性夜间血红蛋白尿和临床上显着的血管外溶血患者：3期长期数据。出席：美国血液学会（ASH）大会；2023年12月9日至12日；加利福尼亚州圣地亚哥。

Oral Session 508..

口头会议508。。

Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811.

布罗茨基RA。阵发性夜间血红蛋白尿。血。2014年；124（18）：2804-2811。

Griffin M, et al. Significant hemolysis is not required for thrombosis in paroxysmal nocturnal hemoglobinuria. Haematologica. 2019;104(3):e94-e96.

Griffin M等人。阵发性夜间血红蛋白尿的血栓形成不需要显着的溶血。血液学。2019年；104（3）：e94-e96。

Hillmen P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233-1243.

Hillmen P等人。补体抑制剂依库利珠单抗治疗阵发性夜间血红蛋白尿。英格兰医学杂志2006；355（12）：1233-1243。

Lee JW, et al. The role of the alternative pathway in paroxysmal nocturnal hemoglobinuria and emerging treatments. Expert Rev Clin Pharmacol. 2022;15(7):851-861.

Lee JW等人。替代途径在阵发性夜间血红蛋白尿和新兴治疗中的作用。专家Rev Clin Pharmacol。2022年；15（7）：851-861。

Kulasekararaj AG, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022;109(3):205-214.

Kulasekararaj AG等人。ravulizumab治疗阵发性夜间血红蛋白尿患者的长期安全性和有效性：两项关键的3期研究的2年结果。欧洲血液学杂志。2022年；109（3）：205-214。

Kulasekararaj AG, et al. P812: Long-term complement inhibition and survival outcomes in Patients with paroxysmal nocturnal hemoglobinuria: an interim analysis of the ravulizumab clinical trials. HemaSphere. 2022;6(Suppl):706-707.

Kulasekararaj AG等人，P812：阵发性夜间血红蛋白尿患者的长期补体抑制和生存结果：ravulizumab临床试验的中期分析。血球。2022年；6（补充）：706-707。

Lee JW, et al. Danicopan, a first-in-class oral complement factor D inhibitor, as add-on treatment to ravulizumab or eculizumab improves hemoglobin response versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis. Presented at: European Hematology Association (EHA) Hybrid Congress.

Lee JW等人，Danicopan是一种一流的口服补体因子D抑制剂，作为ravulizumab或依库利珠单抗的附加治疗，可改善阵发性夜间血红蛋白尿和临床上显着的血管外溶血患者的血红蛋白反应。发表于：欧洲血液学协会（EHA）混合大会。

June 8-11, 2023; Frankfurt, Germany. P771..

2023年6月8日至11日；德国法兰克福。P771页。。

Kulasekararaj AG, et al. Prevalence of clinically significant extravascular hemolysis in stable C5 inhibitor-treated patients with PNH and its association with disease control, quality of life and treatment satisfaction. Presented at: European Hematology Association (EHA) Hybrid Congress. June 8-11, 2023; Frankfurt, Germany.

Kulasekaraj AG等人。稳定的C5抑制剂治疗的PNH患者中临床上显着的血管外溶血的患病率及其与疾病控制，生活质量和治疗满意度的关系。发表于：欧洲血液学协会（EHA）混合大会。2023年6月8日至11日；德国法兰克福。

Abs PB2056..

Abs PB2056。。

Kulasekararaj AG, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540–549.

Kulasekaraj AG等人，在C5抑制剂经历过的成年PNH患者中，Ravulizumab（ALXN1210）与依库利珠单抗的比较：302研究。血。2019年；133（6）：540-549。

Lee JW, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530-539.

Lee JW等人。Ravulizumab（ALXN1210）与依库利珠单抗在未接受补体抑制剂治疗的PNH成年患者中的比较：301研究。血。2019年；133（6）：530-539。

Röth A, et al. Transfusion requirements in adult patients with paroxysmal nocturnal hemoglobinuria naive to complement inhibitors receiving ravulizumab and eculizumab: results from a phase 3 non-inferiority study [abstract]. ECTH 2019. Glasgow, UK ed. Glasgow, UK2019.

Röth A等人。接受ravulizumab和依库利珠单抗治疗的阵发性夜间血红蛋白尿未经补充抑制剂的成年患者的输血要求：3期非劣效性研究的结果[摘要]。ECTH 2019年。英国格拉斯哥编辑，英国格拉斯哥2019。

Piatek C, et al. Patient-reported outcomes: danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis. Presented at: American Society of Hematology (ASH) Congress; December 9-12, 2023; San Diego, CA.

Piatek C等人。患者报告的结果：在阵发性夜间血红蛋白尿和临床上显着的血管外溶血患者中，达尼科班作为ravulizumab或依库利珠单抗与安慰剂的附加疗法。出席：美国血液学会（ASH）大会；2023年12月9日至12日；加利福尼亚州圣地亚哥。

Abs 1346..

Abs 1346。。

Hillmen P, et al. Effect of the complement inhibitor eculizumab on thromboembolism on patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-4128.

Hillmen P等人。补体抑制剂依库利珠单抗对阵发性夜间血红蛋白尿患者血栓栓塞的影响。血。2007年；110（12）：4123-4128。

Brodsky RA. A complementary new drug for PNH. Blood. 2020;135(12):884–885.

布罗茨基RA。PNH的补充新药。血。2020年；135（12）：884-885。

Risitano AM, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157.

Risitano AM等人。阵发性夜间血红蛋白尿的抗补体治疗：近端补体抑制的时间？EBMT SAAWP的立场文件。前免疫。2019年；10： 1157年。

Berentsen S, et al. Novel insights into the treatment of complement-mediated hemolytic anemias. Ther Adv Hematol. 2019;10:2040620719873321.

Berentsen S等人。补体介导的溶血性贫血治疗的新见解。Ther Adv Hematol。2019年；10： 2040620719873321。

Kulasekararaj AG, et al. Monitoring of patients with paroxysmal nocturnal hemoglobinuria on a complement inhibitor. Am J Hematol. 2021;96(7):E232-235.

Kulasekaraj AG等人。用补体抑制剂监测阵发性夜间血红蛋白尿患者。Am J Hematol。2021年；96（7）：E232-235。

ClinicalTrials.gov. Danicopan as add-on therapy to a C5 inhibitor in paroxysmal nocturnal hemoglobinuria (PNH) participants who have clinically evident extravascular hemolysis (EVH)(ALPHA). NCT Identifier: NCT04469465. Available here. Accessed November 2023.

ClinicalTrials.gov。Danicopan作为C5抑制剂的附加疗法，用于阵发性夜间血红蛋白尿（PNH）参与者，这些参与者具有临床上明显的血管外溶血（EVH）（α）。NCT标识符：NCT04469465。此处提供。2023年11月访问。