首款，诺华IgA肾病疗法获FDA加速批准-动脉网

Novartis receives FDA accelerated approval for Vanrafia® (atrasentan), the first and only selective endothelin A receptor antagonist for proteinuria reduction in primary IgA nephropathy (IgAN)

CISION 等信源发布 2025-04-03 08:29

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Vanrafia can be seamlessly added to supportive care in IgAN and used as a foundational therapy with no requirement for a REMS (Risk Evaluation Mitigation Strategy) program

Vanrafia 可以无缝添加到 IgAN 的支持治疗中，并作为基础疗法使用，且无需 REMS（风险评估与缓解策略）计划。

Phase III data showed Vanrafia achieved proteinuria reduction of 36.1% (P<0.0001) vs. placebo with improvements seen at Week 6 and sustained through Week 36 and favorable safety

三期数据显示，与安慰剂相比，Vanrafia实现了36.1%的蛋白尿减少（P<0.0001），在第6周时显示出改善，并持续到第36周，且安全性良好。

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IgAN is a progressive, rare kidney disease; up to 50% of patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis

IgAN是一种进展性的罕见肾病；持续蛋白尿的患者中，多达50%会在诊断后的10到20年内进展为肾衰竭。

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With third FDA approval in under 1 year across its renal portfolio, Novartis is uniquely positioned to lead a transformation in kidney care

诺华在不到一年的时间内，其肾脏产品组合获得了第三次FDA批准，这使其在引领肾脏护理变革方面占据了独特地位。

EAST HANOVER, N.J.

新泽西州东汉诺威

，

April 2, 2025

2025年4月2日

/PRNewswire/ -- Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Vanrafia

/PRNewswire/ -- 诺华公司今天宣布，美国食品药品监督管理局 (FDA) 已授予Vanrafia加速批准。

(atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g

(atrasentan)，一种强效且选择性的内皮素A（ETA）受体拮抗剂，用于减少患有原发性免疫球蛋白A肾病（IgAN）且疾病进展迅速风险的成人的蛋白尿。这通常定义为尿蛋白与肌酐比值（UPCR）≥1.5 g/g。

. Vanrafia is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor

Vanrafia 是一种每日一次、非甾体类的口服治疗药物，可添加到支持性治疗中，包括肾素-血管紧张素系统（RAS）抑制剂，无论是否有钠-葡萄糖共转运蛋白-2（SGLT2）抑制剂。

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Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo

Vanrafia 基于预先设定的中期分析获得了加速批准，该分析来自第 III 期 ALIGN 研究，评估了与安慰剂相比在 36 周时蛋白尿减少的情况。

. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN. The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 136.

尚未确定Vanrafia是否能减缓IgAN患者的肾功能下降。Vanrafia的持续批准可能取决于正在进行的III期ALIGN研究的临床益处验证，该研究评估Vanrafia是否能够通过第136周的估算肾小球滤过率（eGFR）下降来减缓疾病进展。

. The eGFR data are expected in 2026 and intended to support traditional FDA approval.

预计eGFR数据将在2026年出炉，旨在支持传统的FDA批准。

'Today's approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,' said

“今天的批准标志着对IgA肾病患者而言是一个重要的里程碑，提供了一个可以无缝融入他们现有治疗计划的新选择，且没有REMS要求，”

Richard Lafayette

理查德·拉斐特

, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at

医学博士、美国医师学会会员、医学教授、肾病学专家及肾小球疾病中心主任

Stanford University

斯坦福大学

Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member. 'Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. Taking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.'.

医学中心，Vanrafia ALIGN 研究调查员和指导委员会成员。“Vanrafia 是一种选择性 ETA 受体拮抗剂，能有效减少蛋白尿，而蛋白尿是IgAN 的主要风险因素。及早采取果断行动对于帮助改善这些经常走向肾衰竭的患者的预后至关重要。”

IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria

IgAN 是一种进行性的罕见肾病，免疫系统会攻击肾脏，常导致肾小球炎症和蛋白尿。

. With almost 13 out of every million people in the US diagnosed per year, it is one of the most common autoimmune kidney diseases, and each person's journey is unique

在美国，每年每百万人中约有13人被诊断出患有这种疾病，它是最常见的自身免疫性肾病之一，每个人的病情历程都是独特的。

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. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation

在持续蛋白尿的IgAN患者中，多达50%的人在诊断后10到20年内会进展为肾衰竭，通常需要进行维持性透析和/或肾移植。

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, and response to treatment can vary

，且治疗反应可能有所不同

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. Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients

有效的、有针对性的疗法，其作用机制各异，可以帮助医生为患者选择最合适的治疗方案。

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'My son was diagnosed with IgA nephropathy long before there were any medicines approved to treat this condition, so the availability of multiple treatment options is incredibly meaningful for this community,' said

“我的儿子在有批准的药物治疗这种疾病之前很久就被诊断出患有IgA肾病，所以多种治疗选择的出现对这个群体来说意义非凡，”

Bonnie Schneider

邦妮·施耐德

, Director and Co-Founder, IgA Nephropathy Foundation. 'The approval of Vanrafia broadens the treatment landscape and expands the opportunity to tailor care in a disease that can impact each patient so differently.'

，IgA肾病基金会董事兼联合创始人。“Vanrafia的获批拓宽了治疗领域，并增加了在一种可能对每位患者产生不同影响的疾病中定制护理的机会。”

Data supporting approval

支持批准的数据

In the ongoing Phase III ALIGN study, patients receiving Vanrafia in combination with a RAS inhibitor achieved clinically meaningful and statistically significant proteinuria reduction of 36.1% (P<0.0001) compared to placebo, with results seen as early as week 6 and sustained through week 36

在正在进行的III期ALIGN研究中，与安慰剂相比，接受Vanrafia联合RAS抑制剂治疗的患者实现了临床显著且统计学意义的蛋白尿减少36.1%（P<0.0001），早在第6周就显现效果，并持续至第36周。

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. The effect of Vanrafia on UPCR was consistent across subgroups, including age, sex, race, and baseline disease characteristics, such as eGFR and proteinuria levels, in the main study cohort

在主要研究队列中，Vanrafia对UPCR的影响在各亚组中一致，包括年龄、性别、种族以及基线疾病特征，如eGFR和蛋白尿水平。

. A similar treatment effect of Vanrafia was seen in an additional group of patients treated with both a RAS inhibitor and an SGLT2 inhibitor (37.4% reduction in UPCR vs. placebo)

在同时使用RAS抑制剂和SGLT2抑制剂的另一组患者中，也观察到了Vanrafia类似的治疗效果（与安慰剂相比，UPCR减少37.4%）。

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The ALIGN study showed that Vanrafia has a favorable safety profile consistent with previously reported data

ALIGN研究表明，Vanrafia的安全性良好，与之前报告的数据一致。

. Adverse events reported in ≥2% of patients treated with Vanrafia, and more frequently than placebo, include peripheral edema, anemia, and liver transaminase elevation

据报道，≥2%的接受Vanrafia治疗的患者发生了不良事件，比安慰剂组更频繁，包括外周水肿、贫血和肝转氨酶升高。

. Because some endothelin receptor antagonists have caused elevations of aminotransferases, hepatotoxicity, and liver failure, clinicians should obtain liver enzyme testing before initiating Vanrafia and during treatment when clinically indicated. Vanrafia may cause serious birth defects

因为一些内皮素受体拮抗剂会引起转氨酶升高、肝毒性和肝衰竭，临床医生应在开始使用Vanrafia之前及治疗期间根据临床需要进行肝酶检测。Vanrafia可能导致严重的出生缺陷。

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Vanrafia does not require a REMS program.

Vanrafia 不需要 REMS 计划。

Transforming care in kidney disease

转化肾脏病的护理

'We are proud to expand the treatment landscape in IgA nephropathy with today's FDA accelerated approval of Vanrafia. IgAN is a heterogenous condition that requires differentiated therapies with unique mechanisms of action, and with our multi-asset kidney disease portfolio, we are well positioned to support a broad patient population and advance care for this disease,' said Victor Bultó, President, US, Novartis.

“我们很自豪能够通过今天FDA对Vanrafia的加速批准，进一步拓展IgA肾病的治疗领域。IgAN是一种异质性疾病，需要具有独特作用机制的差异化疗法，而凭借我们多元化的肾病资产组合，我们有能力支持广泛的患者群体，并推动该疾病的治疗进展。”诺华美国区总裁Victor Bultó表示。

'Building on our longstanding legacy in nephrology, we continue to rapidly grow our capabilities in this space. Each launch enables us to more effectively reach patients with the most suitable treatment option and deliver on our promise to transform kidney disease care.' .

“基于我们在肾病学领域的长期传承，我们不断快速增强这方面的能力。每次推出新产品都能使我们更有效地为患者提供最合适的治疗方案，并兑现我们改变肾病护理的承诺。”

This is the third US approval received by Novartis for its kidney disease portfolio in the last year, with Fabhalta

这是诺华在过去一年中获得的第三次美国批准，用于其肾脏疾病产品组合，包括Fabhalta。

having been granted FDA approval in C3 glomerulopathy (C3G) in

已在C3肾小球病（C3G）中获得FDA批准

March 2025

2025年3月

and accelerated approval in IgAN in

并在IgAN中加速批准

August 2024

2024年8月

. Fabhalta is also being studied in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN). Studies are ongoing to evaluate the safety and efficacy profiles in these investigational indications and support potential regulatory submissions.

法巴尔塔也在广泛的罕见肾病中进行研究，包括非典型溶血性尿毒症综合征（aHUS）、免疫复合物膜增生性肾小球肾炎（IC-MPGN）和狼疮性肾炎（LN）。相关研究正在进行中，以评估其在这些研究适应症中的安全性和有效性，并为潜在的监管提交提供支持。

An investigational subcutaneously administered anti-APRIL monoclonal antibody, zigakibart, is currently in Phase III development in IgAN, with results expected in 2026.

目前，一种研究性皮下注射的抗APRIL单克隆抗体齐卡巴特（zigakibart）正在IgAN的III期临床开发中，预计结果将在2026年公布。

。

About ALIGN

关于ALIGN

The ALIGN study (

ALIGN研究（

NCT04573478

) is a global, randomized, multicenter, double-blind, placebo-controlled Phase III clinical trial comparing the efficacy and safety of Vanrafia versus placebo in patients with IgAN at risk of progressive loss of kidney function

）是一项全球性、随机、多中心、双盲、安慰剂对照的 III 期临床试验，比较了 Vanrafia 与安慰剂在有进行性肾功能丧失风险的 IgAN 患者中的疗效和安全性。

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. In total, 340 individuals with biopsy-proven IgAN with baseline total proteinuria ≥1 g/day despite optimized RAS inhibitor treatment were randomized to receive once-daily, oral Vanrafia (0.75 mg) or placebo for approximately 132 weeks

共有340名经活检证实为IgAN的患者参与了随机试验，这些患者在使用优化的RAS抑制剂治疗后，基线总蛋白尿仍≥1克/天。他们被随机分配接受每日一次口服Vanrafia（0.75毫克）或安慰剂，持续约132周。

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. Patients continue receiving a maximally tolerated and stable dose of a RAS inhibitor as supportive care (unless they are unable to tolerate RAS inhibitor therapy)

患者继续接受最大耐受剂量且稳定的RAS抑制剂作为支持治疗（除非他们无法耐受RAS抑制剂治疗）。

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. An additional group of 64 patients receiving an SGLT2 inhibitor for at least 12 weeks was also enrolled

另外还招募了一组至少服用SGLT2抑制剂12周的64名患者。

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. The primary efficacy endpoint for the interim analysis is change in proteinuria, a marker of kidney damage, as measured by 24-hour UPCR from baseline to 36 weeks

中期分析的主要疗效终点是蛋白尿的变化，这是通过从基线到36周的24小时尿蛋白/肌酐比值（UPCR）测量的肾损伤标志物。

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. Secondary and exploratory objectives include evaluating the change in kidney function from baseline to 136 weeks as measured by eGFR, as well as safety and tolerability

。次要和探索性目标包括评估从基线到136周通过eGFR测量的肾功能变化，以及安全性和耐受性。

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Novartis in kidney disease

诺华在肾脏疾病领域

Building on a 40-year legacy that began in transplant, Novartis is on a mission to empower breakthroughs and transform care in kidney health, starting with kidney conditions that have significant unmet need. Historically, these conditions have had considerably less funding and research, leading to a treatment landscape largely focused on reactive or end-stage disease management, often with significant physical, emotional, and financial burdens.

基于始于移植领域的40年传承，诺华致力于推动肾病健康领域的突破，并从那些存在显著未满足需求的肾脏疾病开始，彻底改变治疗现状。历史上，这些疾病获得的资金和研究关注较少，导致目前的治疗格局主要集中于对疾病的被动应对或终末期管理，通常伴随着巨大的身体、情感和经济负担。

Our pipeline targets the underlying causes of disease, with an aim to protect kidney health and delay or prevent dialysis and/or transplantation. Our goal is to help patients get back to living life on their terms—whether at work, in school, or with loved ones, and by partnering with patients, advocates, clinicians and policymakers, we aim to raise awareness, accelerate diagnosis and get patients the right care, sooner..

我们的治疗方案旨在针对疾病的根本原因，保护肾脏健康，延缓或预防透析和/或移植。我们的目标是帮助患者重新按照自己的意愿生活——无论是在工作中、在学校里，还是与亲人在一起。通过与患者、倡导者、临床医生和政策制定者合作，我们力求提高意识、加速诊断，并让患者更快获得适当的治疗。

VANRAFIA Indications

范拉菲亚适应症

VANRAFIA is a prescription medicine used to reduce protein in the urine (proteinuria) in adults with a kidney disease called primary immunoglobulin A nephropathy (IgAN) who are at risk of their disease getting worse quickly. It is not known if VANRAFIA is safe and effective in children.

VANRAFIA 是一种处方药，用于减少患有原发性免疫球蛋白A肾病（IgAN）的成年患者尿液中的蛋白质（蛋白尿），这些患者有疾病快速恶化的风险。目前尚不清楚 VANRAFIA 在儿童中是否安全有效。

VANRAFIA is approved based on a reduction of proteinuria. Continued approval may require results from an ongoing study to determine whether VANRAFIA slows decline in kidney function.

VANRAFIA 的批准是基于蛋白尿减少的结果。继续批准可能需要来自一项正在进行的研究的结果，以确定 VANRAFIA 是否能减缓肾功能下降。

VANRAFIA Important Safety Information

范拉菲亚重要安全信息

VANRAFIA can cause serious birth defects if taken during pregnancy. Females should not be pregnant when they start taking VANRAFIA, become pregnant during treatment, or for 2 weeks after stopping treatment. Females who can become pregnant should have a negative pregnancy test before starting VANRAFIA..

如果在怀孕期间服用VANRAFIA，可能会导致严重的出生缺陷。女性在开始服用VANRAFIA时不应怀孕，在治疗期间不应怀孕，并且在停止治疗后的两周内也不应怀孕。能够怀孕的女性在开始服用VANRAFIA之前应该进行妊娠试验并结果为阴性。

Patients who can become pregnant are those who have entered puberty, even if they have not started their menstrual period, and have a uterus, and have not gone through menopause. Menopause means that patients have not had a menstrual period for at least 12 months for natural reasons, or that patients have had their ovaries removed.

能够怀孕的患者是指那些已经进入青春期、即使尚未开始月经、且拥有子宫、并未经历绝经的患者。绝经意味着患者因自然原因至少12个月没有月经，或者患者的卵巢已被切除。

Patients who cannot become pregnant are those who have not yet entered puberty, or do not have a uterus, or have gone through menopause. Females who can become pregnant should use effective birth control before starting treatment with VANRAFIA, during treatment with VANRAFIA and for 2 weeks after stopping VANRAFIA because the medicine may still be in their body.

不能怀孕的患者是那些尚未进入青春期、或没有子宫、或已绝经的患者。在开始使用VANRAFIA治疗之前、治疗期间以及停止使用VANRAFIA后两周内，能够怀孕的女性应采取有效的避孕措施，因为药物可能仍留在她们体内。

Patients should talk to their health care provider or gynecologist (a health care provider who specializes in reproduction) to find out about options for effective forms of birth control that they may use to prevent pregnancy during treatment with VANRAFIA. If the patient decides that they want to change the form of birth control that they use, they should talk to their health care provider or gynecologist to be sure that they choose another effective form of birth control.

患者应与其医疗服务提供者或妇科医生（专门从事生殖健康的医疗专业人员）交谈，以了解在使用VANRAFIA治疗期间可使用的有效避孕方法。如果患者决定更换所使用的避孕方式，他们应咨询医疗服务提供者或妇科医生，以确保选择另一种有效的避孕方法。

Patients should not have unprotected sex. Patients should talk to their health care provider or pharmacist right away if they have unprotected sex or if patients think their birth control has failed. Their health care provider may talk to them about using emergency birth control..

患者不应进行无保护措施的性行为。如果患者发生无保护措施的性行为，或者认为自己的避孕措施失败，应立即咨询医疗保健提供者或药剂师。他们的医疗保健提供者可能会与他们讨论使用紧急避孕措施。

Patients should tell their health care provider right away if they miss a menstrual period or think that they may be pregnant. Patients should not take VANRAFIA if they are pregnant, plan to become pregnant, or become pregnant during treatment with VANRAFIA. VANRAFIA can cause serious birth defects.

患者如果月经期错过或认为自己可能怀孕了，应立即告诉他们的医疗保健提供者。如果患者怀孕、计划怀孕或在使用万拉非治疗期间怀孕，则不应服用万拉非。万拉非可导致严重的出生缺陷。

Patients should not take VANRAFIA if they are allergic to atrasentan or any of the ingredients in VANRAFIA..

患者如果对atrasentan或VANRAFIA中的任何成分过敏，则不应服用VANRAFIA。

Before taking VANRAFIA, patients should tell their health care provider about all their medical conditions, including if they have liver problems, are pregnant or plan to become pregnant during VANRAFIA treatment (VANRAFIA can cause serious birth defects) or if they are breastfeeding or plan to breastfeed.

在服用VANRAFIA之前，患者应告诉他们的医疗保健提供者有关他们所有的医疗状况，包括他们是否有肝脏问题、是否怀孕或计划在VANRAFIA治疗期间怀孕（VANRAFIA可能导致严重的出生缺陷），或者他们是否正在哺乳或计划哺乳。

It is not known if VANRAFIA passes into their breast milk. Patients should not breastfeed during treatment with VANRAFIA. Patients should talk to their health care provider about the best way to feed their baby if they take VANRAFIA. .

目前尚不清楚VANRAFIA是否会进入母乳。患者在使用VANRAFIA治疗期间不应进行母乳喂养。如果患者服用VANRAFIA，应与其医疗保健提供者讨论喂养婴儿的最佳方法。

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking VANRAFIA with certain medications may affect the way VANRAFIA, and the other medicine works and may increase their risk for side effects.

患者应告知医疗保健提供者他们正在使用的所有药物，包括处方药、非处方药、维生素和草药补充剂。将VANRAFIA与某些药物一起使用可能会影响VANRAFIA及其他药物的作用效果，并可能增加副作用的风险。

Patients should not start any new medicine until they check with their health care provider..

患者在咨询医疗保健提供者之前不应开始任何新药。

VANRAFIA may cause serious side effects, including those mentioned above as well as liver problems, fluid retention and decreased sperm counts. Medicines like VANRAFIA can cause liver problems, including liver failure. VANRAFIA can increase liver enzymes in the patients' blood. The patients' healthcare provider will do blood tests to check their liver enzymes before starting treatment and if needed during treatment.

VANRAFIA 可能导致严重的副作用，包括上述提到的以及肝脏问题、体液潴留和精子数量减少。像 VANRAFIA 这样的药物可能引起肝脏问题，包括肝功能衰竭。VANRAFIA 会提高患者血液中的肝酶水平。患者的医疗保健提供者会在开始治疗前进行血液检测以检查肝酶水平，并在治疗期间根据需要进行检测。

The patients' healthcare provider may temporarily stop or permanently stop treatment with VANRAFIA if their liver enzymes increase or if they develop symptoms of liver problems. Patients should tell their health care provider if they have any of the following symptoms of liver problems while taking VANRAFIA; nausea or vomiting, pain in the upper right stomach, tiredness, loss of appetite, yellowing of their skin or whites of their eyes, dark urine, fever, itching.

如果患者的肝酶升高或出现肝脏问题的症状，他们的医疗服务提供者可能会暂时或永久停止使用VANRAFIA进行治疗。患者在服用VANRAFIA时如果出现以下任何肝脏问题的症状，应告知他们的医疗服务提供者：恶心或呕吐、右上腹部疼痛、疲劳、食欲不振、皮肤或眼白发黄、尿液变深、发烧、瘙痒。

VANRAFIA can cause their body to hold too much water (fluid retention). Patients should tell their healthcare provider if they develop any unusual weight gain, trouble breathing, or swelling of their ankles or legs during treatment. Their healthcare provider may prescribe other medicines (diuretics) and may temporarily stop VANRAFIA if they develop fluid retention.

VANRAFIA 可能导致身体储存过多水分（体液潴留）。患者在治疗期间如果出现异常体重增加、呼吸困难或脚踝或腿部肿胀，应告知医疗保健提供者。他们的医疗保健提供者可能会开具其他药物（利尿剂），并在出现体液潴留时暂时停止使用 VANRAFIA。

VANRAFIA may cause decreased sperm counts in males and may affect the ability to father a child. Patients should tell their doctor if being able to have children is important to them..

范拉非可能降低男性的精子数量，并可能影响生育能力。如果拥有孩子的能力对患者很重要，他们应该告诉医生。

The most common side effects of VANRAFIA include swelling of the hands, legs, ankles, and feet (peripheral edema) and low red blood cell count (anemia).

VANRAFIA最常见的副作用包括手、腿、脚踝和脚的肿胀（外周水肿）以及红细胞计数低（贫血）。

Please see full

请查看全文

Prescribing Information

处方信息

, including Boxed WARNING and

，包括盒装警告和

Medication Guide

药物指南

。

FABHALTA Indications

FABHALTA 适应症

FABHALTA is a prescription medicine used to:

FABHALTA 是一种处方药，用于：

Treat adults with paroxysmal nocturnal hemoglobinuria (PNH).

治疗患有阵发性夜间血红蛋白尿 (PNH) 的成人。

Reduce protein in the urine (proteinuria) in adults with a kidney disease called primary immunoglobulin A nephropathy (IgAN), who are at risk of their disease progressing quickly.

减少患有原发性免疫球蛋白A肾病（IgAN）的成人尿液中的蛋白质（蛋白尿），这些患者有疾病快速进展的风险。

FABHALTA is approved based on a reduction of proteinuria. Continued approval may require results from an ongoing study to determine whether FABHALTA slows decline in kidney function.

FABHALTA 的批准是基于蛋白尿的减少。持续批准可能需要来自正在进行的研究的结果，以确定 FABHALTA 是否能减缓肾功能下降。

Treat adults with a kidney disease called complement 3 glomerulopathy (C3G), to reduce protein in the urine (proteinuria).

治疗患有补体3肾小球病（C3G）的成人，以减少尿液中的蛋白质（蛋白尿）。

It is not known if FABHALTA is safe and effective in children with PNH, IgAN, or C3G.

目前尚不清楚FABHALTA对患有PNH、IgAN或C3G的儿童是否安全有效。

FABHALTA Important Safety Information

FABHALTA重要安全信息

FABHALTA is a medicine that affects part of the immune system and may lower one's ability to fight infections. FABHALTA increases the chance of getting serious infections caused by encapsulated bacteria, including

FABHALTA 是一种影响免疫系统部分功能的药物，可能会降低人体抵抗感染的能力。FABHALTA 会增加由封装细菌引起的严重感染的风险，包括

Streptococcus pneumoniae

肺炎链球菌

，

Neisseria meningitidis

脑膜炎奈瑟菌

, and

，以及

Haemophilus influenzae

流感嗜血杆菌

type b. These serious infections may quickly become life-threatening or fatal if not recognized and treated early. Patients must complete or update vaccinations against

b型。这些严重的感染如果不能及早发现和治疗，可能迅速危及生命或导致死亡。患者必须完成或更新针对

Streptococcus pneumoniae

肺炎链球菌

and

和

Neisseria meningitidis

脑膜炎奈瑟菌

at least 2 weeks before the first dose of FABHALTA. If patients have not completed vaccinations and FABHALTA must be started right away, they should receive the required vaccinations as soon as possible. If patients have not been vaccinated and FABHALTA must be started right away, they should also receive antibiotics to take for as long as their health care provider tells them.

在FABHALTA第一剂至少两周前完成接种。如果患者尚未完成疫苗接种但必须立即开始使用FABHALTA，则应尽快接种所需的疫苗。如果患者尚未接种疫苗且必须立即开始使用FABHALTA，他们还应根据医护人员的建议服用抗生素，并按照要求持续服用。

If patients have been vaccinated against these bacteria in the past, they might need additional vaccinations before starting FABHALTA. Their health care provider will decide if they need additional vaccinations. Vaccines do not prevent all infections caused by encapsulated bacteria. Patients should call their health care provider or get emergency medical care right away if they have any of these signs and symptoms of a serious infection: fever with or without shivers or chills; fever with chest pain and cough; fever with high heart rate; headache and fever; confusion; clammy skin; fever and rash; fever with breathlessness or fast breathing; headache with nausea or vomiting; headache with stiff neck or stiff back; body aches with flu-like symptoms; or eyes sensitive to light.

如果患者过去曾接种过针对这些细菌的疫苗，在开始使用FABHALTA之前，他们可能需要额外的疫苗接种。他们的医疗保健提供者将决定是否需要额外接种疫苗。疫苗并不能预防所有由荚膜细菌引起的感染。如果患者出现严重感染的以下任何症状和体征，应立即致电其医疗保健提供者或寻求紧急医疗护理：发冷或寒战伴发烧；胸痛和咳嗽伴发烧；高心率伴发烧；头痛伴发烧；意识模糊；皮肤湿冷；发烧伴皮疹；呼吸急促或快速呼吸伴发烧；恶心或呕吐伴头痛；颈项强直或背部僵硬伴头痛；类似流感症状的全身酸痛；或眼睛对光敏感。

Health care providers will give their patients a Patient Safety Card about the risk of serious infections. Patients must carry it with them at all times during treatment and for 2 weeks after the last dose of FABHALTA. The risk of serious infections may continue for a few weeks after their last dose of FABHALTA.

医护人员将向患者提供一份关于严重感染风险的患者安全卡。患者在治疗期间以及最后一次服用FABHALTA后两周内必须随身携带该卡片。在最后一次服用FABHALTA后的几周内，严重感染的风险可能仍然存在。

It is important for patients to show this card to any health care provider who treats them. This will help health care providers diagnose and treat patients quickly..

患者向任何治疗他们的医疗保健提供者出示此卡很重要。这将帮助医疗保健提供者快速诊断和治疗患者。

FABHALTA is only available through a program called the FABHALTA Risk Evaluation and Mitigation Strategy (REMS). Before patients can take FABHALTA, their health care provider must enroll in the FABHALTA REMS program, counsel their patients about the risk of serious infections caused by certain bacteria, give their patients information about the symptoms of serious infections, make sure that their patients are vaccinated against serious infections caused by encapsulated bacteria and that they receive antibiotics if they need to start FABHALTA right away and are not up-to-date on vaccinations, as well as give patients a Patient Safety Card about the risk of serious infections..

FABHALTA 只能通过名为 FABHALTA 风险评估与减轻策略 (REMS) 的计划获取。在患者服用 FABHALTA 之前，其医疗保健提供者必须注册参加 FABHALTA REMS 计划，向患者说明某些细菌引起的严重感染风险，提供有关严重感染症状的信息，确保患者已接种针对包裹性细菌引起的严重感染的疫苗，并在需要立即开始使用 FABHALTA 且疫苗接种不完全的情况下给予抗生素，同时向患者提供一份关于严重感染风险的患者安全卡。

Patients should not take FABHALTA if they are allergic to FABHALTA or any of the ingredients in FABHALTA. Patients should not take FABHALTA if they have a serious infection caused by encapsulated bacteria, including

患者如果对FABHALTA或FABHALTA中的任何成分过敏，则不应服用FABHALTA。如果患者患有由包被细菌引起的严重感染，包括

Streptococcus pneumoniae

肺炎链球菌

，

Neisseria meningitidis

脑膜炎奈瑟菌

, or

，或者

Haemophilus influenzae

流感嗜血杆菌

type b, when starting FABHALTA.

类型 b，在启动 FABHALTA 时。

Before taking FABHALTA, patients should tell their health care provider about all their medical conditions, including if they have an infection or fever, have liver problems, are pregnant or plan to become pregnant (it is not known if FABHALTA will harm an unborn baby), or are breastfeeding or plan to breastfeed as it is not known if FABHALTA passes into breast milk.

在服用FABHALTA之前，患者应告知医疗保健提供者所有健康状况，包括是否有感染或发烧、是否有肝脏问题、是否怀孕或计划怀孕（目前尚不清楚FABHALTA是否会伤害未出生的婴儿），或是否正在哺乳或计划哺乳，因为目前尚不清楚FABHALTA是否会进入母乳。

Patients should not breastfeed during treatment and for 5 days after the final dose of FABHALTA..

患者在治疗期间和最后一次服用FABHALTA后5天内不应进行母乳喂养。

Patients should tell their health care provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking FABHALTA with certain other medicines may affect the way FABHALTA works and may cause side effects. Patients should know the medicines they take and the vaccines they receive.

患者应告知其医疗保健提供者他们正在使用的所有药物，包括处方药、非处方药、维生素和草药补充剂。将FABHALTA与某些其他药物一起使用可能会影响FABHALTA的作用方式，并可能导致副作用。患者应了解他们所服用的药物以及接种的疫苗。

Patients should keep a list of them to show their health care provider and pharmacist when they get a new medicine..

患者在获得新药时应保留一份清单，以便向医疗服务提供者和药剂师展示。

FABHALTA may cause serious side effects, including those mentioned above as well as increased cholesterol and triglyceride (lipid) levels in the blood. Health care providers will do blood tests to check patients' cholesterol and triglycerides during treatment with FABHALTA. Health care providers may start patients on medicine to lower cholesterol if needed..

FABHALTA 可能导致严重的副作用，包括上述提到的副作用，以及血液中胆固醇和甘油三酯（脂质）水平升高。医疗保健提供者会在 FABHALTA 治疗期间进行血液检测，以检查患者的胆固醇和甘油三酯水平。如有需要，医疗保健提供者可能会让患者开始服用降低胆固醇的药物。

The most common side effects of FABHALTA in adults include: headache; nasal congestion, runny nose, cough, sneezing, and sore throat (nasopharyngitis); diarrhea; pain in the stomach (abdomen); infections (bacterial and viral); nausea; and rash.

成人使用FABHALTA最常见的副作用包括：头痛；鼻塞、流鼻涕、咳嗽、打喷嚏和喉咙痛（鼻咽炎）；腹泻；腹痛（腹部）；感染（细菌性和病毒性）；恶心；以及皮疹。

Please see full

请查看完整内容

Prescribing Information

处方信息

, including Boxed WARNING

，包括盒装警告

and

和

Medication Guide

药品指南

。

Disclaimer

免责声明

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as 'potential,' 'can,' 'will,' 'plan,' 'may,' 'could,' 'expected,' 'investigational,' 'pipeline,' 'launch,' 'transformation,' 'continue,' 'continued,' 'opportunity,' 'ongoing,' 'to evaluate,' 'progressive,' 'aim,' 'goal,' or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for Vanrafia (atrasentan), or regarding potential future revenues from Vanrafia.

本新闻稿包含1995年美国私人证券诉讼改革法意义上的前瞻性陈述。前瞻性陈述通常可以通过诸如“潜在”、“可以”、“将”、“计划”、“可能”、“能够”、“预期”、“研究性”、“管线”、“推出”、“转型”、“继续”、“持续”、“机会”、“正在进行”、“评估”、“渐进”、“目标”、“目的”等词语或关于Vanrafia（atrasentan）潜在市场批准、新适应症或标签的明确或隐含讨论，或关于Vanrafia潜在未来收入的类似术语来识别。

You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements.

您不应过分依赖这些声明。此类前瞻性声明基于我们对当前对未来事件的信念和预期，并受到重大已知和未知风险及不确定性的约束。如果其中一项或多项风险或不确定性成为现实，或者如果基本假设被证明不正确，实际结果可能与前瞻性声明中所述的结果存在重大差异。

There can be no guarantee that Vanrafia will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that Vanrafia will be commercially successful in the future. In particular, our expectations regarding Vanrafia could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for incr.

不能保证 Vanrafia 会被提交或批准在任何市场销售，或获得任何额外的适应症或标签，也不能保证其会在特定时间获批。同时，也不能保证 Vanrafia 在未来会取得商业成功。特别是，我们对 Vanrafia 的期望可能受到多种因素的影响，其中包括研发过程中固有的不确定性，包括临床试验结果及对现有临床数据的进一步分析；监管行动或延迟以及总体政府法规；全球范围内控制医疗成本的趋势，包括来自政府、支付方和公众的定价与报销压力，以及对增加相关要求的趋势。

About Novartis

关于诺华

Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide.

诺华是一家创新药物公司。每一天，我们都在努力重新构想药物，以改善和延长人们的生命，使患者、医疗专业人员和社会能够在面对严重疾病时得到更好的支持。我们的药物惠及全球近3亿人口。

Reimagine medicine with us: Visit us at

重新构想医学的未来：访问我们 tại

https://www.novartis.com

and

和

https://www.novartis.us

and connect with us on

并关注我们

领英

，

LinkedIn US

领英美国

，

Facebook

脸书

，

X/Twitter

，

X/Twitter US

X/Twitter 美国

and

和

Instagram

。

References

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：诺华制药公司；

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新英格兰医学杂志

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谢J，基里柳克K，王W，等。预测IgA肾病的进展：新的临床进展风险评分。

PLoS One

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. 2012;7(6):e38904. doi:10.1371/journal.pone.0038904

Rodrigues JC, Haas M, Reich HN. IgA Nephropathy.

罗德里格斯 JC，哈斯 M，赖希 HN。IgA肾病。

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. 2017;12(4):677-686. doi:10.2215/CJN.07420716

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Pitcher D, Braddon F, Hendry B, 等。IgA肾病的长期结局。

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Sim JJ et al. Poster TH-PO615 presented at: ASN Kidney Week 2023;

Sim JJ 等。海报 TH-PO615 在：ASN 肾脏周 2023 上展示；

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2023年11月2日至5日

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Bobart SA、Alexander MP、Shawwa K 等。微量血尿与IgA肾病中系膜增生、内皮细胞增生、新月体评分及肾脏结局的关联。

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. 2021;36(5):840-847. doi:10.1093/ndt/gfz267

Saha MK et al. Poster TH-PO1016 presented at: ASN Kidney Week 2019;

Saha MK 等。海报 TH-PO1016 在：ASN 肾脏周 2019 展示；

November 5-10, 2019

2019年11月5日至10日

;

；

Washington, DC

华盛顿特区

。

National Institute of Diabetes and Digestive and Kidney Diseases. IgA nephropathy. Available from:

国家糖尿病、消化和肾脏疾病研究所。IgA肾病。可从以下网址获取：

https://www.niddk.nih.gov/health-information/kidney-disease/iga-nephropathy

. Accessed

. 已访问

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。

Kwon CS, Daniele P, Forsythe A, Ngai C. A systematic literature review of the epidemiology, health-related quality of life impact, and economic burden of immunoglobulin A nephropathy.

权CS，达尼埃莱P，福赛思A，倪C。免疫球蛋白A肾病的流行病学、健康相关生活质量影响和经济负担的系统文献综述。

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健康经济学与结果研究杂志

. 2021;8(2):36-45. doi:10.36469/001c.26129

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肾脏疾病：改善全球预后（KDIGO）2021年肾小球疾病管理临床实践指南。

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肾脏国际

. 2021;100(4):S1-S276. doi:10.1016/j.kint.2021.05.021

Boyd JK, Cheung CK, Molyneux K, Feehally J, Barratt J. An Update on the Pathogenesis and Treatment of IgA Nephropathy.

博伊德 JK，张 CK，莫利纽克斯 K，菲哈利 J，巴拉特 J。关于IgA肾病发病机制与治疗的最新进展。

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肾脏国际

. 2012;81(9):833-843.

ClinicalTrials.gov. NCT04573478. A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Atrasentan in Patients With IgA Nephropathy at Risk of Progressive Loss of Renal Function. Available from

ClinicalTrials.gov. NCT04573478. 一项针对有进行性肾功能丧失风险的IgA肾病患者使用Atrasentan的III期、随机、双盲、安慰剂对照研究。可从以下网址获取：

https://clinicaltrials.gov/study/NCT04573478

. Accessed

. 已访问

March 2025

2025年3月

。

FABHALTA prescribing information.

FABHALTA处方信息。

East Hanover, NJ

新泽西州东汉诺威

: Novartis Pharmaceuticals Corp;