诺和诺德向美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）提交etavopivat的审批申请，该药是一种在研的每日一次疗法，用于治疗患有镰状细胞病的青少年和成人患者-动脉网

Novo submits etavopivat, an investigational once-daily therapy for adolescents and adults living with sickle cell disease, to the FDA and EMA for approval

CISION 等信源发布 2026-11-13 19:00

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The FDA New Drug Application (NDA) and EMA Marketing Authorization Application (MAA) submissions for etavopivat are based on data from the Phase 3 HIBISCUS trial showing efficacy and safety results in adolescents and adults living with sickle cell disease

etavopivat 的美国食品药品监督管理局（FDA）新药申请（NDA）和欧洲药品管理局（EMA）上市许可申请（MAA）均基于 III 期 HIBISCUS 试验的数据，该试验展示了其在患有镰状细胞病的青少年和成人患者中的有效性和安全性结果。

Sickle cell disease is a progressive, life-threatening, debilitating blood disease with limited disease-modifying therapeutic options that affects an estimated 100,000 Americans, 110,000 Europeans – and around 8 million people worldwide

镰状细胞病是一种进行性、危及生命且致残的血液疾病，目前可改变疾病进程的 therapeutic 选择有限。该病估计影响10万美国人、11万欧洲人，以及全球约800万人。

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Etavopivat is designed to help reduce the sickling of red blood cells and is being evaluated for potential improvement of hemoglobin response (>1 g/dL) rate at week 24 and the reduction of annualized 'vaso-occlusive crises' (VOCs) rates at week 52

Etavopivat 旨在帮助减少红细胞镰变，目前正对其在以下方面的潜在改善作用进行评估：第24周时血红蛋白反应率（>1 g/dL）的提高，以及第52周时年化“血管阻塞危象”（VOC）发生率的降低。

PLAINSBORO, N.J. and BAGSVÆRD, Denmark

新泽西州普莱恩斯伯勒和丹麦巴格斯韦德

，

Nov. 13, 2026

2026年11月13日

/PRNewswire/ -- Novo Nordisk today announced the submissions of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for etavopivat, an investigational, once-daily, orally available, selective activator of pyruvate kinase red blood cell isozyme (PKR), for the treatment of adolescents and adults (aged 12 years and older) living with sickle cell disease (SCD).

/美通社/ -- 诺和诺德今日宣布，已向美国食品药品监督管理局（FDA）提交新药申请（NDA），并向欧洲药品管理局（EMA）提交上市许可申请（MAA），申请批准etavopivat用于治疗12岁及以上青少年和成人镰状细胞病（SCD）患者。Etavopivat是一种在研的、每日一次口服给药的选择性红细胞丙酮酸激酶（PKR）同工型激活剂。

If approved, etavopivat could offer a new approach to treating SCD as a disease-modifying therapy..

如果获得批准，etavopivat 可能为治疗镰状细胞病（SCD）提供一种新的疾病修饰疗法。

The FDA has previously granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations and is expected to take action on the NDA in 2027. Etavopivat was also granted Orphan Drug designation by the European Commission who is expected to take action on the MAA in 2027. A MAA is the formal application for seeking approval to market a medicine in the European Union (EU)..

美国食品药品监督管理局（FDA）此前已授予etavopivat快速通道、罕见儿科疾病和孤儿药资格认定，并预计将于2027年对该新药申请（NDA）采取行动。欧盟委员会也已授予etavopivat孤儿药资格认定，并预计将于2027年对该上市许可申请（MAA）采取行动。上市许可申请（MAA）是寻求在欧盟（EU）获批上市药品的正式申请。

'For people living with sickle cell disease, severe vaso-occlusive crises and other symptoms can have a profound impact on everyday life, and treatment options remain limited,' said Martin Holst Lange, executive vice president, chief scientific officer and head of Research & Development at Novo. 'If approved, we are hopeful that etavopivat can provide a new disease-modifying treatment option for people living with sickle cell disease, and we will continue to work alongside the community to help advance health equity and overcome the longstanding disparities that impact those affected by this disease.'.

诺和诺德执行副总裁、首席科学官兼研发负责人马丁·霍尔斯特·兰格表示：“对于镰状细胞病患者而言，严重的血管阻塞危象及其他症状会对日常生活产生深远影响，而目前的治疗选择仍然有限。如果获得批准，我们希望etavopivat能为镰状细胞病患者提供一种新的疾病修饰治疗选择。我们将继续与社区携手合作，推动健康公平，消除长期影响该病患者的健康差距。”

The NDA and MAA submissions are grounded in the results from the HIBISCUS study program, which was designed to establish the efficacy and safety profile of etavopivat, and evaluated the annualized rate of vaso-occlusive crises (VOCs) at week 52 and hemoglobin response (>1 g/dL) rate at week 24 compared to placebo, in adults and adolescents living with SCD.

新药申请（NDA）和上市许可申请（MAA）的提交基于HIBISCUS研究计划的结果，该研究计划旨在确立etavopivat的疗效和安全性特征，并在患有镰状细胞病（SCD）的成人和青少年患者中，评估了第52周时血管阻塞危象（VOCs）的年发生率以及第24周时血红蛋白反应率（>1 g/dL），并与安慰剂进行了比较。

Participants were also allowed to receive standard of care treatment throughout the trial..

受试者在整个试验期间也可接受标准治疗。

In topline results announced in April 2026, etavopivat showed a 27% reduction in VOCs and a ~4-month delay to the first VOC compared to placebo. More detailed results from the HIBISCUS phase 3 trial will be presented at a future scientific conference and published in a peer-reviewed journal.

在2026年4月公布的顶线结果中，与安慰剂相比，etavopivat使血管闭塞危象（VOCs）减少了27%，并将首次VOC的发生时间延迟了约4个月。HIBISCUS III期临床试验更详细的结果将在未来的科学会议上公布，并发表在同行评审期刊上。

What is sickle cell disease?

什么是镰状细胞病？

Sickle cell disease (SCD) is driven by polymerization of mutated sickle hemoglobin (HbS) in red blood cells. The two main hallmarks of SCD are hemolytic anemia and vaso-occlusive crises (VOCs). Around 8 million people worldwide live with SCD, with the majority in low and middle-income countries.

镰状细胞病（SCD）是由红细胞中突变的镰状血红蛋白（HbS）聚合驱动的。该病的两个主要特征是溶血性贫血和血管阻塞危象（VOCs）。全球约有800万人患有镰状细胞病，其中大多数生活在低收入和中等收入国家。

Approximately 100,000 people living with SCD are in the United States, as well as approximately 110,000 in Europe, 5.7 million in sub-Saharan Africa, 1.28 million in India, and 618,000 in the Middle East and North Africa.

美国约有10万名镰状细胞病（SCD）患者，欧洲约有11万名，撒哈拉以南非洲有570万名，印度有128万名，中东和北非地区有61.8万名。

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The disease is characterized by several associated acute and chronic symptoms and complications such as acute chest syndrome, stroke and kidney, liver and heart disease.

该疾病的特征包括多种相关的急性和慢性症状及并发症，例如急性胸廓综合征、卒中以及肾脏、肝脏和心脏疾病。

Despite recent advances in treatment, many people with SCD experience significant levels of pain and reduced lifespan by approximately 20 years compared to the general population.

尽管治疗方面近期取得了进展，但许多镰状细胞病患者仍遭受显著的疼痛，且预期寿命较普通人群缩短约20年。

What is etavopivat?

什么是etavopivat？

Etavopivat is an investigational, once-daily, orally available, selective activator of pyruvate kinase red blood cell isozyme (PKR), a key enzyme in glycolysis. In SCD, PKR activation reduces 2,3-diphosphoglycerate (2,3-DPG) levels and increases adenosine triphosphate (ATP) production. Reduction of 2,3-DPG improves hemoglobin (Hb)-oxygen affinity, averting sickle hemoglobin polymerization and subsequent sickling.

Etavopivat 是一种在研的、每日一次口服给药的选择性红细胞丙酮酸激酶（PKR）激活剂，而 PKR 是糖酵解途径中的关键酶。在镰状细胞病（SCD）中，激活 PKR 可降低 2,3-二磷酸甘油酸（2,3-DPG）水平并增加三磷酸腺苷（ATP）的生成。2,3-DPG 的降低可提高血红蛋白（Hb）与氧的亲和力，从而避免镰状血红蛋白聚合及随后的红细胞镰变。

The increase in ATP production helps preserve RBC membrane integrity and deformability, improving RBC survival. These disease-modifying effects have the potential to reduce VOC events and improve Hb levels..

ATP生成的增加有助于维持红细胞膜的完整性和变形能力，从而延长红细胞的存活时间。这些疾病修饰效应有可能减少血管闭塞危象（VOC）事件并提高血红蛋白（Hb）水平。

The US Food and Drug Administration has granted etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations. Additionally, etavopivat was granted Orphan Drug designation by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency for the treatment of patients with SCD..

美国食品药品监督管理局已授予etavopivat快速通道、罕见儿科疾病和孤儿药资格认定。此外，基于欧洲药品管理局孤儿药产品委员会的积极意见，欧盟委员会已授予etavopivat孤儿药资格认定，用于治疗镰状细胞病患者。

Etavopivat was acquired by Novo as part of the 2022 acquisition of Forma Therapeutics.

Etavopivat 作为诺和诺德 2022 年收购 Forma Therapeutics 交易的一部分被纳入旗下。

What is the HIBISCUS program?

什么是HIBISCUS计划？

The HIBISCUS clinical development program investigates etavopivat as a disease-modifying treatment for people with SCD. The pivotal program includes:

HIBISCUS 临床开发计划旨在研究 etavopivat 作为镰状细胞病（SCD）患者的疾病修正疗法。该关键性计划包括：

HIBISCUS (NCT04624659) - a 52-week efficacy and safety design phase 2/3 trial, comparing once-daily etavopivat versus placebo, enrolling approximately 440 people aged 12 years and over with SCD who had 2 to 15 VOCs within 12 months prior to enrollment. Participants were allowed to receive standard of care treatment throughout the trial.

HIBISCUS（NCT04624659）——一项为期52周的2/3期疗效和安全性试验，比较每日一次etavopivat与安慰剂的疗效，招募约440名年龄在12岁及以上、在入组前12个月内发生过2至15次血管闭塞危象（VOC）的镰状细胞病（SCD）患者。参与者在整个试验期间可以接受标准治疗。

Following the completion of the 12-week phase 2 interim readout, the phase 3 dose was selected, and participants were randomized to the phase 3 main phase of the trial, which continued for 52 weeks..

在完成12周的第二阶段中期数据解读后，确定了第三阶段的剂量，并将参与者随机分配至试验的第三阶段主要部分，该阶段持续52周。

HIBISCUS 2 (NCT06612268) - a 52-week efficacy and safety phase 3b trial, comparing once-daily etavopivat versus placebo, with expected enrollment of 510 people aged 12 years and older with SCD.

HIBISCUS 2（NCT06612268）——一项为期52周的3b期疗效和安全性试验，比较每日一次etavopivat与安慰剂的效果，预计招募510名12岁及以上的镰状细胞病（SCD）患者。

FLORAL (NCT06609226) – an open-label extension following participation in etavopivat trials. The study allows the collection of long-term safety data.

FLORAL（NCT06609226）——一项在参与etavopivat临床试验后开展的开放标签扩展研究。该研究旨在收集长期安全性数据。

Novo is the global healthcare company that believes lasting health starts now. For over a century, we've combined leading scientific expertise with a deep understanding of people's lives. We develop treatments and support that help millions of people make progress they can see, feel and sustain now and in the future.

诺和诺德是一家全球性医疗健康企业，坚信持久健康始于当下。百余年来，我们将领先的科学专长与对人们生活的深刻理解相结合。我们开发的治疗方案和支持服务，帮助数百万人取得如今及未来可见、可感且可持续的进展。

Every day, over 66,500 employees around the world advance our purpose to drive change for lasting health. Through our partnerships, programs and investments, we're working to prevent disease, expand access to treatments and reduce our environmental impact to help even more people live healthier lives.

每天，全球66,500多名员工致力于推动变革，以实现持久健康的使命。通过我们的合作伙伴关系、项目和投资，我们努力预防疾病、扩大治疗可及性并减少对环境的影响，帮助更多人过上更健康的生活。

In the United States, Novo has a 40-year presence, is headquartered in New Jersey and employs approximately 10,000 people. For more information, visit .

在美国，诺和诺德已有40年的发展历程，总部设在新泽西州，员工人数约10,000人。欲了解更多信息，请访问。

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Novo Media:

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Liz Skrbkova (US)

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Novo Investors:

诺和投资者：

Michael Novod (Global)

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+45 3075 6050

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Sina Meyer (Global)

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References

参考文献

HIBISCUS Trial. Novo Nordisk Data on file. [Primary analysis of VOC based on negative binomial regression model with exposure time as offset variable. Primary analysis of Hb response based on exact Cochran-Mantel-Haenszel test with missing data ascribed as non-responders. Time to VOC based on Kaplan-Meier estimates and a stratified log-rank test.].

HIBISCUS 试验。诺和诺德内部数据。[基于以暴露时间为偏移变量的负二项回归模型对血管危象（VOC）进行主要分析。基于精确 Cochran-Mantel-Haenszel 检验对血红蛋白（Hb）反应进行主要分析，缺失数据视为无应答者。基于 Kaplan-Meier 估计和分层 log-rank 检验分析至血管危象（VOC）的时间。]

Belfer I, Chen W, Weber W, Edwards E, Langevin HM. Unmet need: mechanistic and translational studies of sickle cell disease pain as a whole-person health challenge. J Pain. 2024;25(10):104603. doi:10.1016/j.jpain.2024.104603 2. ASH sickle cell disease research priorities. American Society of Hematology.

Belfer I, Chen W, Weber W, Edwards E, Langevin HM. 未满足的需求：将镰状细胞病疼痛作为整体健康挑战的机制与转化研究。J Pain. 2024;25(10):104603. doi:10.1016/j.jpain.2024.104603 2. 美国血液学会镰状细胞病研究优先事项。美国血液学会。

Accessed October 2026..

2026年10月访问。

Centers for Disease Control and Prevention. Data and statistics on sickle cell disease. Sickle Cell Disease (SCD). Published May 15, 2024. Reviewed August 7, 2026. Accessed October 2026.

疾病控制与预防中心。镰状细胞病的数据和统计资料。镰状细胞病（SCD）。发布于2024年5月15日，审核于2026年8月7日，访问于2026年10月。

GBD 2021 Sickle Cell Disease Collaborators. 2023. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000-2021: a systemic analysis from the Global Burden of Disease Study 2021. The Lancet Haematology, 10, e585-e599.

全球疾病负担研究2021镰状细胞病合作组. 2023. 2000-2021年全球、区域和国家层面的镰状细胞病患病率及死亡负担：来自2021年全球疾病负担研究的系统分析. 《柳叶刀·血液病学》, 10, e585-e599.

https://doi.org/10.1016/S2352-3026

(23)00118-7

European Medicines Agency. EU/3/14/1263: Public summary of opinion on orphan designation.

欧洲药品管理局。EU/3/14/1263：孤儿药资格认定意见的公开摘要。

https://www.ema.europa.eu/en/documents/orphan-designation/eu3141263-public-summary-opinion-orphan-designation-autologous-cd34-haematopoietic-stem-cells-transduced-lentiviral-vector-encoding-human-beta-t87q-globin-gene-treatment-sickle-cell-dise_en.pdf

. Accessed October 2026.

访问于2026年10月。

SOURCE Novo Nordisk

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