Octapharma USA宣布美国食品药品监督管理局（FDA）扩大批准 wilate®，用于6岁以下儿童的血管性血友病预防治疗-动脉网

Octapharma USA Announces Expanded FDA Approval of wilate® for von Willebrand Disease Prophylaxis in Children Younger Than 6 Years

PR Newswire 等信源发布 2026-07-08 22:21

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可切换为仅中文

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PARAMUS, N.J.

新泽西州帕拉默斯

，

July 8, 2026

2026年7月8日

/PRNewswire/ -- On July 2, 2026, the U.S. Food and Drug Administration (FDA) approved the use of wilate

/美通社/ -- 2026年7月2日，美国食品药品监督管理局（FDA）批准使用wilate

(von Willebrand Factor/Coagulation Factor VIII Complex [Human]) for routine prophylaxis to reduce the frequency of bleeding episodes in pediatric patients with von Willebrand Disease (VWD) who are younger than 6 years of age.

（人源性血管性血友病因子/凝血因子VIII复合物）用于常规预防，以减少6岁以下患有血管性血友病（VWD）的儿科患者出血发作的频率。

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This approval expands access to the first von Willebrand Factor (VWF) concentrate indicated for prophylactic treatment across all ages and forms of VWD, the most common inherited bleeding disorder.

此次批准扩大了首个血管性血友病因子（VWF）浓缩物的适用范围，该药适用于所有年龄段和所有类型的血管性血友病（VWD）的预防性治疗，而血管性血友病是最常见的遗传性出血性疾病。

继续阅读

Octapharma USA announced that pediatric patients, including children younger than 6 years of age, with von Willebrand Disease (VWD) now have access to routine prophylaxis to help reduce the frequency of bleeding episodes. The FDA’s expanded approval for wilate® (von Willebrand Factor/Coagulation Factor VIII Complex [Human]) Lyophilized Powder for Solution for Intravenous Injection, expands access to the first VWF concentrate indicated for routine prophylaxis in both adult and pediatric patients..

Octapharma USA 宣布，包括6岁以下儿童在内的血管性血友病（VWD）儿科患者现在可以使用常规预防疗法，以帮助减少出血发作的频率。美国食品药品监督管理局（FDA）对 wilate®（人血管性血友病因子/凝血因子VIII复合物）注射用冻干粉针剂的批准范围扩大，使首个适用于成人和儿科患者常规预防治疗的VWF浓缩物的可及性得以提升。

Octapharma USA has submitted a Biologics License Application Supplement (sBLA) to the U.S. Food and Drug Administration (FDA) to expand the approval of wilate®, von Willebrand Factor/Coagulation Factor VIII Complex

奥克塔法玛美国公司已向美国食品药品监督管理局（FDA）提交了一份生物制品许可申请补充材料（sBLA），以扩大wilate®（血管性血友病因子/凝血因子VIII复合物）的批准范围。

(Human) Lyophilized Powder for Solution for Intravenous Injection, to include routine prophylaxis to reduce the frequency of bleeding episodes in children and adults with any type of von Willebrand disease (VWD).

（人）冻干粉针剂，用于静脉注射，包括常规预防以减少任何类型的冯·维勒布兰德病（VWD）儿童和成人出血发作的频率。

The expanded label is based on data from the Phase 3 WIL-33 clinical trial (ClinicalTrials.gov ID:

扩展标签基于III期WIL-33临床试验的数据（ClinicalTrials.gov ID：

NCT04953884

), an international study to research the safety and efficacy of VWF prophylaxis in children with VWD under the age of 6.

），一项国际研究，旨在调查6岁以下血管性血友病（VWD）儿童使用血管性血友病因子（VWF）预防治疗的安全性和有效性。

'Evidence-based treatment guidelines recommend prophylactic treatment for VWD patients with a history of severe, frequent bleeds,

循证治疗指南建议对有严重、频繁出血史的血管性血友病（VWD）患者进行预防性治疗，

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' said Flemming Nielsen, President, Octapharma USA. 'Until now, pediatric hematologists have not had an FDA-approved option for routine prophylaxis in young children with VWD. With this expanded indication, children younger than 6 years of age can now receive routine prophylaxis to reduce the frequency of bleeding episodes, consistent with treatment options available for older children and adults.'.

Octapharma USA 总裁 Flemming Nielsen 表示：“迄今为止，儿科血液学家尚未拥有获美国食品药品监督管理局（FDA）批准的选项，用于对患有血管性血友病（VWD）的幼儿进行常规预防性治疗。随着此次适应症的扩大，6岁以下儿童现在可以接受常规预防性治疗，以减少出血发作的频率，这与适用于较大儿童和成人的治疗选择保持一致。”

The open-label WIL-33 trial enrolled and treated 12 patients, all under the age of 6 and diagnosed with severe VWD. Wilate

开放标签的WIL-33试验招募并治疗了12名患者，所有患者年龄均小于6岁，并被诊断为严重血管性血友病（VWD）。Wilate

was administered two to three times per week at a recommended dose of 30 to 50 international units (IU)/kg over 12 months. The primary endpoint was the total annualized bleeding rate (TABR) during prophylaxis, with safety and tolerability assessed throughout the study.

在12个月内，以30至50国际单位（IU）/kg的推荐剂量，每周给药两到三次。主要终点为预防治疗期间的总年化出血率（TABR），并在整个研究期间评估安全性和耐受性。

Researchers concluded that wilate

研究人员得出结论，wilate

was efficacious in the prevention and treatment of bleeding episodes in children under age 6, across all VWD types. The annual bleeding rate (ABR) measured during 12 months of prophylaxis was low (4.6 ± 6.1). Of the 56 bleeding episodes that occurred, 98.2% were determined to be minor in nature. In terms of treatment efficacy, of the 45 bleeding episodes treated, 95.6% required only one infusion..

在预防和治疗6岁以下儿童各型血管性血友病（VWD）的出血事件方面均显示出疗效。在12个月的预防治疗期间，年出血率（ABR）较低（4.6 ± 6.1）。在发生的56次出血事件中，98.2%被判定为轻度。就治疗 efficacy 而言，在接受治疗的45次出血事件中，95.6%仅需一次输注即可控制。

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The WIL-33 findings complement results from the WIL-31 study, the largest prospective study to date evaluating prophylaxis with wilate

WIL-33 的研究结果补充了 WIL-31 研究的结果，后者是迄今为止评估 wilate 预防性治疗的最大规模的前瞻性研究。

in adults and children aged 6 years and older with VWD. WIL-33 revealed a similar prophylactic effect in younger children to the efficacy demonstrated in WIL-31.

适用于6岁及以上患有血管性血友病（VWD）的成人和儿童。WIL-33在年幼儿童中显示出与WIL-31所证实的疗效相似的预防效果。

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Prophylaxis with wilate

使用Wilate进行预防性治疗

was well tolerated in this population, with no thrombotic events or FVIII accumulation observed over the course of the trial.

在该人群中耐受性良好，在试验期间未观察到血栓事件或FVIII蓄积。

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'Prior research into prophylactic treatment for VWD has been very limited in this age group,' said

“此前针对该年龄段血管性血友病（VWD）预防性治疗的研究非常有限，”

Akshat Jain, MD, MPH

阿克沙特·贾恩，医学博士，公共卫生硕士

, principal investigator for the WIL-33 trial. 'We now have the pharmacokinetic data and insights to confirm that VWF prophylaxis is safe, effective and well-tolerated in pediatric patients with VWD, including children younger than 6 years of age.'

WIL-33 试验的主要研究者表示：“我们现在拥有药代动力学数据和见解，证实 von Willebrand 因子（VWF）预防性治疗在患有血管性血友病（VWD）的儿科患者（包括 6 岁以下儿童）中安全、有效且耐受性良好。”

The FDA previously granted wilate

FDA此前批准了wilate

with an

用一个

orphan drug designation

孤儿药资格认定

for routine prophylaxis to reduce the frequency of bleeding episodes in adults and children 6 years of age and older with VWD, granting Octapharma a potential seven years of market exclusivity from the date of approval (December 1, 2023).

用于成人及6岁及以上血管性血友病（VWD）儿童的常规预防，以减少出血发作频率，从而使奥克塔法玛公司自批准之日（2023年12月1日）起获得潜在的市场独占期七年。

About the study

关于研究

WIL-33 was an open-label, prospective, non-controlled, international, multicenter phase 3 clinical trial investigating the efficacy, immunogenicity, and safety of wilate

WIL-33 是一项开放标签、前瞻性、非对照、国际多中心 III 期临床试验，旨在评估 wilate 的疗效、免疫原性和安全性。

prophylaxis and pharmacokinetics in 12 pediatric patients under 6 years old with severe VWD (VWF ristocetin cofactor activity [VWF:RCo] <20%). At baseline the median age was 2.0 (1.0–5.0), there were six male patients (50%), four patients (33.3%) had VWD type 2, and eight patients (66.7%) had VWD type 3..

12名6岁以下重度血管性血友病（VWD）（血管性血友病因子瑞斯托霉素辅因子活性[VWF:RCo] <20%）儿科患者的预防治疗及药代动力学研究。基线时，中位年龄为2.0岁（范围1.0–5.0岁），男性患者6名（50%），4名患者（33.3%）为2型VWD，8名患者（66.7%）为3型VWD。

Three patients were excluded from the per-protocol robustness (PPR) set, with two participants removed due to deviations from the recommended prophylactic dose; one additional patient prematurely discontinued treatment due to adverse events that were not considered treatment-emergent.

三名患者被排除在符合方案集稳健性（PPR）分析集之外，其中两名参与者因偏离推荐的预防剂量而被剔除；另外一名患者因发生不良事件而提前终止治疗，该不良事件被认为与治疗无关。

About wilate

关于 wilate

Wilate

维拉特

(von Willebrand Factor/Coagulation Factor VIII Complex [Human]) Lyophilized Powder for Solution for Intravenous Injection, is indicated in adult and pediatric patients with von Willebrand disease (VWD) for:

（人血管性血友病因子/凝血因子Ⅷ复合物）冻干粉针剂，用于配制静脉注射溶液，适用于成人和儿童血管性血友病（VWD）患者：

On-demand treatment and control of bleeding episodes.

按需治疗和控制出血发作。

Perioperative management of bleeding.

围手术期出血管理

Routine prophylaxis to reduce the frequency of bleeding episodes.

常规预防性治疗以减少出血发作的频率。

Wilate

维拉特

is indicated in adult and pediatric patients 12 years of age and older with hemophilia A for:

适用于12岁及以上的成人和儿童血友病A患者，用于：

On-demand treatment and control of bleeding episodes.

按需治疗和控制出血发作。

Routine prophylaxis to reduce the frequency of bleeding episodes.

常规预防性治疗以减少出血发作频率。

Important Safety Information

重要安全信息

CONTRAINDICATIONS

禁忌症

Do not use in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reactions, to human plasma-derived products, any ingredient in the formulation, or components of the container.

对于已知对人血浆衍生产品、制剂中的任何成分或容器组件有超敏反应（包括过敏性或严重全身性反应）的患者，禁止使用。

WARNINGS AND PRECAUTIONS

警告和注意事项

Anaphylaxis and severe hypersensitivity reactions are possible. Thromboembolic events may occur. Monitor plasma levels of FVIII activity. Neutralizing antibodies (inhibitors) to VWF and FVIII have occurred following administration of wilate

可能发生过敏性休克和严重超敏反应。可能发生血栓栓塞事件。监测血浆中FVIII活性水平。在使用wilate后，已出现针对VWF和FVIII的中和抗体（抑制物）。

. Test for neutralizing antibodies if plasma VWF and/or FVIII level fail to increase as expected or if bleeding is not controlled after wilate

如果血浆血管性血友病因子（VWF）和/或凝血因子VIII（FVIII）水平未按预期升高，或使用Wilate后出血未得到控制，则应检测中和抗体。

administration. Wilate

行政管理。威拉特

is made from human plasma and carries the risk of transmitting infectious agents.

由人血浆制成，存在传播感染性病原体的风险。

For full prescribing information, please visit

如需完整的处方信息，请访问

wilateusa.com/pi

。

About Octapharma

关于奥克塔法玛

Octapharma

奥克塔法玛

is one of the world's largest human protein manufacturers, developing and producing therapies from human plasma and human cell lines. Headquartered in

是全球最大的人体蛋白制造商之一，致力于从人血浆和人细胞系中开发和生产治疗产品。总部设在

Lachen

笑

, Switzerland,

，瑞士，

Octapharma

奥克塔法玛

has over 11,000 employees and serves patients in 117 countries across

拥有超过11,000名员工，为遍布全球的117个国家的患者提供服务

Immunotherapy

免疫疗法

, Hematology, and Critical Care.

、血液学和重症监护。

With seven R&D sites, five manufacturing facilities in Europe, and more than 200 plasma donation centers in the U.S. and Europe, Octapharma has delivered trusted therapies for over 40 years. The company is committed to improving lives through innovation, quality, and a deep focus on patient care and clinical partnership.

欧克法玛拥有七个研发中心、五个位于欧洲的生产设施，以及在美国和欧洲设立的200多个血浆捐献中心，四十余年来始终提供值得信赖的治疗方案。公司致力于通过创新、卓越品质以及对患者护理和临床合作的高度重视，改善人们的生活。

To learn more please visit .

欲了解更多信息，请访问。

www.octapharma.com

。

References

参考文献

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Wilate

威拉特

Prescribing Information

处方信息

Sholzberg M, Weyand AC, Lai JS, et al. Diagnosis and Disease Burden of Von Willebrand Disease in a Large US Population Based-Dataset.

Sholzberg M, Weyand AC, Lai JS 等. 基于美国大型人群数据集的血管性血友病诊断与疾病负担研究。

Blood

血液

. 2024; 144:3980-3981.

https://doi.org/10.1182/blood-2024-206631

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Connell NT, Flood VH, Brignardello-Peterson R, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease.

Connell NT, Flood VH, Brignardello-Peterson R, 等. ASH ISTH NHF WFH 2021年血管性血友病管理指南。

Blood Adv.

血液进展

2021; 5(1):301-325.

2021；5(1):301-325。

https://doi.org/10.1182/bloodadvances.2020003264

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Jain A, Leissinger C, Vdovin V, et al. Plasma-Derived VWF/FVIII Prophylaxis in Children Under 6 With VWD: First Results From WIL-33 [Poster abstract].

Jain A, Leissinger C, Vdovin V, 等. 血浆源性VWF/FVIII预防性治疗6岁以下血管性血友病儿童：WIL-33研究的初步结果 [海报摘要].

Blood

血液

. 2025; 146:4855-4856.

https://doi.org/10.1182/blood-2025-4855

WIL-0795

SOURCE Octapharma

来源：Octapharma

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