NEURELIS宣布 FDA批准立即使用抗癫痫药物 VALTOCO®（地西泮鼻喷雾剂）于 2至 5岁儿童-动脉网

NEURELIS ANNOUNCES FDA APPROVAL FOR IMMEDIATE USE SEIZURE MEDICATION VALTOCO® (DIAZEPAM NASAL SPRAY) IN AGES 2 TO 5

CISION 等信源发布 2025-04-16 21:00

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VALTOCO maintains orphan drug exclusivity for VALTOCO to treat episodes of frequent seizures

VALTOCO 拥有治疗频繁癫痫发作的孤儿药独占权。

SAN DIEGO

圣地亚哥

，

April 16, 2025

2025年4月16日

/PRNewswire/ -- Neurelis, Inc., today announced that the U.S. Food and Drug Administration (FDA) has approved VALTOCO® (diazepam nasal spray) for short-term treatment of seizure clusters (also known as 'acute repetitive seizures') that are different from a person's normal seizure pattern in people 2 years of age and older.

/PRNewswire/ -- Neurelis, Inc. 今日宣布，美国食品药品监督管理局 (FDA) 已批准 VALTOCO®（地西泮鼻喷雾剂）用于治疗2岁及以上人群的癫痫集群（也称为“急性重复性癫痫发作”），这些癫痫集群不同于患者的正常癫痫模式。

VALTOCO is a proprietary formulation which utilizes an absorption enhancement technology, INTRAVAIL®, to enable the noninvasive, enhanced intranasal delivery of diazepam. The intranasal formulation of Neurelis' VALTOCO was previously recognized by the FDA as clinically superior to the rectal gel formulation of diazepam resulting in its orphan drug exclusivity designation. .

VALTOCO 是一种采用吸收增强技术 INTRAVAIL® 的专利配方，可实现非侵入性、增强的鼻腔内递送地西泮。Neurelis 的 VALTOCO 鼻腔喷雾剂此前被 FDA 认定在临床上优于地西泮的直肠凝胶剂型，从而获得了孤儿药独占权。

'We are so grateful for all those who participated in the clinical study to enable VALTOCO to reach this milestone, especially the patients and families whose participation in the trial helped expand access to a unique immediate-use medication to help stop an episode of frequent seizures,' said

“我们非常感谢所有参与临床研究的人，使VALTOCO达到了这一里程碑，特别是那些参与试验的患者和家属，他们的参与帮助扩大了获得一种独特的即用型药物的机会，以帮助阻止频繁癫痫发作的发作，”

Craig Chambliss

克雷格·查姆布利斯

, Neurelis Founder and CEO.

，Neurelis创始人兼首席执行官。

'The FDA's decision to approve VALTOCO for use in early childhood highlights the established balanced safety and efficacy profile,' commented

“FDA批准VALTOCO用于幼儿期的决定突显了其已确立的平衡安全性和有效性。”评论道

Eric Segal

埃里克·西格尔

, MD, Director of Pediatric Epilepsy at Northeast Regional Epilepsy Group and Hackensack University Medical Center. 'VALTOCO fills a large unmet need for children with seizures and their families. I am hopeful that this product will improve quality of life for this specific population.'

医学博士，东北地区癫痫组和哈肯萨克大学医学中心的儿科癫痫主任。 “VALTOCO 满足了癫痫儿童及其家人的巨大未满足需求。我希望该产品能提高这部分人群的生活质量。”

Approximately 3.4 million people in the U.S. have epilepsy, including 400,000 children. While chronic epilepsy can be controlled by medications, some patients are at risk of episodes of frequent seizures, or acute repetitive seizures.

美国约有 340 万人患有癫痫症，其中包括 40 万名儿童。虽然慢性癫痫可以通过药物控制，但有些患者面临频繁发作或急性反复发作的风险。

'For children who have episodes of frequent seizures, the current standard of care requiring rectal administration of medication to stop a seizure can be a significant challenge for caregivers and children alike,' added Jurriaan M Peters, MD, PhD, Director, Localization Laboratory, Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital, and Associate Professor in Neurology at Harvard Medical School. 'Approval of an immediate-use intranasal diazepam treatment that can be given at any time, even while a child is actively seizing, in this young age group is an important advance for the epilepsy community.'.

“对于频繁发作癫痫的儿童，目前需要通过直肠给药来停止癫痫发作的标准护理方案，对护理人员和儿童来说都是一个重大挑战，”波士顿儿童医院癫痫与临床神经生理学部定位实验室主任、哈佛医学院神经病学副教授Jurriaan M Peters医学博士补充道。“批准一种可在任何时间（甚至在儿童癫痫发作时）立即使用的鼻腔内给药地西泮治疗方案，是癫痫领域在这个年轻群体中的重要进步。”

Data from the phase 1/2a clinical study evaluating safety and pharmacokinetics demonstrated that VALTOCO is safe and effective with easy nasal administration for patients aged 2 years and older who have episodes of frequent seizures. The company presented data on the study at the

评估VALTOCO安全性和药代动力学的1/2a期临床研究数据显示，对于2岁及以上频繁发作癫痫的患者，通过鼻腔给药非常安全、有效且简便。该公司在会议上展示了这项研究的数据。

Annual Child Neurology Society Meeting

年度儿童神经病学学会会议

在

November 2024

2024年11月

在

San Diego

圣地亚哥

and

和

Annual American Epilepsy Society Meeting

美国癫痫学会年会

在

December 2024

2024年12月

在

Los Angeles

洛杉矶

。

About Neurelis

关于Neurelis

Neurelis, Inc., is a neuroscience company focused on the development and commercialization of therapeutics for the treatment of epilepsy and neurologic disorders characterized by high unmet medical need. The FDA has approved Neurelis' VALTOCO® (diazepam nasal spray) as an acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from an individual's usual seizure pattern in adult and pediatric patients 2 years of age and older.

Neurelis, Inc.是一家神经科学公司，专注于开发和商业化用于治疗癫痫和神经系统疾病（具有高度未满足医疗需求）的药物。美国食品药品监督管理局（FDA）已批准Neurelis公司的VALTOCO®（地西泮鼻喷雾剂）作为急性治疗方法，用于治疗间歇性、刻板型频繁发作的癫痫活动（即癫痫丛集发作、急性重复性癫痫），这些发作与2岁及以上成人和儿童患者的日常癫痫模式不同。

VALTOCO is a proprietary formulation of diazepam incorporating the science of INTRAVAIL®, a transmucosal absorption enhancement technology that enables the noninvasive delivery of a broad range of protein, peptide, and small-molecule drugs. For more information on VALTOCO, please visit .

VALTOCO 是一种含有地西泮的专利配方，融合了 INTRAVAIL® 技术，这是一种透黏膜吸收增强技术，能够以非侵入性方式递送多种蛋白质、多肽和小分子药物。如需更多关于 VALTOCO 的信息，请访问。

www.valtoco.com

. For the latest scientific information on VALTOCO, please visit

如需了解VALTOCO的最新科学信息，请访问

http://www.neurelismedicalaffairs.com/

. Neurelis is also developing NRL-1004, an investigational, Phase 1 stage intranasal olanzapine for treatment of acute agitation episodes associated with schizophrenia and bipolar disorder. In addition, Neurelis is also developing NRL-1049 (previously known as BA-1049), an investigational, Phase 1 new chemical entity Rho kinase (ROCK) inhibitor, for the treatment of cerebral cavernous malformations (CCMS), a rare disorder of the central nervous system (CNS).

Neurelis公司还在研发NRL-1004，一种处于第一阶段试验的鼻腔给药型奥氮平，用于治疗与精神分裂症和双相情感障碍相关的急性躁动发作。此外，Neurelis还在开发NRL-1049（之前称为BA-1049），一种处于第一阶段试验的新型化学实体Rho激酶（ROCK）抑制剂，用于治疗脑海绵状血管瘤（CCMS），这是一种罕见的中枢神经系统（CNS）疾病。

For more information on Neurelis, please visit .

如需更多关于Neurelis的信息，请访问。

www.neurelis.com

。

Important Safety Information about VALTOCO:

关于VALTOCO的重要安全信息：

Indication

适应症

VALTOCO

瓦尔托科

(diazepam nasal spray) is indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient's usual seizure pattern in patients with epilepsy 2 years of age and older.

（地西泮鼻喷雾剂）适用于急性治疗间歇性、刻板型频繁发作的癫痫活动（即，癫痫丛集发作、急性重复性癫痫发作），这些发作不同于2岁及以上癫痫患者的常规癫痫模式。

WARNING: RISKS FROM CONCOMITANT USE WITH OPIOIDS; ABUSE, MISUSE, AND ADDICTION; and DEPENDENCE AND WITHDRAWAL REACTIONS

警告：与阿片类药物同时使用带来的风险；滥用、误用和成瘾；以及依赖性和戒断反应

Concomitant use of benzodiazepines and opioids may result in profound sedation, respiratory depression, coma, and death. Reserve concomitant prescribing of these drugs for patients for whom alternative treatment options are inadequate. Limit dosages and durations to the minimum required. Follow patients for signs and symptoms of respiratory depression and sedation..

同时使用苯二氮卓类药物和阿片类药物可能会导致深度镇静、呼吸抑制、昏迷甚至死亡。仅在其他治疗方案不足的情况下，才考虑同时开具这些药物。限制剂量和疗程至最低必要水平。监测患者是否出现呼吸抑制和镇静的迹象和症状。

The use of benzodiazepines, including VALTOCO, exposes users to risks of abuse, misuse, and addiction, which can lead to overdose or death. Abuse and misuse of benzodiazepines commonly involve concomitant use of other medications, alcohol, and/or illicit substances, which is associated with an increased frequency of serious adverse outcomes.

使用苯二氮卓类药物（包括VALTOCO）会使用户面临滥用、误用和成瘾的风险，这可能导致过量服用或死亡。苯二氮卓类药物的滥用和误用通常涉及同时使用其他药物、酒精和/或非法物质，这与严重不良后果发生频率增加相关。

Before prescribing VALTOCO and throughout treatment, assess each patient's risk for abuse, misuse, and addiction..

在开处方VALTOCO之前及整个治疗过程中，评估每位患者滥用、误用和成瘾的风险。

The continued use of benzodiazepines may lead to clinically significant physical dependence. The risks of dependence and withdrawal increase with longer treatment duration and higher daily dose. Although VALTOCO is indicated only for intermittent use, if used more frequently than recommended, abrupt discontinuation or rapid dosage reduction of VALTOCO may precipitate acute withdrawal reactions, which can be life-threatening.

苯二氮卓类药物的持续使用可能导致临床上显著的生理依赖。依赖和戒断的风险随着治疗时间的延长和每日剂量的增加而增加。尽管VALTOCO仅适用于间歇性使用，但如果使用频率高于推荐，突然停止使用或快速减少VALTOCO剂量可能引发急性戒断反应，这可能是危及生命的。

For patients using VALTOCO more frequently than recommended, to reduce the risk of withdrawal reactions, use a gradual taper to discontinue VALTOCO..

对于使用VALTOCO频率高于推荐的患者，为降低戒断反应的风险，应逐步减量直至停止使用VALTOCO。

Contraindications:

禁忌症：

VALTOCO is contraindicated in patients with:

VALTOCO禁用于以下患者：

Hypersensitivity to diazepam

对地西泮过敏

Acute narrow-angle glaucoma

急性闭角型青光眼

Central Nervous System (CNS) Depression

中枢神经系统（CNS）抑制

Benzodiazepines

苯二氮卓类药物

, including

，包括

VALTOCO

瓦尔托科

, may produce

，可能会产生

CNS

中枢神经系统

depression. Caution patients against engaging in hazardous activities requiring mental alertness, such as operating machinery, driving a motor vehicle, or riding a bicycle, until the effects of the drug, such as drowsiness, have subsided, and as their medical condition permits.

抑郁症。提醒患者在药物作用（如嗜睡）消退且其健康状况允许之前，避免从事需要精神警觉的危险活动，例如操作机械、驾驶机动车或骑自行车。

The potential for a synergistic CNS-depressant effect when VALTOCO is used with alcohol or other CNS depressants must be considered, and appropriate recommendations made to the patient and/or care partner.

使用VALTOCO与酒精或其他中枢神经系统抑制剂时，必须考虑潜在的协同中枢神经系统抑制效应，并向患者和/或护理伙伴提出适当建议。

Suicidal Behavior and Ideation

自杀行为与意念

Antiepileptic

抗癫痫的

drugs (

药物 (

AEDs

自动体外除颤器

), including

），包括

VALTOCO

瓦尔托科

, increase the risk of suicidal ideation and behavior. Patients treated with any

，增加自杀意念和行为的风险。接受任何治疗的患者

AED

自动体外除颤器

for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behavior, and/or unusual changes in mood or behavior.

对于任何适应症，应监测是否出现或加重抑郁、自杀念头或行为，以及/或情绪或行为的异常变化。

Glaucoma

青光眼

Benzodiazepines, including VALTOCO, can increase intraocular pressure in patients with glaucoma. VALTOCO may only be used in patients with open-angle glaucoma only if they are receiving appropriate therapy. VALTOCO is contraindicated in patients with narrow-angle glaucoma.

苯二氮卓类药物，包括VALTOCO，会增加青光眼患者的眼内压。仅当开角型青光眼患者正在接受适当治疗时，才可以使用VALTOCO。VALTOCO禁用于窄角型青光眼患者。

Neonatal Sedation and Withdrawal Syndrome

新生儿镇静和戒断综合征

Use of VALTOCO late in pregnancy can result in sedation (respiratory depression, lethargy, hypotonia) and/or withdrawal symptoms (hyperreflexia, irritability, restlessness, tremors, inconsolable crying, and feeding difficulties) in the neonate. Monitor neonates exposed to VALTOCO during pregnancy or labor for signs of sedation and monitor neonates exposed to VALTOCO during pregnancy for signs of withdrawal; manage these neonates accordingly..

在妊娠晚期使用VALTOCO可能导致新生儿出现镇静（呼吸抑制、嗜睡、肌张力低下）和/或戒断症状（反射亢进、易激惹、不安、震颤、无法安抚的哭泣以及喂养困难）。对在妊娠期或分娩期间暴露于VALTOCO的新生儿监测镇静迹象，并对在妊娠期暴露于VALTOCO的新生儿监测戒断症状；相应地对这些新生儿进行管理。

Risk of Serious Adverse Reactions in Infants due to Benzyl Alcohol Preservative

苯甲醇防腐剂导致婴儿严重不良反应的风险

VALTOCO is not approved for use in neonates or infants. Serious and fatal adverse reactions, including 'gasping syndrome,' can occur in neonates and low-birth-weight infants treated with benzyl alcohol–preserved drugs, including VALTOCO. The 'gasping syndrome' is characterized by central nervous system depression, metabolic acidosis, and gasping respirations.

VALTOCO未被批准用于新生儿或婴儿。使用含苯甲醇的药物（包括VALTOCO）治疗新生儿和低出生体重婴儿时，可能会发生严重甚至致命的不良反应，包括“喘息综合征”。“喘息综合征”的特征是中枢神经系统抑制、代谢性酸中毒和喘息样呼吸。

The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known..

未确定可能发生严重不良反应的苯甲醇最低量。

Adverse Reactions

不良反应

The most common adverse reactions (at least 4%) were somnolence, headache, and nasal discomfort.

最常见的不良反应（至少4%）为嗜睡、头痛和鼻部不适。

Diazepam, the active ingredient in VALTOCO, is a Schedule IV controlled substance.

地西泮是VALTOCO的活性成分，属于第四类管制物质。

To report SUSPECTED ADVERSE REACTIONS, contact Neurelis, Inc. at 1-866-696-3873 or FDA at 1-800-FDA-1088 (

要报告可疑的不良反应，请联系 Neurelis, Inc.，电话：1-866-696-3873，或 FDA，电话：1-800-FDA-1088 (

www.fda.gov/medwatch