Praxis Precision Medicines宣布FDA接受Ulixacaltamide HCl用于治疗原发性震颤患者的新药申请-动脉网

Praxis Precision Medicines Announces FDA Acceptance of New Drug Application for Ulixacaltamide HCl in Patients with Essential Tremor

Praxis 等信源发布 2026-04-14 23:58

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FDA assigned PDUFA target action date of

FDA指定的PDUFA目标行动日期为

January 29, 2027

2027年1月29日

No advisory committee meeting expected

不召开咨询委员会会议

BOSTON

波士顿

，

April 14, 2026

2026年4月14日

(GLOBE NEWSWIRE) --

（环球新闻网）--

Praxis Precision Medicines

实践精准医药

, Inc. (NASDAQ:

，公司（纳斯达克：

PRAX

实践

), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced that the

），一家完全整合的领先的中枢神经系统（CNS）精准神经科学生物制药公司，今天宣布了

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) has accepted for review its New Drug Application (NDA) for ulixacaltamide HCl for the treatment of essential tremor (ET) in adults. The FDA has set a target action date under the Prescription Drug User Fee Act (PDUFA) of

美国食品药品监督管理局（FDA）已接受其新药申请（NDA），用于审核盐酸乌利沙酰胺治疗成人原发性震颤（ET）。FDA根据《处方药使用者费用法案》（PDUFA）设定了目标行动日期。

January 29, 2027

2027年1月29日

and is not planning to hold an advisory committee meeting.

并不计划召开咨询委员会会议。

“Today’s announcement brings us one step closer to delivering something patients living with essential tremor have been waiting for, a therapy developed specifically for their condition,” said

“今天的公告使我们距离为特发性震颤患者提供他们期待已久的疗法更近了一步，这种疗法是专门为他们的病情开发的，”

Marcio Souza

马辛霍·苏扎

, president and chief executive officer. “We look forward to continuing to work with the FDA through the review process while we prepare for the commercial launch.”

，总裁兼首席执行官。“我们期待在准备商业发布的同时，继续与FDA合作完成审查过程。”

Ulixacaltamide for treatment of essential tremor

乌利沙卡酰胺治疗原发性震颤

The NDA is supported by positive results from the Essential3 Phase 3 program, which comprised two simultaneously enrolled pivotal studies in adults with essential tremor. The statistically and clinically significant

新药申请（NDA）得到了Essential3第三阶段项目的积极结果的支持，该项目包括两项同时进行的、针对患有特发性震颤的成人的关键研究。统计学和临床上显著的

results

结果

from the Essential3 program provide the primary evidence of effectiveness for the NDA submission. Ulixacaltamide was generally well tolerated, with a safety profile consistent with previous trials and no drug-related serious adverse events. Ulixacaltamide received Breakthrough Therapy Designation (BTD) from the FDA in .

来自Essential3项目的主要证据证明了NDA提交的有效性。Ulixacaltamide通常耐受性良好，其安全性与之前的试验一致，且没有药物相关的严重不良事件。Ulixacaltamide获得了FDA的突破性疗法认定（BTD）。

December 2025

2025年12月

。

About Ulixacaltamide

关于乌利沙酰胺

Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide has received Breakthrough Therapy Designation from the FDA and is the most advanced program within Praxis’ Cerebrum™ small molecule platform..

Ulixacaltamide 是一种差异化且高度选择性的小分子T型钙通道抑制剂，旨在阻断与震颤活动相关的脑小脑-丘脑-皮层（CTC）回路中的异常神经元突发放电。Ulixacaltamide 已获得FDA的突破性疗法认定，是Praxis公司Cerebrum™小分子平台中最先进的项目。

About Praxis

关于实践

Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain.

Praxis Precision Medicines是一家完全整合的领先中枢神经系统（CNS）精准神经科学生物制药公司，将遗传性癫痫的见解转化为针对以神经元兴奋-抑制失衡为特征的中枢神经系统疾病的疗法开发。Praxis通过我们专有的小分子平台Cerebrum™和反义寡核苷酸（ASO）平台Solidus™，利用对大脑中共有生物靶点和回路的理解，将基因洞察应用于罕见及更普遍的神经系统疾病疗法的发现与开发。

Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four late-stage product candidates. For more information, please visit .

Praxis 已建立了多元化的、多模式的中枢神经系统（CNS）产品组合，涵盖运动障碍和癫痫的多个项目，拥有四种处于后期阶段的产品候选药物。欲了解更多信息，请访问。

www.praxismedicines.com

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Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the anticipated timing of clinical trials, the anticipated timing of regulatory submissions and interactions and potential market opportunity and commercial potential of Praxis’ product candidates, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995. .

本新闻稿包含《1995年私人证券诉讼改革法案》及其他联邦证券法所指的前瞻性声明，包括明示或暗示的关于Praxis未来预期、计划和前景的声明，特别是有关临床试验预计时间、监管提交及互动的预期时间，以及Praxis产品候选者的潜在市场机会和商业潜力的声明。此外，其他包含“预期”、“相信”、“继续”、“可能”、“努力”、“估计”、“预计”、“打算”、“或许”、“计划”、“潜力”、“预测”、“预计”、“寻求”、“应该”、“目标”、“将”或“会”等词汇及其类似表达的声明均构成《1995年私人证券诉讼改革法案》所指的前瞻性声明。

The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2025 and other filings made with the Securities and Exchange Commission.

本新闻稿中包含的明示或暗示的前瞻性陈述仅为预测，且受多种风险、不确定性和假设的影响，包括但不限于：临床试验固有的不确定性；临床试验、数据读取及其结果的预期时间安排，以及向监管机构或政府当局提交审批或审查的时间；进行试验所需的监管批准；以及Praxis在其截至2025年12月31日的年度报告Form 10-K及其他向证券交易委员会提交的文件中所述的有关其项目和运营的其他风险。

Although Praxis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made.

尽管 Praxis 的前瞻性陈述反映了其管理层的善意判断，但这些陈述仅基于 Praxis 目前已知的信息和因素。因此，提醒您不要依赖这些前瞻性陈述。本新闻稿中发布的任何前瞻性陈述仅在发布之日有效。

Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise..

实践不承担公开更新或修改任何前瞻性陈述的义务，无论是由于新信息、未来发展或其他原因。

Investor Contact:

投资者联系人：

Praxis Precision Medicines

实践精准医疗

investors@praxismedicines.com

投资者@普拉克西斯医药.com

857-702-9452

Media Contact:

媒体联系人：

Dan Ferry

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