BioArctic在美国获得exidavnemab的孤儿药资格认定-动脉网

BioArctic receives Orphan Drug Designation for exidavnemab the US

CISION 等信源发布 2025-03-18 01:54

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STOCKHOLM

斯德哥尔摩

，

March 17, 2025

2025年3月17日

/PRNewswire/ --

BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) announced today that the US FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation (ODD) to exidavnemab for the treatment of Multiple System Atrophy (MSA), providing incentives for the development of treatments for rare diseases with a high medical need.

BioArctic AB (publ)（纳斯达克斯德哥尔摩：BIOA B）今天宣布，美国 FDA 孤儿产品开发办公室 (OOPD) 已授予 exidavnemab 用于治疗多系统萎缩 (MSA) 的孤儿药资格 (ODD)，为开发针对高医疗需求的罕见病治疗方法提供了激励。

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Multiple System Atrophy (MSA) is a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems. MSA is characterized by pathological alpha-synuclein aggregation, that causes gradual damage to nerve cells in the brain. This affects balance, movement and the autonomic nervous system, which controls several basic functions, such as breathing, digestion and bladder control.

多系统萎缩（MSA）是一种快速进展且致命的罕见疾病，影响中枢和自主神经系统。MSA 的特征是病理性 α-突触核蛋白聚集，这会导致大脑中的神经细胞逐渐受损。这会影响平衡、运动以及控制呼吸、消化和膀胱功能等基本功能的自主神经系统。

Currently there is no cure and no available treatment to slow its progression..

目前尚无治愈方法，也没有可用的治疗手段来减缓其进展。

Exidavnemab is being developed as a novel disease-modifying treatment for synucleinopathies such as MSA and Parkinson's disease. It is a monoclonal antibody (mAb) that selectively targets soluble alpha-synuclein aggregates, such as oligomers or protofibrils. By promoting the clearance of aggregated alpha-synuclein, exidavnemab may reduce the spreading and the negative effects of alpha-synuclein.

Exidavnemab 正在被开发为一种针对突触核蛋白病（如多系统萎缩症和帕金森病）的新型疾病修饰治疗药物。它是一种单克隆抗体 (mAb)，能够选择性靶向可溶性α-突触核蛋白聚集体，例如寡聚体或原纤维。通过促进聚集的α-突触核蛋白的清除，exidavnemab 可能减少α-突触核蛋白的扩散及其负面影响。

Thereby, neuronal function and survival may be preserved, and disease progression ultimately slowed down..

因此，神经元的功能和存活可能得以保存，疾病进展最终得以减缓。

The FDA's Orphan Drug Designation program provides orphan status to drugs or biologics intended for the treatment of diseases that affect fewer than 200,000 people in

FDA的孤儿药指定计划为用于治疗在美国影响不到20万人的疾病的药物或生物制品提供孤儿药资格。

the United States

美国

. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives and regulatory assistance, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

获得孤儿药资格认定的药物赞助商将享有某些激励措施和监管协助，包括符合资格的临床试验税收抵免、处方药用户费用豁免，以及在FDA批准后可能获得七年的市场独占权。

This release discusses investigational uses of an agent in development and is not intended to convey conclusions about efficacy or safety. There is no guarantee that such investigational agents will successfully complete clinical development or gain health authority approval.

本发布稿讨论了正在开发中的药物的试验性用途，并非旨在传达有关有效性和安全性的结论。不能保证此类试验性药物将成功完成临床开发或获得卫生当局的批准。

This information is information that BioArctic AB (publ) is obliged to disclose pursuant to the EU Market Abuse Regulation. The information was released for public disclosure, through the agency of the contact person below, on

此信息是BioArctic AB（publ）根据欧盟市场滥用法规有义务披露的信息。该信息通过以下联系人发布，供公众查阅，于

March 17, 2025

2025年3月17日

, at

，在

18:25 CET

18:25 中欧时间

。

For further information, please contact:

如需更多信息，请联系：

Oskar Bosson

奥斯卡·博森

, VP Communications and IR

，副总裁，通信和投资者关系

Phone: +46 70 410 71 80

电话：+46 70 410 71 80

E-mail:

电子邮件：

oskar.bosson@bioarctic.com

Charlotte af Klercker, Senior Director Sustainability and Communications

夏洛特·阿夫·克莱克尔，可持续发展与传播高级总监

Telephone: +46 73 515 09 70

电话：+46 73 515 09 70

E-mail:

电子邮件：

charlotte.afklercker@bioarctic.com

夏洛特·阿夫克勒克@bioarctic.com

About MSA

关于MSA

Multiple System Atrophy (MSA) is a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems. MSA is a synucleinopathy, a group of neurodegenerative diseases characterized by an abnormal alpha-synuclein aggregation, that causes gradual damage to nerve cells in the brain.

多系统萎缩（MSA）是一种进展迅速且致命的罕见疾病，影响中枢和自主神经系统。MSA属于突触核蛋白病，这是一组以异常α-突触核蛋白聚集为特征的神经退行性疾病，会导致大脑中的神经细胞逐渐受损。

This affects balance, movement and the autonomic nervous system, which controls several basic functions, such as breathing, digestion and bladder control. Currently there is no cure and no available treatment to slow its progression..

这会影响平衡、运动和控制一些基本功能（如呼吸、消化和膀胱控制）的自主神经系统。目前尚无治愈方法，也没有可减缓其进展的可用治疗方案。

MSA is a condition with very high unmet medical need and poor prognosis. Currently, no cure or treatment is available to slow the progression of the disease. Patients typically live about 6 to 10 years after MSA symptoms first appear, with few patients surviving more than 15 years

MSA是一种具有很高未满足医疗需求且预后较差的疾病。目前尚无治愈方法或治疗手段可以减缓疾病的进展。患者通常在MSA症状首次出现后大约能存活6到10年，很少有患者能存活超过15年。

[1]

，

[2]

. MSA is significantly debilitating and classified as a rare disease, affecting less than 42,000 persons in the U.S.

MSA是一种严重的衰弱性疾病，被归类为罕见病，在美国影响不到42,000人。

About Exidavnemab

关于Exidavnemab

Exidavnemab is a monoclonal antibody drug candidate that is designed to selectively bind and eliminate aggregated forms of alpha-synuclein such as oligomers and protofibrils and fibrillar forms, which participates in neurodegenerative disorders including Parkinson's disease and MSA. The goal is to develop a disease modifying treatment that stops or slow down the progression of Parkinson's disease and MSA. .

Exidavnemab是一种单克隆抗体候选药物，旨在选择性结合并消除α-突触核蛋白的聚集形式，如寡聚体、原纤维和纤维形式，这些形式参与包括帕金森病和多系统萎缩（MSA）在内的神经退行性疾病。目标是开发一种能够阻止或减缓帕金森病和MSA进展的疾病修饰疗法。

About BioArctic AB

关于BioArctic AB

BioArctic AB (publ) is a Swedish research-based biopharma company focusing on innovative treatments that can delay or stop the progression of neurodegenerative diseases. The company is the originator of Leqembi® (lecanemab) – the world's first drug proven to slow the progression of the disease and reduce cognitive impairment in early Alzheimer's disease.

BioArctic AB（publ）是一家瑞典研究型生物制药公司，专注于开发能够延缓或阻止神经退行性疾病进展的创新疗法。该公司是Leqembi®（lecanemab）的创始者——这是全球首款被证明可以减缓疾病进展并在早期阿尔茨海默病中减少认知障碍的药物。

Leqembi has been developed together with Eisai. BioArctic has a broad research portfolio within Alzheimer's disease, Parkinson's disease, ALS and enzyme deficiency diseases. Several of the projects utilize the company's proprietary BrainTransporter™ technology, which improves the transport of drugs into the brain.

Leqembi 是与卫材共同开发的。BioArctic 在阿尔茨海默病、帕金森病、肌萎缩侧索硬化症（ALS）和酶缺乏疾病方面拥有广泛的研究组合。其中多个项目利用了该公司专有的 BrainTransporter™ 技术，该技术能够改善药物向大脑的输送。

BioArctic's B share (BIOA B) is listed on Nasdaq Stockholm Large Cap. For more information, please visit .

BioArctic的B股（BIOA B）在纳斯达克斯德哥尔摩大盘股上市。欲了解更多信息，请访问。

www.bioarctic.com.

[1]

Jellinger KA. J Alzheimers Dis. 2018;62(3):1141-1179.

扬林格 KA。《阿尔茨海默病杂志》。2018；62（3）：1141-1179。

[2]

Jellinger et al. Biomedicines. 2022 Mar 3;10(3):599.

杰林格等。《生物医学》。2022年3月3日；10(3):599。

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