BioArctic receives Orphan Drug Designation for exidavnemab the US
BioArctic AB (Nasdaq Stockholm: BIOA B) has received Orphan Drug Designation from the US FDA for exidavnemab, targeting the treatment of Multiple System Atrophy (MSA). MSA is a rare, fatal disease affecting the central and autonomic nervous systems, characterized by alpha-synuclein aggregation that damages brain nerve cells.
Exidavnemab is being developed as a disease-modifying treatment for synucleinopathies including MSA and Parkinson's disease. As a monoclonal antibody, it selectively targets soluble alpha-synuclein aggregates to potentially reduce their spreading and negative effects.
The FDA's Orphan Drug Designation provides several benefits, including:
- Tax credits for qualified clinical trials
- Prescription drug user-fee exemptions
- Potential seven-year marketing exclusivity upon FDA approval
BioArctic AB (Nasdaq Stockholm: BIOA B) ha ricevuto la Designazione di Farmaco Orfano dalla FDA degli Stati Uniti per exidavnemab, mirato al trattamento della Atrofia Muscolare Multipla (MSA). La MSA è una malattia rara e fatale che colpisce i sistemi nervoso centrale e autonomo, caratterizzata dall'aggregazione dell'alfa-sinucleina che danneggia le cellule nervose del cervello.
Exidavnemab è in fase di sviluppo come trattamento modificante la malattia per le sinucleinopatie, inclusa la MSA e il morbo di Parkinson. Come anticorpo monoclonale, mira selettivamente agli aggregati solubili di alfa-sinucleina per ridurre potenzialmente la loro diffusione e gli effetti negativi.
La Designazione di Farmaco Orfano della FDA offre diversi vantaggi, tra cui:
- Crediti d'imposta per studi clinici qualificati
- Esenzioni dalle tasse sui farmaci da prescrizione
- Potenziale esclusività di marketing di sette anni dopo l'approvazione della FDA
BioArctic AB (Nasdaq Estocolmo: BIOA B) ha recibido la Designación de Medicamento Huérfano de la FDA de EE. UU. para exidavnemab, dirigido al tratamiento de Atrofia Multisistémica (MSA). La MSA es una enfermedad rara y fatal que afecta los sistemas nervioso central y autónomo, caracterizada por la agregación de alfa-sinucleína que daña las células nerviosas del cerebro.
Exidavnemab se está desarrollando como un tratamiento modificador de la enfermedad para las sinucleinopatías, incluida la MSA y la enfermedad de Parkinson. Como anticuerpo monoclonal, se dirige selectivamente a los agregados solubles de alfa-sinucleína para reducir potencialmente su propagación y efectos negativos.
La Designación de Medicamento Huérfano de la FDA proporciona varios beneficios, que incluyen:
- Créditos fiscales para ensayos clínicos calificados
- Exenciones de tarifas de medicamentos recetados
- Potencial exclusividad de comercialización de siete años tras la aprobación de la FDA
BioArctic AB (나스닥 스톡홀름: BIOA B)는 exidavnemab에 대해 미국 FDA로부터 희귀의약품 지정을 받았습니다. 이는 다계통 위축증 (MSA) 치료를 목표로 하고 있습니다. MSA는 중추 신경계와 자율 신경계를 침범하는 희귀하고 치명적인 질병으로, 알파-시누클레인 집합체가 뇌 신경 세포를 손상시키는 것이 특징입니다.
Exidavnemab는 MSA와 파킨슨병을 포함한 시누클레인병을 위한 질병 수정 치료제로 개발되고 있습니다. 이 약물은 단클론 항체로서, 용해된 알파-시누클레인 집합체를 선택적으로 표적하여 그 확산과 부정적인 영향을 줄일 수 있습니다.
FDA의 희귀의약품 지정은 다음과 같은 여러 혜택을 제공합니다:
- 자격을 갖춘 임상 시험에 대한 세금 공제
- 처방약 사용자 수수료 면제
- FDA 승인이 시판 독점권을 제공할 수 있는 7년의 잠재력
BioArctic AB (Nasdaq Stockholm : BIOA B) a reçu la Désignation de Médicament Orphelin de la FDA des États-Unis pour exidavnemab, visant à traiter l'Atrophie Multisystémique (MSA). La MSA est une maladie rare et mortelle affectant les systèmes nerveux central et autonome, caractérisée par l'agrégation d'alfa-synucléine qui endommage les cellules nerveuses du cerveau.
Exidavnemab est en cours de développement en tant que traitement modificateur de la maladie pour les synucléinopathies, y compris la MSA et la maladie de Parkinson. En tant qu'anticorps monoclonal, il cible sélectivement les agrégats solubles d'alfa-synucléine afin de réduire potentiellement leur propagation et leurs effets négatifs.
La désignation de médicament orphelin de la FDA offre plusieurs avantages, notamment :
- Crédits d'impôt pour les essais cliniques qualifiés
- Exemptions de frais pour les médicaments sur ordonnance
- Possibilité d'une exclusivité de commercialisation de sept ans après approbation par la FDA
BioArctic AB (Nasdaq Stockholm: BIOA B) hat von der US FDA die Orphan Drug Designation für exidavnemab erhalten, das auf die Behandlung von Multisystematrophie (MSA) abzielt. MSA ist eine seltene, tödliche Krankheit, die das zentrale und autonome Nervensystem betrifft und durch die Aggregation von Alpha-Synuclein gekennzeichnet ist, die Nervenzellen im Gehirn schädigt.
Exidavnemab wird als krankheitsmodifizierende Behandlung für Synucleinopathien, einschließlich MSA und Parkinson-Krankheit, entwickelt. Als monoklonaler Antikörper zielt es selektiv auf lösliche Alpha-Synuclein-Aggregate ab, um deren Ausbreitung und negative Auswirkungen potenziell zu reduzieren.
Die Orphan Drug Designation der FDA bietet mehrere Vorteile, darunter:
- Steuergutschriften für qualifizierte klinische Studien
- Ausnahmen von Gebühren für verschreibungspflichtige Medikamente
- Potenzielle siebenjährige Marktexklusivität nach Genehmigung durch die FDA
- Received FDA Orphan Drug Designation for exidavnemab, providing valuable development incentives
- Potential 7-year market exclusivity upon FDA approval
- Tax benefits and fee exemptions for clinical development
- Drug is still in investigational phase with no guaranteed success
- No current proof of efficacy or safety in treating MSA
Insights
BioArctic's achievement of Orphan Drug Designation (ODD) for exidavnemab represents a significant regulatory milestone in their development pathway for Multiple System Atrophy (MSA) treatment. This rare designation brings substantial economic incentives that materially improve the drug's development economics:
The ODD provides concrete benefits including tax credits for clinical trials, prescription drug user fee exemptions, and potentially seven years of market exclusivity upon approval. These advantages effectively reduce development costs while simultaneously enhancing commercial potential through extended protection from competition.
MSA represents a critical unmet medical need - a rapidly progressive, fatal neurological disease currently without any disease-modifying treatments. The monoclonal antibody approach targeting alpha-synuclein aggregates addresses the core pathology of MSA.
Particularly notable is BioArctic's strategic positioning of exidavnemab for both MSA and the substantially larger Parkinson's disease market, potentially leveraging the same mechanism across multiple indications, which could significantly expand the commercial application beyond the rare disease space.
While this designation doesn't guarantee clinical success or eventual approval, it does provide a clearer regulatory pathway and indicates FDA recognition of the critical need for therapies addressing this devastating condition.
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. Currently there is no cure and no available treatment to slow its progression.
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. Thereby, neuronal function and survival may be preserved, and disease progression ultimately slowed down.
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
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.
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 March 17, 2025, at 18:25 CET.
For further information, please contact:
Oskar Bosson, VP Communications and IR
Phone: +46 70 410 71 80
E-mail: oskar.bosson@bioarctic.com
Charlotte af Klercker, Senior Director Sustainability and Communications
Telephone: +46 73 515 09 70
E-mail: charlotte.afklercker@bioarctic.com
About 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. 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.
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[1],[2]. MSA is significantly debilitating and classified as a rare disease, affecting less than 42,000 persons in the
About 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.
About 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. 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. BioArctic's B share (BIOA B) is listed on Nasdaq Stockholm Large Cap. For more information, please visit www.bioarctic.com.
[1] Jellinger KA. J Alzheimers Dis. 2018;62(3):1141-1179.
[2] Jellinger et al. Biomedicines. 2022 Mar 3;10(3):599.
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FAQ
What benefits does the FDA Orphan Drug Designation provide for BioArctic's exidavnemab (BIOA)?
How does exidavnemab work in treating Multiple System Atrophy (MSA)?
What is Multiple System Atrophy (MSA) and who does it affect?
What conditions is BioArctic's exidavnemab (BIOA) being developed to treat?
