DelveInsight’s analysis reveals that the Spinocerebellar Ataxias pipeline features over 8 key companies actively engaged in developing more than 10 therapeutic candidates targeting the disorder.
Spinocerebellar Ataxias Overview:
Spinocerebellar ataxia (SCA) is a rare, hereditary neurodegenerative disease that progressively affects the cerebellum and, in some cases, other parts of the central nervous system. It encompasses more than 40 genetic subtypes, with SCA1, SCA2, SCA3, and SCA6 being the most frequently observed. The condition is characterized by a gradual decline in voluntary motor coordination, impacting balance, speech, and eye movement. Although the cerebellum and spinal cord are primarily involved, other brain regions such as the basal ganglia, cerebral cortex, and peripheral nerves may also be affected, leading to wide clinical variability. Disease progression typically results in significant motor impairment, with onset age and symptom severity varying widely—even within the same family—making diagnosis and management challenging.
The pathogenesis of SCA is complex and multifactorial. It is most often associated with genetic mutations involving CAG trinucleotide repeat expansions, which produce abnormal ataxin proteins. These misfolded proteins accumulate in Purkinje cells, disrupting their function and initiating neurodegeneration. As Purkinje cells are critical for motor coordination, their loss manifests as the hallmark ataxic symptoms. Additional cellular dysfunctions—including impaired autophagy, mitochondrial defects, transcriptional dysregulation, and ion channel abnormalities—further contribute to neuronal damage. The degenerative process can extend to other CNS structures such as the brainstem and basal ganglia, resulting in a broad range of neurological impairments.
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"Spinocerebellar Ataxias Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Spinocerebellar Ataxias Therapeutics Market.
Key Takeaways from the Spinocerebellar Ataxias Pipeline Report
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DelveInsight’s Spinocerebellar Ataxias pipeline report highlights a dynamic landscape with over 8 active companies developing more than 10 investigational therapies for Spinocerebellar Ataxias treatment.
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In May 2025, the FDA extended the PDUFA decision date by three months—shifting the target to Q4 2025—to allow for additional data review, with an advisory committee meeting being planned. Earlier, in February 2025, the FDA accepted the New Drug Application (NDA) for troriluzole and granted it Priority Review, with a decision anticipated in Q3 2025.
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Leading companies such as Biohaven Pharmaceuticals, Steminent US, Vico Therapeutics B.V., Arrowhead Pharmaceuticals, Neurolixis, Latus Bio, AAV-CB, and others are actively advancing therapies to enhance treatment options for Spinocerebellar Ataxias.
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Notable pipeline candidates at various stages of development include Troriluzole, Stemchymal, VO659, and several others.
Spinocerebellar Ataxias Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Spinocerebellar Ataxias Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinocerebellar Ataxias treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Spinocerebellar Ataxias market.
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Spinocerebellar Ataxias Emerging Drugs
Troriluzole: Biohaven Pharmaceuticals, Inc.
Troriluzole is a novel chemical entity (NCE) and a third-generation prodrug developed to modulate glutamate—the most abundant excitatory neurotransmitter in the brain. It functions by reducing synaptic glutamate levels through the upregulation and enhanced activity of excitatory amino acid transporters (EAATs) on glial cells, which clear excess glutamate from the synaptic cleft. This mechanism positions Troriluzole as a potential therapy for several neurological disorders associated with glutamate dysregulation.
In February 2025, Biohaven Ltd. announced that the U.S. Food and Drug Administration (FDA) had accepted its New Drug Application (NDA) for Troriluzole for the treatment of spinocerebellar ataxia in adults.
Stemchymal: Steminent US, Inc.
Stemchymal is an allogeneic stem cell therapy, meaning it uses stem cells derived from healthy donors to treat patients. The treatment employs mesenchymal stem cells (MSCs) sourced from adipose (fat) tissue, which are processed using a proprietary system and administered via intravenous (IV) infusion. MSCs have low immunogenicity, reducing the likelihood of immune rejection in recipients.
Preclinical studies have shown that MSC infusions can provide therapeutic benefits, including partial restoration of motor function in mouse models of spinocerebellar ataxia (SCA). Stemchymal has been granted orphan drug designation in Japan and is currently in Phase II clinical trials for SCA treatment.
Spinocerebellar Ataxias Companies
Around eight or more leading companies are currently engaged in developing therapies for Spinocerebellar Ataxias. Among these, Biohaven Pharmaceuticals, Inc. has the most advanced drug candidate, which has progressed to the Regulatory Registration stage of development.
DelveInsight’s report covers around 75+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Spinocerebellar Ataxias pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Spinocerebellar Ataxias Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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Spinocerebellar Ataxias Pipeline Therapeutic Assessment
• Spinocerebellar Ataxias Assessment by Product Type
• Spinocerebellar Ataxias By Stage
• Spinocerebellar Ataxias Assessment by Route of Administration
• Spinocerebellar Ataxias Assessment by Molecule Type
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Table of Content
1. Report Introduction
2. Executive Summary
3. Spinocerebellar Ataxias Current Treatment Patterns
4. Spinocerebellar Ataxias - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Spinocerebellar Ataxias Late-Stage Products (Phase-III)
7. Spinocerebellar Ataxias Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Spinocerebellar Ataxias Discontinued Products
13. Spinocerebellar Ataxias Product Profiles
14. Spinocerebellar Ataxias Key Companies
15. Spinocerebellar Ataxias Key Products
16. Dormant and Discontinued Products
17. Spinocerebellar Ataxias Unmet Needs
18. Spinocerebellar Ataxias Future Perspectives
19. Spinocerebellar Ataxias Analyst Review
20. Appendix
21. Report Methodology
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DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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