Spinocerebellar Ataxias Clinical Trials

DelveInsight’s analysis highlights that over 8 key companies are actively engaged in developing more than 10 treatment therapies for Spinocerebellar Ataxias.

Spinocerebellar Ataxias Overview:

Ataxia is characterized by a loss of voluntary muscle coordination, resulting in impaired control of movement, gait instability, abnormal eye movements, and speech difficulties. Spinocerebellar ataxia (SCA) is a rare, progressive, inherited neurodegenerative disorder with an autosomal dominant inheritance pattern that primarily affects the cerebellum. It is part of the broader group of hereditary cerebellar ataxias. To date, more than 40 genetically distinct types of SCA have been identified, classified by their genetic loci in order of discovery, with SCA1 being the first described subtype.

The exact disease mechanisms are not fully understood, but several pathways are implicated. Genetic mutations may cause abnormal protein production, transcriptional dysregulation, defective autophagy, ion channel dysfunction, mitochondrial impairment, and toxic RNA gain-of-function effects. Abnormal proteins, such as ataxins, are usually degraded by the ubiquitin-proteasome system; however, they may form aggregates instead. These mutant ataxins also interact abnormally with other proteins like the TATA-binding transcription factor and CREB-binding protein, leading to disrupted transcription and unregulated gene expression.

Many SCAs demonstrate anticipation, where trinucleotide repeat expansions increase with each generation, worsening disease onset and severity. Expanded CAG repeats are characteristic of SCA1, 2, 3, 6, 7, 8, 12, and 17. Other subtypes involve different repeat expansions, such as ATTCT in SCA10, TGGAA in SCA31, GGCCTG in SCA36, and ATTTT in SCA37. Additionally, some SCAs are linked to single nucleotide variants or structural genomic changes—for example, SCA5, 13, 14, and 19 arise from missense mutations, while SCA15, 20, and 39 are associated with gene deletions or duplications.

Currently, there is no cure for SCA, and treatment focuses on symptom management. Supportive therapies include antiepileptic drugs for seizures, botulinum toxin for dystonia, beta-blockers or primidone for tremors, antidepressants for mood disorders, and levodopa for parkinsonism. These interventions aim to improve quality of life by addressing complications rather than halting disease progression.

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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

• DelveInsight’s Spinocerebellar Ataxias (SCA) pipeline report highlights a strong therapeutic landscape, with 8+ companies actively developing over 10 potential treatment candidates.

• Leading players such as Biohaven Pharmaceuticals, Vico Therapeutics B.V., Steminent Biotherapeutics, and others are advancing novel therapies aimed at improving outcomes for patients with SCA.

• Promising candidates in development include Troriluzole, VO659, and several others in different clinical stages.

• In the fourth quarter of 2024, Biohaven submitted a New Drug Application (NDA) to the U.S. FDA for troriluzole, seeking approval across all SCA genotypes. The application qualifies for priority review, supported by the FDA’s prior orphan drug and fast-track designations for the therapy.

Spinocerebellar Ataxias Pipeline Analysis

The report provides insights into:

• The report provides detailed insights into the key companies that are developing therapies in the Spinocerebellar Ataxias Market.

• The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinocerebellar Ataxias treatment.

• It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• 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.

• 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

• VO659: Vico Therapeutics B. V.

Spinocerebellar Ataxias Companies

There are over eight major companies actively developing therapies for Spinocerebellar Ataxias. Among them, Biohaven Pharmaceuticals has the most advanced drug candidates, currently in Phase III clinical trials.

DelveInsight’s report covers around 10+ products under different phases of clinical development like

• Late stage products (Phase III)

• Mid-stage products (Phase II)

• Early-stage product (Phase I) along with the details of

• Pre-clinical and Discovery stage candidates

• 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

• Intravenous

• Subcutaneous

• Oral

• Intramuscular

Spinocerebellar Ataxias Products have been categorized under various Molecule types such as

• Monoclonal antibody

• Small molecule

• 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

Download Spinocerebellar Ataxias Sample report to know in detail about the Spinocerebellar Ataxias treatment market @ Spinocerebellar Ataxias Therapeutic Assessment

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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About DelveInsight

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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