Spinocerebellar Ataxia (SCA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Spinocerebellar ataxia (SCA) Market Outlook and Forecast

Spinocerebellar Ataxia (SCA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Published Date : 2021-12-07

Updated On : 2023-08-22

Pages : 154

Spinocerebellar Ataxia (SCA) Market Outlook

Thelansis’s “Spinocerebellar Ataxia (SCA) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Spinocerebellar Ataxia (SCA) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Spinocerebellar Ataxia (SCA) Overview

Spinocerebellar ataxia (SCA) represents a diverse and heterogeneous collection of autosomal dominant degenerative disorders. These conditions are characterized by the progressive degeneration of the cerebellum and its associated nerve connections, along with typical involvement of structures like the basal ganglia, brainstem nuclei, pyramidal tracts, posterior column, anterior horn, peripheral nerves, and other parts of the nervous system. Clinically, SCAs manifest as cerebellar gait and limb ataxia, accompanied by various associated symptoms such as dysmetria, dysdiadochokinesia, intention tremor, dysarthria, and nystagmus. Additionally, extracerebellar signs may emerge, including ophthalmoplegia, pyramidal signs, movement disorders (like parkinsonism, dystonia, myoclonia, and chorea), dementia, epilepsy, visual impairments (such as pigmentary retinopathy), lower motor neuron disease, and peripheral neuropathy. Several types of spinocerebellar ataxia exhibit a phenomenon known as anticipation, marked by a tendency for gradual expansion of CAG repeats across successive generations. Notably, CAG repeat expansion is observed in SCA1, 2, 3, 6, 7, 8, 12, and 17. Similarly, SCA 10 is attributed to the expansion of ATTCT (a pentanucleotide repeat), while SCA 31, 36, and 37 involve amplification of TGGAA (pentanucleotide repeat), GGCCTG (hexanucleotide repeat), and ATTTT (pentanucleotide repeat), respectively. Currently, no FDA-approved treatments can address the underlying causes of SCAs. The primary approach to managing these conditions is symptomatic, aimed at alleviating specific symptoms such as seizures, tremors, depression, ataxia, and eye-related issues. This may involve using antiepileptic drugs for seizures, botulinum toxin injections for dystonia, beta-blockers, primidone for tremors, antidepressants for depression, and levodopa for parkinsonism, among other interventions.

  • SCA accounts for 3% to 5% of the ataxic diagnosed population, with an estimated prevalence of 1 to 5 cases in 100,000 people.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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Spinocerebellar Ataxia (SCA) Competitive Landscape

S. no Asset Company Stage
1 SLS-005 Seelos Therapeutics, Inc. Phase 3
2 Troriluzole Biohaven Pharmaceuticals, Inc. Phase 3
3 VO659 Vico Therapeutics B. V. Phase 2
4 Stemchymal® Steminent Biotherapeutics Inc. Phase 2

KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

Spinocerebellar Ataxia (SCA) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Spinocerebellar ataxia (SCA) Market Forecast

1.       Spinocerebellar Ataxia (SCA) – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. Spinocerebellar Ataxia (SCA) market scenario 2021
                     1.2.2. Spinocerebellar Ataxia (SCA) market scenario 2025
                     1.2.3. Spinocerebellar Ataxia (SCA) market scenario 2032

2.       Spinocerebellar Ataxia (SCA) Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for Spinocerebellar Ataxia (SCA)
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. Spinocerebellar Ataxia (SCA) Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in Spinocerebellar Ataxia (SCA) management
         2.16.  Market Opportunity for Spinocerebellar Ataxia (SCA)
         2.17. KOL Comments on current and upcoming/expected treatment practices in Spinocerebellar Ataxia (SCA)

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1.  Spinocerebellar Ataxia (SCA) Epidemiology in US (2021-2032)
                             3.2.1.1.              Incidence of Spinocerebellar Ataxia (SCA)
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2.  Spinocerebellar Ataxia (SCA) Epidemiology in EU-5 (2021-2032)
                             3.2.2.1.              Incidence of Spinocerebellar Ataxia (SCA)
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3.  Spinocerebellar Ataxia (SCA) Epidemiology in Japan (2021-2032)
                             3.2.3.1.              Incidence of Spinocerebellar Ataxia (SCA)
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4.  Spinocerebellar Ataxia (SCA) Epidemiology in China (2021-2032)
                             3.2.4.1.              Incidence of Spinocerebellar Ataxia (SCA)
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China Spinocerebellar Ataxia (SCA) Market Forecast 2021-2032
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in Spinocerebellar Ataxia (SCA)
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from Spinocerebellar Ataxia (SCA) Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in Spinocerebellar Ataxia (SCA)
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on Spinocerebellar Ataxia (SCA) Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer