Giant Axonal Neuropathy (GAN) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Giant Axonal Neuropathy (GAN) Market Outlook

Thelansis’s “Giant Axonal Neuropathy (GAN) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" 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 Giant Axonal Neuropathy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Giant Axonal Neuropathy (GAN) Overview

Giant Axonal Neuropathy (GAN) is a rare autosomal recessive neurodegenerative disorder impacting both the central and peripheral nervous systems, typically characterized by progressive weakness and ataxia. Alongside these symptoms, individuals experience sensory loss and eventual loss of ambulation, often leading to respiratory failure. The condition stems from an anomaly within the GAN gene, found on chromosome 16 at 16q24.1, responsible for encoding the gigaxonin protein. This abnormal protein results in the swelling of axons, where tiny thread-like deposits of neurofilaments accumulate, creating the appearance of giant axons. Consequently, this leads to degeneration and dysfunctional activity within the peripheral nervous system. Neuropathological investigations have revealed a loss of gigaxonin expression affecting regions such as the cerebellar cortex, brainstem, and posterior columns of the spinal cord. Consequently, this disorder is considered a promising candidate for intrathecal delivery of AAV9 gene transfer, aiming to restore gigaxonin function. Gigaxonin plays a crucial role in organizing and degrading intermediate filaments. Thus, its absence leads to axonal enlargement characterized by densely bundled intermediate filaments. Standard features of Giant Axonal Neuropathy include hypotonia (low muscle tone), muscle weakness, diminished reflexes, ataxia, seizures, and intellectual disability. Regrettably, there are currently no FDA-approved treatments for GAN. Management primarily revolves around counseling and symptomatic approaches, including bracing, physical therapy, aquatic therapy, and pain management.

• In the United States, the prevalence of Giant Axonal Neuropathy ranges from 0.4 to 0.78 cases per million population.

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: 2023-2033

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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