Sandhoff Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Sandhoff Disease Market Outlook

Thelansis’s “Sandhoff Disease Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Sandhoff Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Sandhoff Disease Overview

Sandhoff disease, an inherited lysosomal storage disorder, stems from detrimental genetic mutations within the HEXB gene. This gene encodes instructions for an enzyme called β-hexosaminidase, pivotal in breaking down the fatty protein GM2 ganglioside, which is normally harmless after breakdown within cells. Nonetheless, in individuals with Sandhoff disease, this degradation process is impaired, leading to the accumulation of GM2 ganglioside and other molecules within the brain, causing damage to nerve cells. The disease is categorized into three types based on the onset of symptoms:

1. Infantile (Classic) Form - The most severe form of Sandhoff disease, the classic infantile type, becomes evident shortly after birth. Affected infants between three to six months of age lose developmental milestones and experience weakening muscles, hindering activities like rolling over, sitting up, and crawling. An exaggerated startle response to stimuli emerges. Seizures, vision and hearing loss, intellectual disabilities, paralysis, enlarged organs, bone abnormalities, and the distinctive "cherry-red spot" in the eye characterize the progression of the disease.
2. Juvenile-Onset Form - A rarer variant occurs when mutations only partially compromise the enzyme. Signs and symptoms vary, and onset may be in childhood or adolescence. Muscle weakness, coordination difficulties, speech problems, recurrent respiratory infections, and seizures are some manifestations of these cases.
3. Late-Onset Form - Diagnosing the late-onset form can be challenging. Initial indicators include clumsiness and leg muscle weakness. Over time, affected individuals may necessitate mobility aid and struggle with speech and swallowing. Mental health issues, like bipolar disorder or psychotic episodes, affect about 40% of afflicted adults.

Sandhoff disease mirrors Tay-Sachs's clinical presentation, featuring startle reactions, early blindness, progressive motor and mental decline, enlarged head, cherry-red spots on the macula, doll-like facial appearance, and organ enlargement. Urinary oligosaccharide levels are elevated. Initial normal development in children is followed by rapid disease evolution. However, signs might resemble spinocerebellar ataxia or dystonia in cases with later onset or in adults, with variable impact on intellectual capacities. Treatment entails supportive care for symptoms, including seizure management, nutritional support, and breathing assistance. Unfortunately, a cure is lacking. Severe infantile-onset cases witness recurrent seizures by age two, leading to muscle and mental function loss, eventually becoming unresponsive and succumbing to respiratory infections by age three. Juvenile-onset cases experience similar issues at a later age but decline progressively. Late-onset cases, while challenging, do not necessarily curtail lifespan.

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: 2022-2032

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.

Visit our social media pages: