Glycogen Storage Disease (GSD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Glycogen Storage Disease (GSD) Market Outlook

Thelansis’s “Glycogen Storage Disease (GSD) 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 Glycogen Storage Disease (GSD) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Glycogen Storage Disease (GSD) Overview

Glycogen storage diseases (GSDs) are hereditary disorders resulting from inborn errors in carbohydrate metabolism. These disorders lead to abnormal glycogen storage and are categorized as GSDs. They are assigned numerical classifications based on identifying the enzyme defect responsible for the condition. Clinical symptoms can manifest from infancy to adulthood. GSDs can arise due to the accumulation of toxic glycogen and the inability to convert glycogen into energy. However, in all cases, they result in an inability to utilize or store glycogen. There are various types of glycogen storage diseases, with the following being the most prevalent:

Glycogen storage disorders primarily affecting the liver encompass:

• Glycogen synthase-2 deficiency (GSD type 0a)
• Glucose-6-phosphatase deficiency (GSD type Ia)
• Glucose-6-phosphate transporter deficiency (GSD type Ib)
• Glycogen debrancher deficiency (GSD type III)
• Glycogen branching enzyme deficiency (GSD type IV)
• Liver phosphorylase deficiency (GSD type VI)
• Phosphorylase kinase deficiency (GSD type IXa)
• GLUT2 deficiency, also known as Fanconi-Bickel disease

Glycogen storage disorders primarily affecting skeletal muscles include:

• Muscle phosphorylase deficiency (GSD type V)
• Phosphofructokinase deficiency (GSD type VII)
• Phosphoglycerate mutase deficiency (GSD type X)
• Lactate dehydrogenase A deficiency (GSD type XI)
• Aldolase A deficiency (GSD type XII)
• Beta-enolase deficiency (GSD type XIII)
• Phosphoglucomutase-1 deficiency (GSD type XIV)

Glycogen storage disorders affecting both skeletal and cardiac muscles include:

• Lysosomal acid maltase deficiency (GSD type IIa)
• Lysosome-associated membrane protein 2 deficiency (GSD type IIb)
• Glycogenin-1 deficiency (GSD type XV)
• Muscle glycogen synthase deficiency (GSD type 0b)

With early detection and proper management, most GSDs have a favorable prognosis. Effective dietary management reduces complications associated with hypoglycemia and has shown promise in ameliorating renal dysfunction in GSD type I patients. In rare instances, end-stage renal disease necessitating kidney transplantation may arise in GSD type Ib patients. For individuals with GLUT2 deficiency, an antiketogenic diet can significantly reduce liver size and glycogen content. In GSD type Ia patients, growth delays, short stature, renal dysfunction, hypertriglyceridemia, and hepatocellular carcinoma may occur. On the other hand, GSD type Ib can lead to recurrent bacterial infections due to neutropenia. GSD type IV is associated with progressive liver failure and cirrhosis. Patients with GSD type II may develop cardiomyopathy and limb-girdle dystrophy, while GSD type III often presents with hypertrophic cardiomyopathy as a classic complication.

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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