Pompe Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Pompe Disease Market Outlook

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

Pompe Disease Overview

Pompe disease, also known as glycogen storage disease type II (GSD2), is a metabolic disorder caused by mutations in the GAA gene on chromosome 17q25.3. These mutations lead to the synthesis of an insufficient or absent form of the lysosomal enzyme alpha-glucosidase. This results in the accumulation of glycogen within lysosomes in muscular tissue, leading to impairments in striated muscular cells. GSD2 can present as either an early-onset (infantile, classic) or late-onset (non-classic) form. Early-onset GSD2 is severe and often fatal without prompt treatment, with respiratory insufficiency and left ventricular outflow obstruction as common causes of mortality. Late-onset GSD2 also results in respiratory insufficiency but at a later age. Enzyme replacement therapy (ERT) is currently the most effective treatment for GSD2. By introducing an analogous enzyme, lysosomal glycogen accumulation in cardiac and skeletal muscle is reduced. The timing of diagnosis is a critical factor in determining the effectiveness of treatment. Differential diagnoses for early-onset GSD2 include spinal muscular atrophy 1 and Danon disease, while limb-girdle muscular dystrophy and Duchenne-Becker muscular dystrophy are differentials for late-onset.

• The estimated incidence of infantile forms of Pompe disease ranges between 1.5 to 2.7 cases per every 120,000 births, and the late-onset form ranges between 1 to 2 cases per 60,000 births.

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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