Pseudoxanthoma Elasticum (PXE) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Pseudoxanthoma Elasticum (PXE) Market Outlook

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

Pseudoxanthoma Elasticum (PXE) Overview

Pseudoxanthoma elasticum (PXE) arises from mutations in the ABCC6 gene and is a hereditary metabolic disorder. It is characterized by the gradual calcification and breakage of elastic fibers in the skin, retina, and arterial walls. Typically, this condition manifests during adolescence or young adulthood, but it can emerge at any stage of life. The initial clinical indication is nearly always the appearance of small yellow papules on the back of the neck, sides of the neck, and in areas where the skin flexes. In some individuals, PXE may be confined to a single organ (skin, eyes, or blood vessels); in others, it can affect two or all three of these organs. Although cardiac issues like angina pectoris and myocardial infarction are relatively uncommon, they require thorough examination when present. Most PXE cases stem from two pathogenic variations in the ABCC6 gene, located at 16p13.11. In rare instances, patients displaying clinical features resembling PXE may possess pathogenic variations in the ENPP1 gene at 6q23.2, typically associated with generalized arterial calcification of infancy (GACI). There is no specific cure for PXE, and treatment primarily addresses symptoms. This can involve direct injections of vascular endothelial growth factor inhibitors into the eyes to manage choroidal neovascularization, adopting lifestyle changes, implementing lipid-lowering measures and dietary adjustments to reduce vascular risk factors, performing vascular surgery in cases of severe arteriosclerosis, and opting for plastic surgery to address excess skin folds. PXE is a progressive, lifelong condition with significant potential for debilitation, but most patients can expect a normal lifespan.

• According to the literature review conducted by Thelansis, the prevalence of PXE, estimated to be 1 in 50,000, would imply that there are ~8,000–10,000 PXE patients in the United States. With the global prevalence, as many as 160,000 to 200,000 patients could be affected by PXE worldwide.

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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