Pseudoxanthoma Elasticum Market

Market Overview:

The 7 major pseudoxanthoma elasticum markets are expected to exhibit a CAGR of 1.21% during 2024-2034.

The pseudoxanthoma elasticum market has been comprehensively analyzed in IMARC's new report titled "Pseudoxanthoma Elasticum Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Pseudoxanthoma elasticum (PXE) refers to a rare hereditary disorder that primarily affects the elastic tissues in various parts of the body, particularly the skin, eyes, and blood vessels. The condition is characterized by the progressive mineralization and fragmentation of elastic fibers, leading to a loss of flexibility and function in affected tissues. The symptoms of PXE can vary widely but commonly include yellowish, cobblestone-like bumps on the skin, particularly in areas exposed to the sun, such as the neck and underarm. Additionally, individuals suffering from the illness may experience changes in their vision with the development of yellowish deposits in the retina that can result in blurred or distorted vision. Cardiac complications, such as hypertension and irregular heart rhythms, as well as gastrointestinal issues, may also arise due to the abnormal elastic tissue deposition in blood vessels and several other organs. The diagnosis of PXE typically relies on a comprehensive evaluation of clinical features, medical history, and specialized imaging tests.

The escalating cases of genetic mutations that affect the ABCC6 gene, resulting in the aberrant mineralization of connective tissues, mainly in the skin, eyes, and blood vessels, are primarily driving the pseudoxanthoma elasticum market. In addition to this, the increasing utilization of efficacious medications, such as calcium channel blockers, bisphosphonates, anti-angiogenic agents, etc., to manage symptoms and mitigate the progression of the condition is also creating a positive outlook for the market. Moreover, the widespread adoption of supportive therapies like physical and occupational interventions, on account of their numerous advantages, including enhanced muscle strength, improved coordination, and elevated quality of life for individuals suffering from the illness, is further propelling the market growth. Apart from this, the rising usage of laser therapies, particularly fractional carbon dioxide lasers, since they work by targeting the calcified collagen bundles and promoting the regeneration of healthier skin layers in patients, is acting as another significant growth-inducing factor. Additionally, the emerging popularity of innovative approaches, including gene therapy, which involves the introduction of functional genetic material into affected cells to rectify or substitute the faulty genes responsible for the syndrome, is expected to drive the pseudoxanthoma elasticum market during the forecast period.

IMARC Group's new report provides an exhaustive analysis of the pseudoxanthoma elasticum market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for pseudoxanthoma elasticum and represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the pseudoxanthoma elasticum market in any manner.

Recent Developments:

• In September 2023, Inozyme Pharma, Inc. announced positive interim safety, pharmacokinetic (PK), pharmacodynamic (PD), and exploratory efficacy data from the company’s ongoing phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE). INZ-701 exhibited a favorable immunogenicity profile with low titers of non-neutralizing anti-drug antibodies. The study also revealed a rapid, significant, and sustained increase in PPi levels among patients.
• In April 2023, UMC Utrecht began a randomized, double-blind, placebo-controlled, phase 3 clinical trial (NCT05832580) titled “The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum (TEMP-PREVENT)” to assess the effect of etidronate on ectopic calcification in relatively young patients suffering from PXE.

Key Highlights:

• 80% of clinical cases of pseudoxanthoma elasticum have detectable mutations in the ABCC6 gene on the short arm of chromosome 16. This disease is usually inherited in an autosomal recessive pattern.
• The average age of disease onset is 13 years, but it varies between infancy and the seventh decade of life or older, with a peak number of new cases noted between ages 10 and 15.
• The prevalence of PXE is 0.83 per 100,000 persons and is higher in females than males in all age groups.
• The disease occurs in all ethnicities, but Afrikaners are more likely to have PXE as a result of a founder effect.

Drugs:

DS-1211b is a potential first-in-class small molecule developed through a research collaboration between Daiichi Sankyo and Sanford Burnham Prebys Medical Discovery Institute (SBP). This drug molecule is under development for the prevention of vascular calcification (pseudoxanthoma elasticum). It works by inhibiting tissue-nonspecific alkaline phosphatase (TNAP) that may prevent ectopic soft tissue calcification by increasing endogenous pyrophosphate (PPi).

INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. INZ-701 metabolizes ATP to produce PPi, a natural inhibitor of mineralization, and AMP, which can be broken down into phosphate and adenosine, the latter being a natural inhibitor of intimal proliferation. In preclinical studies, the experimental therapy has shown a potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in various devastating disorders, like ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis.

Time Period of the Study

• Base Year: 2023
• Historical Period: 2018-2023
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the pseudoxanthoma elasticum market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the pseudoxanthoma elasticum market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current pseudoxanthoma elasticum marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the pseudoxanthoma elasticum market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
• What was the country-wise size of the pseudoxanthoma elasticum market across the seven major markets in 2023 and what will it look like in 2034?
• What is the growth rate of the pseudoxanthoma elasticum market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of pseudoxanthoma elasticum across the seven major markets?
• What is the number of prevalent cases (2018-2034) of pseudoxanthoma elasticum by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of pseudoxanthoma elasticum by gender across the seven major markets?
• How many patients are diagnosed (2018-2034) with pseudoxanthoma elasticum across the seven major markets?
• What is the size of the pseudoxanthoma elasticum patient pool (2018-2023) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of pseudoxanthoma elasticum?
• What will be the growth rate of patients across the seven major markets?
   

Pseudoxanthoma Elasticum: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for pseudoxanthoma elasticum drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the pseudoxanthoma elasticum market?
• What are the key regulatory events related to the pseudoxanthoma elasticum market?
• What is the structure of clinical trial landscape by status related to the pseudoxanthoma elasticum market?
• What is the structure of clinical trial landscape by phase related to the pseudoxanthoma elasticum market?
• What is the structure of clinical trial landscape by route of administration related to the pseudoxanthoma elasticum market?