알파-만노시도시스 : 시장 인사이트, 역학, 시장 예측(2034년)

Key Highlights:

• The alpha-mannosidosis is inherited in an autosomal recessive manner, making consanguinity a primary risk factor, as it increases the likelihood of inheriting two copies of the defective MAN2B1 gene. A family history of the disorder and being part of a genetically isolated or high-risk population also elevate the risk significantly.
• The current treatment landscape for alpha-mannosidosis includes LAMZEDE, the first approved enzyme replacement therapy for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. Its introduction may stimulate market growth by raising disease awareness and encouraging early diagnosis. However, limited alternatives and unmet neurological needs highlight ongoing opportunities for innovation and broader therapeutic development in this rare disorder.
• Despite the approval of LAMZEDE, the therapeutic pipeline for alpha-mannosidosis remains sparse, with limited innovation and no curative treatments in development. There is a critical need for disease-modifying therapies, especially those targeting central nervous system involvement, and for expanding global accessibility to improve patient outcomes.
• Alpha-mannosidosis remains vastly underdiagnosed due to low disease awareness and limited epidemiological data. There is a pressing need for better diagnostic tools, CNS-targeting therapies, and broader access to treatment to improve early detection and patient outcomes globally.

DelveInsight's comprehensive report titled "Alpha-mannosidosis - Market Insights, Epidemiology, and Market Forecast - 2034" offers a detailed analysis of alpha-mannosidosis. The report presents historical and projected epidemiological data covering Prevalent Cases of Alpha-mannosidosis, Diagnosed Prevalent Cases of Alpha-mannosidosis and Treated Cases of Alpha-mannosidosis. In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements in alpha-mannosidosis. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Alpha-mannosidosis Overview

Alpha-mannosidosis is an autosomal recessive lysosomal storage disorder classified as an inborn error of metabolism. It results from pathogenic variants in the MAN2B1 gene, which encodes the lysosomal alpha-mannosidase enzyme responsible for the degradation of glycoproteins. Deficiency or dysfunction of this enzyme leads to the intracellular accumulation of mannose-rich oligosaccharides, causing progressive cellular toxicity. Clinically, alpha-mannosidosis is characterized by a slowly progressive phenotype that may include mild to moderate intellectual disability, sensorineural hearing loss, cerebellar ataxia, skeletal deformities, immunodeficiency, and coarse facial features. The severity and onset of symptoms can vary among affected individuals, but neurological decline and multisystem involvement are hallmark features of the disease trajectory.

Alpha-mannosidosis Diagnosis and Treatment Overview

Diagnosis of alpha-mannosidosis is based on clinical presentation alongside confirmatory laboratory tests. Biochemical analysis typically reveals reduced or absent activity of the lysosomal alpha-mannosidase enzyme in leukocytes or fibroblasts. Molecular genetic testing of the MAN2B1 gene confirms the diagnosis and enables carrier detection and prenatal screening. MRI imaging may show cerebellar atrophy, and urinary oligosaccharide analysis can reveal elevated mannose-rich compounds.

Treatment is primarily supportive and multidisciplinary, addressing neurological, audiological, and orthopedic complications. Hematopoietic stem cell transplantation (HSCT) may slow disease progression if performed early, particularly in younger patients. Recently, enzyme replacement therapy (ERT) has emerged as a promising treatment strategy, with investigational therapies showing potential to restore deficient enzymatic function. Regular monitoring and individualized care are essential to manage evolving symptoms and improve quality of life.

Alpha-mannosidosis Epidemiology

The epidemiology section of the alpha-mannosidosis market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the prevalence of alpha-mannosidosis. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

• According to Genetic and Rare Disease Information Center, the estimated prevalence of alpha-mannosidosis suggest that fewer than 5,000 individuals in the US are suffering from this disease, indicating its classification as an ultra-rare genetic disorder.
• According to secondary analysis, the prevalence of alpha-mannosidosis is in the range of 1 to 3 in 1,000,000 in US.
• Based on secondary data analysis, the estimated prevalence of alpha-mannosidosis in Italy ranges from approximately 1 in 250,000 to 1 in 1,000,000 live births, underscoring its classification as an ultra-rare lysosomal storage disorder within the national population.
• Based on secondary data analysis, alpha-mannosidosis has an estimated prevalence rate of approximately 1 per 500,000 live births, reflecting its classification as an exceptionally rare lysosomal storage disorder.

Alpha-mannosidosis Market Outlook

The alpha-mannosidosis therapeutics market is further expected to increase by the major drivers, such as the rising prevalent population, technological advancements, and upcoming therapies in the forecast period [2025-2034].

The treatment landscape for Alpha-mannosidosis remains limited, with LAMZEDE (velmanase alfa-tycv) standing as the sole approved therapy. LAMZEDE, a recombinant ERT, addresses the non-neurological manifestations by supplementing deficient alpha-mannosidase activity, thereby reducing glycoprotein accumulation. While it represents a meaningful advancement, its intravenous administration and limited efficacy on central nervous system symptoms highlight therapeutic gaps. No curative treatments exist, and supportive care remains essential. Ongoing research aims to explore novel approaches such as gene therapy and CNS-targeting strategies. The current landscape underscores an urgent need for more accessible, comprehensive therapies that can modify disease progression and improve long-term outcomes.

With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the alpha-mannosidosis market in the 7MM is expected to change significantly during the study period 2020-2034.

Alpha-mannosidosis Drug Chapters

Marketed Drugs

LAMZEDE (velmanase alfa-tycv): Chiesi Global Rare Diseases

LAMZEDE is a recombinant form of human alpha-mannosidase, designed to supplement the deficient enzyme and reduce the accumulation of mannose-rich oligosaccharides in affected tissues. Administered intravenously once a week, LAMZEDE has shown efficacy in improving biochemical markers and certain clinical symptoms, such as motor function and pulmonary function. However, its inability to cross the blood-brain barrier limits its effectiveness in treating neurological symptoms.

• In February 2023, Chiesi Global Rare Diseases announced that the US FDA had granted approval for LAMZEDE (velmanase alfa-tycv) to treat non-central nervous system manifestations of alpha-mannosidosis in both adult and pediatric patients.
• In April 2018, Chiesi announced that the European Commission had granted marketing authorization for LAMZEDE (velmanase alfa) for the treatment of non-neurological manifestations in individuals with mild to moderate alpha-mannosidosis.
• In February 2006, LAMZEDE received Orphan Drug Designation (ODD) for the treatment of Alpha-mannosidosis.

Alpha-mannosidosis Market Segmentation

DelveInsight's 'Alpha-mannosidosis - Market Insights, Epidemiology, and Market Forecast - 2034' report provides a detailed outlook of the current and future alpha-mannosidosis market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Alpha-mannosidosis Market Size by Countries

The Alpha-mannosidosis market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2024, the United States held a significant share of the overall 7MM (Seven Major Markets) alpha-mannosidosis market, primarily attributed to the country's higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.

Alpha-mannosidosis Drugs Uptake

This section focuses on the sales uptake of potential alpha-mannosidosis drugs that have recently been launched or are anticipated to be launched in the alpha-mannosidosis market between 2020 and 2034. It estimates the market penetration of alpha-mannosidosis drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the alpha-mannosidosis market.

The emerging alpha-mannosidosis therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the alpha-mannosidosis market.

Alpha-mannosidosis Market Access and Reimbursement

DelveInsight's 'Alpha-mannosidosis - Market Insights, Epidemiology, and Market Forecast - 2034' report provides a descriptive overview of the market access and reimbursement scenario of alpha-mannosidosis.

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current alpha-mannosidosis market trends and fill gaps in secondary findings, we interview KOLs and SMEs' working in the alpha-mannosidosis domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or alpha-mannosidosis market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the alpha-mannosidosis unmet needs.

Alpha-mannosidosis: KOL Insights

DelveInsight's analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as, Femme Mere Enfant Hospital, Lyon, France; University Medical Center Mainz, Germany; CHU La Reunion Site GHSR, Saint Pierre, France; Rare Disease Research Partners, Amersham, UK, among others.

"Alpha-mannosidosis is caused by genetic mutations, also known as pathogenic variants. Genetic mutations can be hereditary, when parents pass them down to their children, or they may occur randomly when cells are dividing."

"Diagnosis of alpha-mannosidosis in a proband is confirmed by demonstrating reduced activity (typically 5-10% of normal levels) of the lysosomal enzyme alpha-mannosidase in leukocytes or other nucleated cells. Alternatively, or in conjunction, the presence of biallelic pathogenic variants in the MAN2B1 gene, identified through molecular genetic testing, also establishes the diagnosis."

"The marketing authorization of LAMZEDE in the EU is a significant stepping stone for patients suffering from this devastating disease and reinforces the Chiesi Group's role in the complex and challenging world of rare diseases."

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the alpha-mannosidosis. Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Alpha-mannosidosis Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for alpha-mannosidosis. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging alpha-mannosidosis therapies.

Alpha-mannosidosis Report Insights

• Alpha-mannosidosis Patient Population
• Therapeutic Approaches
• Alpha-mannosidosis Pipeline Analysis
• Alpha-mannosidosis Market Size and Trends
• Alpha-mannosidosis Market Opportunities
• Impact of Upcoming Therapies

Alpha-mannosidosis Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• Alpha-mannosidosis Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Alpha-mannosidosis Market
• Alpha-mannosidosis Drugs Uptake

Alpha-mannosidosis Report Assessment

• Alpha-mannosidosis Current Treatment Practices
• Unmet Needs
• Alpha-mannosidosis Product Profiles
• Alpha-mannosidosis Market Attractiveness

Key Questions:

• How common is alpha-mannosidosis?
• What are the key findings of alpha-mannosidosis epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020-2034)?
• What are the currently available treatments for alpha-mannosidosis?
• What are the disease risk, burden, and unmet needs of alpha-mannosidosis?
• At what CAGR is the alpha-mannosidosis market and its epidemiology is expected to grow in the 7MM during the forecast period (2025-2034)?
• How would the unmet needs impact the alpha-mannosidosis market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of alpha-mannosidosis in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2025-2034)?
• How many companies are currently developing therapies for the treatment of alpha-mannosidosis?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the alpha-mannosidosis market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of current treatment in the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the attribute analysis section to provide visibility around leading classes.
• Highlights of Market Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Frequently Asked Questions:

1. What are the treatment goals for alpha-mannosidosis?

The primary treatment goals for alpha-mannosidosis are to reduce the accumulation of mannose-rich oligosaccharides, alleviate systemic symptoms, and slow disease progression. Therapeutic strategies aim to improve physical function, support immune response, stabilize neurological outcomes, and enhance quality of life. Early intervention is crucial to preserve cognitive and motor abilities and minimize irreversible organ damage.

2. What are the challenges in managing alpha-mannosidosis?

Managing alpha-mannosidosis is challenging due to its progressive, multisystem nature and rarity. Limited treatment options-such as LAMZEDE, which does not address neurological symptoms-pose a major hurdle. Delayed diagnosis, variability in clinical presentation, and lack of widespread awareness further complicate care. High treatment costs and access barriers, especially in low-resource settings, add to the complexity of disease management.

3. What are the key factors driving the growth of the alpha-mannosidosis market?

Key factors driving growth in the alpha-mannosidosis market include increased disease awareness, improved diagnostic capabilities, and the approval of LAMZEDE-the first enzyme replacement therapy. Advancements in rare disease research, expanded newborn screening programs, and supportive regulatory frameworks further boost interest. Additionally, rising investments in novel treatments, including gene and CNS-targeted therapies, contribute to future market expansion.

4. How will the alpha-mannosidosis Market and Epidemiology Forecast Report benefit the clients?

The Alpha-mannosidosis Market and Epidemiology Forecast Report offers clients strategic insights into disease prevalence, patient segmentation, and market dynamics. It helps identify commercial opportunities, assess treatment gaps, and support planning for product development, market entry, or expansion. The report also aids stakeholders in understanding regulatory landscapes, forecasting trends, and making data-driven investment or policy decisions in rare diseases.

Table of Contents

1. Key Insights

2. Report Introduction

3. Market Overview at a Glance

• 3.1. Market Share (%) Distribution of Alpha-mannosidosis in 2020
• 3.2. Market Share (%) Distribution of Alpha-mannosidosis in 2034

4. Epidemiology and Market Methodology

5. Executive Summary

6. Key Events

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Types
• 7.3. Causes
• 7.4. Pathophysiology
• 7.5. Symptoms
• 7.6. Risk Factor
• 7.7. Diagnosis
  • 7.7.1. Diagnostic Algorithm
  • 7.7.2. Diagnostic Guidelines
• 7.8. Treatment and Management
  • 7.8.1. Treatment Algorithm
  • 7.8.2. Treatment Guidelines

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale:7MM
• 8.3. Prevalent Cases of Alpha-mannosidosis in the 7MM
• 8.4. Diagnosed Prevalent Cases of Alpha-mannosidosis in the 7MM
• 8.5. The United States
  • 8.5.1. Prevalent Cases of Alpha-mannosidosis
  • 8.5.2. Diagnosed Prevalent Cases of Alpha-mannosidosis
  • 8.5.3. Treated Cases of Alpha-mannosidosis
• 8.6. EU4 and the UK
  • 8.6.1. Prevalent Cases of Alpha-mannosidosis
  • 8.6.2. Diagnosed Prevalent Cases of Alpha-mannosidosis
  • 8.6.3. Treated Cases of Alpha-mannosidosis
• 8.7. Japan
  • 8.7.1. Prevalent Cases of Alpha-mannosidosis
  • 8.7.2. Diagnosed Prevalent Cases of Alpha-mannosidosis
  • 8.7.3. Treated Cases of Alpha-mannosidosis

9. Patient Journey

10. Marketed Therapies

• 10.1. Key Cross Competition
• 10.2. LAMZEDE (velmanase alfa-tycv): Chiesi Global Rare Diseases
  • 10.2.1. Product Description
  • 10.2.2. Other Development Activities
  • 10.2.3. Clinical Trials Information
  • 10.2.4. Safety and Efficacy

11. Alpha-mannosidosis: Seven Major Market Analysis

• 11.1. Key Findings
• 11.2. Market Outlook
• 11.3. Attribute Analysis
• 11.4. Key Market Forecast Assumptions
  • 11.4.1. Cost Assumptions and Rebates
  • 11.4.2. Pricing Trends
  • 11.4.3. Analogue Assessment
  • 11.4.4. Launch Year and Therapy Uptake
• 11.5. Total Market Size of Alpha-mannosidosis in the 7MM
• 11.6. Market Size of Alpha-mannosidosis by Therapies in the 7MM
• 11.7. The United States Market Size
  • 11.7.1. Total Market Size of Alpha-mannosidosis
  • 11.7.2. Market Size of Alpha-mannosidosis by Therapies
• 11.8. EU4 and the UK Market Size
  • 11.8.1. Total Market Size of Alpha-mannosidosis
  • 11.8.2. Market Size of Alpha-mannosidosis by Therapies
• 11.9. Japan Market Size
  • 11.9.1. Total Market Size of Alpha-mannosidosis
  • 11.9.2. Market Size of Alpha-mannosidosis by Therapies

12. Key Opinion Leaders' Views

13. Unmet Needs

14. SWOT Analysis

15. Market Access and Reimbursement

16. Appendix

• 16.1. Bibliography
• 16.2. Abbreviations and Acronyms
• 16.3. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

19. About DelveInsight