DelveInsight’s “Mucopolysaccharidosis Type III Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Mucopolysaccharidosis Type III market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Mucopolysaccharidosis Type III drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Mucopolysaccharidosis Type III treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Mucopolysaccharidosis Type III: An Overview
Mucopolysaccharidosis type III also known as Sanfilippo syndrome is a rare autosomal recessive lysosomal storage disease that primarily affects the brain and spinal cord. It belongs to a group of disorders called mucopolysaccharidoses, which are characterized by the accumulation of complex sugar molecules called glycosaminoglycans (GAGs) in various tissues and organs of the body.
GAGs are stored in the cell lysosome and are degraded by enzymes such as glycosidases, sulfatases, and acetyltransferases. Deficiency in these enzymes leads to MPS III. Sanfilippo syndrome is divided into four subtypes, which are differentiated by their genetic cause: MPS IIIA, IIIB, IIIC, and IIID. The severity of symptoms can vary between the subtypes. The disease is inherited in an autosomal recessive manner.
MPS III manifests in young children. It is progressive, and symptoms typically become more severe as the child gets older. The life expectancy of individuals with Sanfilippo syndrome is significantly reduced, and many affected children do not live past their teenage years.
Currently, there is no cure for Sanfilippo syndrome. Treatment primarily focuses on managing symptoms and providing supportive care. Therapies such as speech and occupational therapy, behavioral interventions, and medications to manage certain symptoms may be helpful. Various gene therapies and enzyme replacement therapies are currently under development for Sanfilippo syndrome.
Mucopolysaccharidosis Type III Market Key Facts
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The findings from the National Organization for Rare Disorders also quoted that MPS IIIA is the most common subtype affecting around 1 in 100,000 births, closely followed by type B at 1 in 200,000.
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According to Celik et al. (2021), in France, the prevalence of MPS III was found to be 0.73 per 100,000 live births.
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According to the National MPS Society, MPS III is the most common form of mucopolysaccharidosis, and 1 in 70,000 newborns are born with the disease. MPS III A and MPS III B are more common than MPS III C and MPS III D.
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As per research conducted by Khan et al. (2017), in Japan, the birth prevalence was found to be 1 out of 385,000 for all MPS III types (1 out of 1,000,000 for MPS IIIA, 1 out of 834,000 for MPS IIIB, 1 out of 2.5 million for MPS IIIC).
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According to a retrospective study by Puckett et al. (2021), in the United States using the National MPS Society database records, the prevalence of MPS III (Sanfilippo syndrome) was found to be 0.71 per 1,000,000. Subtypes MPS IIIA, MPS IIIB, MPS IIIC, and MPS IIID had prevalence of 0.52, 0.14, 0.04, and 0 per 1,000,000.
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According to DelveInsight’s analysis, the risk of having Sanfilippo Syndrome is equal for males and females.
Mucopolysaccharidosis Type III Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Mucopolysaccharidosis Type III pipeline therapies. It also thoroughly assesses the Mucopolysaccharidosis Type III market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Mucopolysaccharidosis Type III drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Mucopolysaccharidosis Type III Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Mucopolysaccharidosis Type III epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Mucopolysaccharidosis Type III epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Mucopolysaccharidosis Type III Epidemiology, Segmented as –
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Total Prevalent Cases of Sanfilippo Syndrome in the 7MM [2019–2032]
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Total Diagnosed Prevalent Cases of Sanfilippo Syndrome in the 7MM [2019–2032]
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Subtype-specific Cases of Sanfilippo Syndrome on the Basis of Severity in the 7MM [2019–2032]
Mucopolysaccharidosis Type III Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Mucopolysaccharidosis Type III market or expected to be launched during the study period. The analysis covers the Mucopolysaccharidosis Type III market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Mucopolysaccharidosis Type III drugs based on their sale and market share.
The report also covers the Mucopolysaccharidosis Type III pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Mucopolysaccharidosis Type III companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Mucopolysaccharidosis Type III Therapeutics Analysis
Till date, there is no FDA-approved treatment or cure for Sanfilippo syndrome. However, various treatment approaches are being explored to manage the symptoms and slow the progression of the disease. Treatment remains largely supportive. Therapies such as speech and occupational therapy, behavioral interventions, and medications to manage certain symptoms may be helpful. Gene therapies, stem cell therapies, and enzyme replacement therapies are currently under development for Sanfilippo syndrome.
It is important for children with MPS III to be managed by a multidisciplinary team of specialists to give them the best quality of life. At different stages, this could include a combination of the following specialists: neurologist, developmental pediatrician, metabolic/genetics specialist, orthopedics, gastroenterologist, ophthalmologist, cardiologist, endocrinologist, allied health (e.g. physiotherapy, OT, behavioral therapists, speech therapist) and an ENT (ear, nose and throat) specialist.
Several major pharma and biotech companies are developing therapies for Mucopolysaccharidosis III. Currently, Lysogene is leading the therapeutics market with its Mucopolysaccharidosis III drug candidates in the most advanced stage of clinical development.
In September 2023, Medipal Holdings and JCR Pharmaceuticals entered into an agreement for JR-441 on MPS III treatment.
In July 2023, the PEI (Paul-Ehrlich Institute) accepted the clinical trial application (CTA) for JR-441 to start a global Phase I/II clinical trial for the treatment of mucopolysaccharidosis Type III A (Sanfilippo Syndrome).
Mucopolysaccharidosis Type III Companies Actively Working in the Therapeutics Market Include
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Lysogene
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Orchard therapeutics
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Abeona Therapeutics
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Amsterdam Molecular Therapeutics
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Swedish Orphan Biovitrum
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Alexion Pharmaceuticals
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Allievex Corporation
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Denali therapeutics
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Esteve
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REGENXBIO
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Seelos Therapeutics
And Many Others
Emerging and Marketed Mucopolysaccharidosis Type III Therapies Covered in the Report Include:
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LYS-SAF302: Lysogene
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Tralesinidase alfa: Allievex Corporation
And Many More
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Mucopolysaccharidosis Type III Competitive Intelligence Analysis
4. Mucopolysaccharidosis Type III Market Overview at a Glance
5. Mucopolysaccharidosis Type III Disease Background and Overview
6. Mucopolysaccharidosis Type III Patient Journey
7. Mucopolysaccharidosis Type III Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Mucopolysaccharidosis Type III Treatment Algorithm, Current Treatment, and Medical Practices
9. Mucopolysaccharidosis Type III Unmet Needs
10. Key Endpoints of Mucopolysaccharidosis Type III Treatment
11. Mucopolysaccharidosis Type III Marketed Therapies
12. Mucopolysaccharidosis Type III Emerging Drugs and Latest Therapeutic Advances
13. Mucopolysaccharidosis Type III Seven Major Market Analysis
14. Attribute Analysis
15. Mucopolysaccharidosis Type III Market Outlook (In US, EU5, and Japan)
16. Mucopolysaccharidosis Type III Companies Active in the Market
17. Mucopolysaccharidosis Type III Access and Reimbursement Overview
18. KOL Views on the Mucopolysaccharidosis Type III Market
19. Mucopolysaccharidosis Type III Market Drivers
20. Mucopolysaccharidosis Type III Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance.
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