United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market Expected to Grow Faster According to New Research Report 2023-2028

Report Overview

The UK Sanfilippo Syndrome Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Sanfilippo Syndrome (MPS III) is an ultra-rare autosomal recessive inherited disorder and a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses (MPS) which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). MPS III is a type of childhood dementia that causes fatal brain damage. Sanfilippo syndrome is caused by a lack of an enzyme that normally breaks down and recycles a large, complex sugar molecule called heparan sulfate. This heparan sulfate accumulates and causes damage to the cells of the central nervous system, including the brain.

Children with MPS III usually appear healthy at birth, but the developmental delay is usually evident by age 2-5 years. Mental and motor development peak by 3-6 years of age, after which intellectual decline usually occurs. Behavioral problems such as hyperactivity and irritability may become obvious earlier. The severe behavioral disturbance is a very common feature of Sanfilippo syndrome and one of the more difficult aspects of the disorder to manage.

Sanfilippo syndrome causes progressive intellectual disability and the loss of previously acquired skills (developmental regression). In later stages of the disorder, children with Sanfilippo Syndrome may develop seizures and movement disorders. The typical life expectancy for affected children is between 10-20 years old. MPS III affects males and females equally. It is estimated that nearly 6% of the UK population (around 3.5 million people) will be affected by a rare disease at some point in their lives. There are four subtypes of MPS III: types A, B, C, and D. Each type is caused by a mutation in a different gene. MPS IIIA is the most common subtype affecting around 1 in 100,000 births, closely followed by type B at 1 in 200,00.

Market Growth Drivers

The growing necessity for advanced therapeutics will impel the market players to launch novel therapies, which in turn, will boost the Sanfilippo syndrome therapeutics market growth in the forthcoming years. Moreover, the increasing acquisitions and deals between key players will also bolster the healthy growth of the market.

Additionally, the growing unmet clinical needs of patients along with better treatment outcomes, financial incentives for orphan drug development to recover R&D costs, success for the drugs in pipeline with faster market access, high burden of rare diseases, favorable regulations for orphan products development will spur demand for Sanfilippo syndrome therapeutics market during the forecast period.

Market Restraints

While huge treatment costs and lack of awareness are more likely to hamper the Sanfilippo syndrome therapeutics market growth.

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Top Major Players

The UK Sanfilippo syndrome therapeutics market provides a great opportunity for market players to enter and now more focused on the adoption of inorganic growth strategies to garner maximum market share and limit the high price tag of the therapeutics. The prominent players in the MPS III treatment market include Shire Human Genetic Therapies Inc, Alexion Pharmaceuticals Inc, Sobi Pharma, Allievex Corporation, and Abeona Therapeutics Inc. among others.

The United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market 2023 Report makes available the current and forthcoming technical and financial details of the industry. It is one of the most comprehensive and important additions to the Prudent Markets archive of market research studies. It offers detailed research and analysis of key aspects of the global United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics market. This report explores all the key factors affecting the growth of the global United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics market, including demand-supply scenario, pricing structure, profit margins, production, and value chain analysis.

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Market Segmentation

By Diagnosis:

Based on the diagnosis, the MPS III therapeutics market can be bifurcated into

Urine tests
Blood tests
Genetic testing
Preimplantation diagnosis
Others

To diagnose MPS III, mucopolysaccharides are usually first measured in urine, followed by measurement of enzyme activity in blood or a small skin sample. Increased heparan sulfate in urine and a decrease in the activity of any one of the four enzymes and will identify the MPS III type (A, B, C, or D). Genetic testing of a blood sample will allow the identification of the exact changes in the DNA. It can also be used for prenatal testing of future pregnancies (testing a fetus while still in the womb) and/or preimplantation diagnosis (testing of embryos created through IVF to select those that do not carry the relevant gene mutation).

By Therapeutics:

On the basis of therapeutics, the MPS III therapeutics market can be categorized into

Enzyme Replacement Therapies (ERT)
Hematopoietic Stem Cell Transplantation (HSCT)
Stem Cell Therapy (SCT)
Chaperone Advanced Replacement Therapy (CART)
Gene Therapy
Other medications

At present, clinical trials for Enzyme replacement therapy (ERT) are underway for Sanfilippo syndrome treatment. Gene Therapy although highly complex, is the most viable option for rectifying the genes. Another challenge the therapy poses is the insertion of the normal gene into hundreds of millions of cells that contain the defective gene; which requires a carrier to transport the gene inside the cells.

By Route of Administration:

Based on route of administration, the MPS III therapeutics market can be classified into o

Oral
Intravenous (IV)
Intracerebroventricular (ICV)
Intrathecal
Others

Various approved drugs are given by various different routes of administration. Like, therapeutics of Shire, Allievex Corporation, Sobi, and Alexion Pharma administered by intrathecal, intracerebroventricular, and intravenous routes of administration.

By Distribution Channels:

In terms of distribution channels, the MPS III therapeutics market can be categorized into

Hospital pharmacies
Retail pharmacies
Online pharmacies

The hospital pharmacies segment has accounted for the highest market share in 2020 as the major therapeutics are prescription products. Therefore, hospital pharmacies are further anticipated also to witness growth during the forecast period.

By End Users:

Based on end-user, the MPS III therapeutics market can be segmented into
Hospitals
Specialty Clinics
Medical Research Centres
Home-infusion
Others

The key factor aiding in the dominance of the hospital segment is the appropriate and safe administration of critical therapeutics used in MPS III treatment by trained medical professionals who can administer only in hospitals with care and proper guidelines. Thus, the potential growth of the hospital segment can be anticipated during the forecast period.

Top Major Players

The UK Sanfilippo syndrome therapeutics market provides a great opportunity for market players to enter and now more focused on the adoption of inorganic growth strategies to garner maximum market share and limit the high price tag of the therapeutics. The prominent players in the MPS III treatment market include Shire Human Genetic Therapies Inc, Alexion Pharmaceuticals Inc, Sobi Pharma, Allievex Corporation, and Abeona Therapeutics Inc. among others.

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The report covers the competitive analysis of the market. As the demand is driven by a buyer's paying capacity and the rate of item development, the report shows the important regions that will direct growth. This section exclusively shares insight into the budget reports of big-league members of the market helping key players and new entrants understand the potential of investments in the Global United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market. It can be better employed by both traditional and new players in the industry for complete know-how of the market.

Strategic Points Covered in Table of Content of Global United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics
Chapter 4: Presenting the United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics Market Factor Analysis, Porters Five Forces, Supply/Value Chain, PESTEL analysis, Market Entropy, Patent/Trademark Analysis.
Chapter 5: Displaying the by Type, End User and Region/Country
Chapter 6: Evaluating the leading manufacturers of the United Kingdom Sanfilippo Syndrome (MPS III) Therapeutics market which consists of its Competitive Landscape, Peer Group Analysis, BCG Matrix & Company Profile
Chapter 7: To evaluate the market by segments, by countries and by Manufacturers/Company with revenue share and sales by key countries in these various regions
Chapter 8 & 9: Displaying the Appendix, Methodology and Data Source

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