UK Sanfillipo Syndrome (MPS-III) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2586

  • Format: PPT, PDF

UK Sanfilippo Syndrome (MPS III) Therapeutics Market: Segmented by Diagnosis, Therapeutics, Route of Administration, Distribution Channels, and End Users – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

SKU: 2586 Categories: ,

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.


1. UK Sanfilippo Syndrome Therapeutics Market Overview…………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Need of Advanced Therapeutics & Introduction of Novel Therapies
B. Financial Incentives and High Burden of Rare Disease
C. Rise in Prevalence and Incidence of Sanfilippo Syndrome
Market Restraints
A. High Treatment Costs
B. Lack of Awareness
3. Major Types of Sanfilippo Syndrome………………………………………………….
A. Sanfilippo Syndrome Type A (MPS IIIA)
B. Sanfilippo Syndrome Type B (MPS IIIB)
C. Sanfilippo Syndrome Type C (MPS IIIC)
D. Sanfilippo Syndrome Type D (MPS IIID)
4. Sanfilippo Syndrome Therapeutics Market Segmentation………………..
A. By Diagnosis
I. Urine Test
II. Blood Test
III. Genetic Testing
IV. Preimplantation Diagnosis
V. Others
B. By Therapeutics
I. Enzyme Replacement Therapy (ERP)
II. Haematopoietic Stem Cell Transplantation (HSCT)
III. Stem Cell Therapy (SCT)
IV. Chaperone Advanced Replacement Therapy (CART)
V. Gene Therapy
VI. Other Medications
C. By Route of Administration
I. Oral
II. Intravenous (IV)
III. Intracerebroventicular (ICV)
IV. Intrathecal (IT)
V. Others
D. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
E. By End Users
I. Hospitals
II. Speciality Clinics
III. Home-Infusion
IV. Medical Research Centres
V. Others
5. Sanfilippo Syndrome Major Drugs Market Share………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Shire Company overview, Product & Services, Strategies & Financials
B. Alexion Company overview, Product & Services, Strategies & Financials
C. Allievex Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of Sanfilippo Syndrome Therapeutics Market
B. Future Opportunities
10. Conclusion

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.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

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