UK Lysosomal Storage Disorders Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2579

  • Format: PPT, PDF

UK Lysosomal Storage Disorders Therapeutics Market: Segmented by Treatment Type, and End Users – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021


SKU: 2579 Categories: ,

Report Overview

The UK Lysosomal Storage Disorders 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.

LSDs usually is caused by a deficiency of a single lysosomal enzyme or protein, which results in a build-up of lipid or sugar in the lysosome. There are many lysosomal enzymes and they each work in sequence with each other to break down lipids and sugars into simpler forms for removal from the cell. A deficiency in the function of one enzyme means that the process of breaking down and recycling lipids and sugars stops, and they remain stored in the lysosomes of each cell. This affects normal cell function.

There are nearly 50 of these disorders altogether, and they may affect different parts of the body, including the skeleton, brain, skin, heart, and central nervous system. According to NHS Foundation Trust, the incidence is approximately 1 in 7,000 births.  Although affected children usually appear normal at birth, each disorder progressively develops symptoms, which can include bone and skeletal deformity, visual and hearing problems, enlarged head and tongue, coarse hair, developmental delays, and reduction over time of the mental function. One of the main objectives of the service is to increase the benefits of treatment for patients with LSDs with particular reference to the use of enzyme replacement therapy.

The types of lysosomal storage disorders that occur most often in babies and children include Gaucher Disease (Types I, II, and III), Batten Disease, Cystinosis, Fabry Disease, Pompe Disease, Aspartylglucosaminuria, Niemann Pick Disease, and Tay Sachs Disease.

Market Growth Drivers

An increasing diagnosis rate due to growing public awareness and financial incentives for orphan drug development to recover R&D costs is the primary factor fuelling the growth of the LSD therapeutics market. Also, an increasing focus of major biopharmaceutical companies on the research and development of drugs for the treatment of lysosomal storage disorders is expected to boost the LSD therapeutics market growth during the forecast period.

Moreover, the number of treatment options currently in the pipeline is further expected to bolster the revenue growth of the LSD therapeutics market over the forecast period. The advent of therapies targeting neuropathic manifestations by crossing the blood-brain barrier (BBB) and therapies that overcome immune response and have better tissue selectivity has also been anticipated to fuel the LSD therapeutics market growth in the upcoming years.

Market Restraints

However, the UK LSD therapeutics market also faces a few restraints that include heterogeneity of the disease leading to underdiagnoses of lysosomal storage disorders, lack of treatment options, expensive drugs, change in payer policies, lack of proper training and knowledge, nature of the disease, and concerns in diagnosis. The restraints can be overcome by the drugs that aid in improving the lifespan and quality of life for the patients.


1. UK Lysosomal Storage Disorders Therapeutics Market Overview……………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Increasing Diagnosis Rate
B. Growing Public Awareness
C. Financial Incentives for Orphan Drug Development
D. Increase in R&D Expenditure & Intensive Research on Novel Treatment
E. Rising Incidence and Prevalence of Major Lysosomal Storage Disorders
Market Restraints
A. Lack of Treatment Options
B. Expensive Drugs
C. Lack of Experienced Professionals
D. Change in Payer Policies
3. Major Types of Lysosomal Storage Disorders……………………………….
A. Gaucher Disease
B. Fabry Disease
C. Pompe Disease
D. Mucopolysaccharidosis
E. Batten Disease
F. Niemann Pick Disease
G. Tay Sachs Disease
H. Others
4. Lysosomal Storage Disorders Therapeutics Market Segmentation………………
A. By Treatment Type
I. Enzyme Replacement Therapy (ERT)
II. Stem Cell Therapy (SCT)
III. Substrate Reduction Therapy (SRT)
IV. Other Therapies
B. By End Users
I. Hospitals
II. Clinics
III. Diagnostics Centres
IV. Academic and Research Organisations
V. Others
5. Lysosomal Storage Disorders Major Drugs Market Share……………………
A. Market Analysis, Insights and Forecast – By Revenue
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Alexion Company overview, Product & Services, Strategies & Financials
B. BioMarin Company overview, Product & Services, Strategies & Financials
C. Synageva Company overview, Product & Services, Strategies & Financials
D. Amicus Company overview, Product & Services, Strategies & Financials
E. Genzyme Company overview, Product & Services, Strategies & Financials
F. Audentes 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 Lysosomal Storage Disorders Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Treatment Type:

Based on the treatment, the lysosomal storage disorders therapeutics market can be bifurcated into

  • Enzyme Replacement Therapy
  • Stem Cell Therapy
  • Substrate Reduction Therapy
  • Other Therapies

Enzyme replacement therapy is a treatment that replaces an enzyme that is absent or deficient in the body. Some of the available enzyme replacement therapies for lysosomal storage disorders are Sebelipase Alfa, Velaglucerase Alfa, Taliglucerase Alfa, Agalsidase beta, Laronidase, and Imiglucerase among others.

The enzyme replacement therapy segment holds a significant market share in the lysosomal storage disorders therapeutics market and is anticipated to show a similar trend over the forecast period due to improving organ function and effective in reducing toxic storage material in patients suffering from lysosomal disorders.

By End Users:

In terms of end-users, the lysosomal storage disorders therapeutics market can be categorized into

  • Hospitals
  • Clinics
  • Diagnostic Centres
  • Academic and Research Organisations
  • Others

In terms of value, the hospitals’ segment accounted for the leading market share in 2020, which is attributable to the fact that the effective diagnosis and medications for LSD treatment can be only administered in the hospitals under the surveillance of trained medical professionals. Hence, the hospitals’ segment has been further anticipated to enhance the market share over the forecast period.

Top Major Players

The UK lysosomal storage disorders therapeutics market is a highly competitive market and major key players are adopting strategies; organic as well as inorganic market strategies. Kanuma by Alexion Pharmaceuticals Inc. is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages for the treatment of LSD. The other prominent key players prevailing in the LSD therapeutics market are BioMarin Pharmaceuticals Inc., Synageva Biopharma Corporation, Amicus Therapeutics Inc., Genzyme Corporation, and Audentes Therapeutics Inc. among others.

Research Methodology

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  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

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