UK Gaucher Disease Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2581

  • Format: PPT, PDF

UK Gaucher Disease Therapeutics Market: Segmented by Diagnosis, Therapy, Routes of Administration, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

SKU: 2581 Categories: ,

Report Overview

The UK Gaucher Disease 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.

Gaucher disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase results in the accumulation of harmful quantities of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body especially within the bone marrow, spleen, and liver. It is the most common lysosomal storage disorder (LSD). Gaucher disease is caused by mutations of a single gene called GBA, which leads to very low levels of glucocerebrosidase enzyme leading to low degradation of glucocerebroside.

The symptoms and physical findings associated with Gaucher disease vary greatly from patient to patient. Some individuals are asymptomatic; develop few or no symptoms while others may have serious complications include skeletal abnormalities, blood disorders, and abdominal complaints. Three separate forms of Gaucher disease have been identified and are distinguished by the absence of, or the presence and extent of, neurological complications. All three forms of Gaucher disease are inherited in an autosomal recessive pattern.

Among these, Gaucher disease type 1 is the most common type. Over 90% of people affected have type 1 Gaucher disease. The overall frequency of all types of Gaucher disease is approximately 1 in 50,000 to 1 in 100,000 live births. Clinical experts estimate that there are between 350 to 400 patients with Gaucher disease in the UK; out of these 84% reside in England and 86% over 18 years of age. Each pregnant female has a 1 in 4 chance that the baby will be born with the disease. The age of onset for Gaucher disease type 2 is during early infancy. The age of onset of Gaucher disease type 3 varies, but the disorder generally begins during childhood or adolescence. The mean age at diagnosis is 24 years and at symptom onset is 11 years.

Market Growth Drivers

The Gaucher disease treatment market is likely to exhibit modest growth during the forecast period. Rising incidence of the disease and increasing efforts for the identification of more Gaucher disease patients are among the primary growth stimulants for the market.

An increase in the number of drug approvals for the management of disease and high investments by drug companies in research and development activities to develop novel therapies for its treatment are projected to shape the future of the Gaucher disease treatment market during the forecast period.

Market Restraints

Whereas, lack of awareness about Gaucher disease among people is expected to restrain the market growth during the forecast period. Additionally, the lack of drugs in the late-stage pipeline and the rising cost of treatment are limiting the Gaucher disease treatment market from realizing its utmost potential.


1. UK Gaucher Disease Therapeutics Market Overview………………………
A. Market Size
2. Market Growth Drivers & Restraints……………………………………………….
Market Growth Drivers
A. Rising Incidence and Prevalence of Gaucher Disease
B. Huge Investment in Research and Development for Novel Interventions
C. Increase in Number of Drug Approvals and Lucrative Emerging Markets
Market Restraints
A. Lack of Approved Drugs
B. High Cost of Treatment
3. Major Clinical Types of Gaucher Disease
A. Type 1 (Non-Neuronopathic)
B. Type 2 (Acute Neuronopathic)
C. Type 3 (Chronic Neuronopathic)
4. UK Gaucher Disease Therapeutics Market Segmentation…………………..
A. By Diagnosis
I. Physical Examination
II. Blood Tests
III. Imaging Tests
IV. Preconception Screening
V. Prenatal Testing
VI. Others
B. By Therapy
I. Enzyme Replacement Therapy (ERP)
II. Substrate Reduction Therapy (SRT)
III. Osteoporosis Drugs
IV. Glucosylceramide Synthase Inhibitors
V. Surgery
VI. Others
C. By Routes of Administration
I. Oral
II. Intravenous Infusions
III. Others
D. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. UK Gaucher Disease Major Products 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. Genzyme Company overview, Product & Services, Strategies & Financials
B. Shire Plc. Company overview, Product & Services, Strategies & Financials
C. Actelion 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 Gaucher Disease Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

On the basis of diagnosis, the Gaucher disease market is segmented into

  • Physical examination
  • Blood tests
  • Imaging tests
  • Preconception screening
  • Prenatal testing
  • Others

Testing for Gaucher disease involves a standard blood test available at a hospital. Physicians rely primarily on the BGL test for enzyme activity, which will almost certainly show if the patient has Gaucher disease. All patients with Gaucher disease have low glucocerebrosidase enzyme activity, which is why their bodies cannot break down glucocerebroside (a fatty chemical). Rarely, BGL test results can be inconclusive, in which case genetic testing can usually help clarify whether you have Gaucher disease. The activity of the acid beta-glucosidase enzyme can be measured in peripheral blood leukocytes or cultured skin fibroblasts to confirm the diagnosis.

By Therapy:

Based on the therapy type, the Gaucher disease therapeutics market can be segmented as

  • Enzyme replacement therapy (ERT – imiglucerase or velaglucerase)
  • Substrate reduction therapy (SRT)
  • Osteoporosis drugs
  • Glucosylceramide synthase inhibitors
  • Surgery
  • Others

The surgery segment further segmented into spleen removal, bone-marrow transplant, spleen removal, and others. ERT is commonly used for type 1 and type 3 Gaucher disease to address issues unrelated to CNS as replacement enzymes cannot penetrate the blood-brain barrier. However, the treatment landscape is witnessing a gradual shift towards substrate replacement therapy (SRT) which includes blood products, bisphosphonates, and analgesics. Thus, among the various therapy types, it is estimated that enzyme replacement therapy coupled with recent advancement leading the market segment during the forecast period.

By Routes of Administration:

As per the routes of administration, the Gaucher disease therapeutics market is segmented into oral, intravenous infusion, and others. Out of these segments, the intravenous infusion segment is anticipated to hold the largest market share during the forecast period.

By Distribution Channel:

In terms of distribution channel, the Gaucher Disease therapeutics market can be divided into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

The hospital pharmacies segment generated the highest revenue in 2020 and is anticipated to witness growth during the forecast period due to most of the major products are prescription products, which are available in hospital pharmacies.

Top Major Players

The Gaucher disease treatment market is highly competitive, and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major players operating in the market include Genzyme Corporation, Shire Plc, Actelion Pharmaceuticals Ltd, Pfizer, Johnson & Johnson. Primarily among the approved drugs, Cerdelga (eliglustat) and Vpriv (velaglucerase alfa) administered by intravenous infusion while Zavesca (miglustat) given by oral route for the treatment of Gaucher disease.

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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The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

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