United Kingdom Gaucher Disease Therapeutics Market Size, Competitive Landscape, Regional Outlook and Driving Factors Analysis 2028

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.

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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.

The report delivers the challenges in front of the United Kingdom Gaucher Disease Therapeutics Market 2023 that allows a better understanding of the market providing a helpful comprehensive evaluation and clear-cut dynamics associated with this market. The analyst has studied the current market scenario while concentrating on the new business objectives with the lucrative opportunities that are available. This report includes changes in customer requirements, customer preferences, and the vendor landscape in the industry with key factors that are expected to influence the overall dynamics of the United Kingdom Gaucher Disease Therapeuticss Market including the current trends, growth opportunities, restraints, and market drivers.

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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.

The United Kingdom Gaucher Disease Therapeutics Market report is an easy-to-understand document giving section-wise details about the global market. Starting with the brief outline of the overall market, it will put forth the all-inclusive evaluated market statistics and different parameters for the forecast period. The report covers numerous aspects of the United Kingdom Gaucher Disease Therapeutics market divided into product kind, application, and end-use, offering the report to assist the customers in improving their ability to make precise decisions related to the business under the United Kingdom Gaucher Disease Therapeutics Market.

International Market Players will use the correct market facts and figures and applied math studies provided within the report back to perceive this and future growth of the worldwide Electrophoretic United Kingdom Gaucher Disease Therapeutics market. Each section of the research study is specially prepared to explore key aspects of the global United Kingdom Gaucher Disease Therapeutics Market. Buyers of the report will have access to accurate PESTLE, SWOT, and other types of analysis on the global United Kingdom Gaucher Disease Therapeutics market. Moreover, it offers highly accurate estimations on the CAGR, market share, and market size of key regions and countries.

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Strategic Points Covered in Table of Content of Global United Kingdom Gaucher Disease Therapeutics Market:

Chapter 1: Introduction, market driving force product Objective of Study and Research Scope the United Kingdom Gaucher Disease Therapeutics market
Chapter 2: Exclusive Summary - the basic information of the United Kingdom Gaucher Disease Therapeutics Market.
Chapter 3: Displaying the Market Dynamics- Drivers, Trends and Challenges & Opportunities of the United Kingdom Gaucher Disease Therapeutics
Chapter 4: Presenting the United Kingdom Gaucher Disease 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 Gaucher Disease 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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