Gaucher Disease is the rare multi-systemic metabolic disorder occur due to the defective and insufficient activity of the enzyme glucocerebrosidase. It leads to the accumulation of substrate, glucocerebroside in tissue macrophages and damage to hematological, visceral and bone systems. Painless splenomegaly, anemia, thrombocytopenia, chronic fatigue, hepatomegaly, bone pain, pathologic fractures and others are the most frequent manifestations of Gaucher Disease.
Gaucher Disease is classified as Type 1 Gaucher Disease, Type 2 Gaucher Disease and Type 3 Gaucher Disease based on clinical subtypes. The United States accounts for the highest prevalence of 8,000 cases followed by EU5 (the United Kingdom, Germany, France,and Italy) and Japan. Type 1 Gaucher Disease is more prevalent in comparison to Type 2 Gaucher Disease and Type 3 Gaucher Disease.
Market size of Gaucher Disease in seven major marketsis expected to reach USD 1,456 Million in 2026. The United States accounts for the highest market share in comparison to EU5 (United Kingdom, Germany, France and Italy) and Japan owing to the highest prevalent cases, increasing research activities and improved healthcare resources. Among the European countries, Germany accounts for the highest market share followed by France.
Elelyso (taliglucerase alfa), Vpriv (velaglucerase alfa) and Cerezyme (imiglucerase) are the approved enzyme replacement therapy whereas miglustat (Zavesca) and eliglustat (Cerdelga) are the approved substrate reduction therapy for the treatment of Type 1 Gaucher Disease. Use of enzyme replacement therapy has resulted in improving anemia and low platelet counts, reducing enlargement of liver and spleen among the patients. Substrate reduction therapy provides the consistent dosage to patients in comparison to enzyme replacement therapy.
Major players such as Belrose Pharma, Sanofi-Genzyme, AVROBIO, Inc, Lixte Biotechnology Holdings, Inc., Orphazyme A.S and other companies are involved in developing therapies for the treatment of Gaucher Disease. The launch of emerging therapies such as Arimoclomol in 2024 and Venglustat in 2026 shall fuel the market growth. Rising collaborations and agreements between the companies and universities, improved diagnosis and increasing awareness regarding Gaucher Disease would have a positive impact on the market during the forecasted period.
Several factors such as less number of late-stage molecules, lack of awareness among the patients and physicians, high treatment cost, limited access to treatment and approval of generics of marketed therapies are hindering the market growth.
The forecasted market is expected to witness the slow growth due to the less number of late-stage therapies in clinical trials. A large number of therapies are present in pre-clinical and discovery stage of development, which would take several years for the approval in the market. Limited availability of treatment, inaccessibility of certain disease sites and emerging disease manifestations are contributing to the unmet needs of patients. There is immense need of developing novel therapies for the treatment of Type 2 Gaucher Disease and Type 3 Gaucher Disease.
The leading players in the market are Sanofi-Genzyme, Shire Pharmaceuticals, Actelion Pharmaceuticals (Acquired by Johnson & Johnson), and Pfizer. The other prominent players are Belrose Pharma and Orphazyme A.S.
Key questions answered
- Prevalent cases of Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Sub-type specific prevalent cases of Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Diagnosed casesof Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Age-Specific diagnosed cases of Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Market size of Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Therapy wise market size of Gaucher Disease in the US, EU5,and Japan; 2016-2026
- Product Profile of Current Therapies: Cerdelga, Cerezyme, Vpriv, Zavesca, and Elelyso
- Marketed drug analysis of Gaucher Disease
- Pipeline product analysis of Gaucher Disease
- Detailed analysis of growth drivers, and challenges
- Detailed Company Profiling of Key Market Players:Pfizer, Sanofi, Shire Pharmaceuticals, and Johnson & Johnson
1. Executive Summary
2. Gaucher Disease: Overview
I. Signs and Symptoms
II. Advancement in Gaucher Disease
III. Classification
IV. Variants of Gaucher Disease
V. Etiology
VI. Pathophysiology
VII. Inheritance Pattern
VIII. Complications
IX. Diagnostic Algorithm
a. Gaucher Disease patients with Ashkenazi Origin
b. Gaucher Disease patients with Non- Ashkenazi Origin
3. Treatment
I. Treatment Algorithm
II. Treatment Guidelines
a. Screening and Management Guidelines of Hematological Malignancy
b. Criteria for using Enzyme Replacement Therapy
c. Treatment Recommendation for Adults with Type 1 Gaucher Disease
4. Epidemiology
I. United States
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
II. Germany
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
III. France
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
IV. United Kingdom
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
V. Spain
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
VI. Italy
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
VII. Japan
a. Prevalent Cases of Gaucher Disease
b. Sub-type Specific Prevalent Cases of Gaucher Disease
c. Diagnosed Cases of Gaucher Disease
d. Age-Specific Diagnosed Cases for Gaucher Disease
5. Market Size of Gaucher Disease
I. United States
a. Market Size of Gaucher Disease
b. Therapy wise market Size of Gaucher Disease
II. EU5
a. Germany
b. France
c. United Kingdom
d. Spain
e. Italy
III. Japan
a. Market Size of Gaucher Disease
b. Therapy wise market Size of Gaucher Disease
6. Therapeutic Approaches for Gaucher Disease
7. Competitive Landscape
8. Marketed Drug Profiles
I. Cerdelga (eliglustat)
a. Drug Description
b. Development Activities
c. Pharmacokinetics
d. Pharmacodynamics
e. Clinical Studies
f. Adverse Events
g. Cerdelga Sales
II. Cerezyme (imiglucerase)
a. Drug Description
b. Development Activities
c. Pharmacokinetics
d. Pharmacodynamics
e. Clinical Studies
f. Adverse Events
g. Cerezyme Sales
III. Vpriv (velaglucerase alfa)
a. Drug Description
b. Development Activities
c. Pharmacokinetics
d. Pharmacodynamics
e. Clinical Studies
f. Adverse Events
g. Vpriv Sales
IV. Zavesca (Miglustat)
a. Drug Description
b. Development Activities
c. Pharmacokinetics
d. Pharmacodynamics
e. Clinical Studies
f. Adverse Events
g. Zavesca Sales
V. Elelyso (taliglucerase alfa)
a. Drug Description
b. Development Activities
c. Pharmacokinetics
d. Pharmacodynamics
e. Clinical Studies
f. Adverse Events
g. Elelyso Sales
9. Marketed Drug Analysis
I. Marketed Drug Analysis by Molecule Type
II. Marketed Drug Analysis by Route of Administration
10. Pipeline Product Analysis
I. Pipeline Analysis by Stage of Development
II. Pipeline Analysis by Route of Administration
III. Pipeline Analysis by Therapeutic Class
IV. Pipeline Analysis by Targets
V. Pipeline Analysis by Molecule Type
11. Market Dynamics
I. Growth Drivers
a. Technological advancement
b. Emerging Therapies
c. Increasing Research Activities
II. Challenges
a. Diagnostic Barriers
b. Socioeconomic Barriers
12. Companies Profiles
I. Pfizer
a. Business Overview
b. Financial Overview
II. Sanofi
a. Business Overview
b. Financial Overview
III.Shire Pharmaceuticals
a. Business Overview
b. Financial Overview
IV. Johnson & Johnson
a. Business Overview
b. Financial Overview
13. About EffeMarket
14. Disclaimer
15. Contact US
Figure 1: Signs and Symptoms of Gaucher Disease
Figure 2: Advancement in Gaucher Disease (1/1)
Figure 3: Advancement in Gaucher Disease (1/2)
Figure 4: Classification of Gaucher Disease
Figure 5: Pathophysiology of Gaucher Disease
Figure 6: Inheritance Pattern of Gaucher Disease
Figure 7: Complications of Gaucher Disease
Figure 8: Diagnostic Algorithm for Gaucher Disease patients with Ashkenazi Origin
Figure 9: Diagnostic Algorithm for Gaucher Disease patients with the Non-Ashkenazi origin
Figure 10: Treatment Algorithm for Gaucher Disease
Figure 11: Prevalent cases of Gaucher Disease in 7 Major Markets (2016-2026)
Figure 12: Prevalent Cases of Gaucher Disease in the United States (2016-2026)
Figure 13: Sub-type Specific Prevalent Cases of Gaucher Disease in the United States (2016-2026)
Figure 14: Diagnosed Cases of Gaucher Disease in the United States (2016-2026)
Figure 15: Age-Specific Diagnosed Cases for Gaucher Disease in the United States (2016-2026)
Figure 16: Prevalent Cases of Gaucher Disease in Germany (2016-2026)
Figure 17: Sub-type Specific Prevalent Cases of Gaucher Disease in Germany (2016-2026)
Figure 18: Diagnosed Cases of Gaucher Disease in Germany (2016-2026)
Figure 19: Age-Specific Diagnosed Cases for Gaucher Disease in Germany (2017 &2026)
Figure 20: Prevalent Cases of Gaucher Disease in France (2016-2026)
Figure 21: Sub-type Specific Prevalent Cases of Gaucher Disease in France (2016-2026)
Figure 22: Diagnosed Cases of Gaucher Disease in France (2016-2026)
Figure 23: Age-Specific Diagnosed Cases for Gaucher Disease in France (2017 &2026)
Figure 24: Prevalent Cases of Gaucher Disease in the United Kingdom (2016-2026)
Figure 25: Sub-type Specific Prevalent Cases of Gaucher Disease in the United Kingdom (2016-2026)
Figure 26: Diagnosed Cases of Gaucher Disease in the United Kingdom (2016-2026)
Figure 27: Age-Specific Diagnosed Cases for Gaucher Disease in the United Kingdom (2017 &2026)
Figure 28: Prevalent Cases of Gaucher Disease in Spain (2016-2026)
Figure 29: Sub-type Specific Prevalent Cases of Gaucher Disease in Spain (2016-2026)
Figure 30: Diagnosed Cases of Gaucher Disease in Spain (2016-2026)
Figure 31: Age-Specific Diagnosed Cases for Gaucher Disease in Spain (2017 &2026)
Figure 32: Prevalent Cases of Gaucher Disease in Italy (2016-2026)
Figure 33: Sub-type Specific Prevalent Cases of Gaucher Disease in Italy (2016-2026)
Figure 34: Diagnosed Cases of Gaucher Disease in Italy (2016-2026)
Figure 35: Age-Specific Diagnosed Cases for Gaucher Disease in Italy (2017 &2026)
Figure 36: Prevalent Cases of Gaucher Disease in Japan (2016-2026)
Figure 37: Sub-type Specific Prevalent Cases of Gaucher Disease in Japan (2016-2026)
Figure 38: Diagnosed Cases of Gaucher Disease in Japan (2016-2026)
Figure 39: Age-Specific Diagnosed Cases for Gaucher Disease in Japan (2017 &2026)
Figure 40: 7 Major Market Size of Gaucher Disease, In USD Million (2016-2026)
Figure 41: Market Size of Gaucher Disease in the United States, in USD Million (2016-2026)
Figure 42: Therapy Wise Market Size of Gaucher Disease in the United States, in USD Million (2016-2026)
Figure 43: EU5 Market Size of Gaucher Disease, in USD Million (2016-2026)
Figure 44: Market Size of Gaucher Disease in Germany, in USD Million (2016-2026)
Figure 45: Therapy Wise Market Size of Gaucher Disease in Germany, in USD Million (2016-2026)
Figure 46: Market Size of Gaucher Disease in France, in USD Million (2016-2026)
Figure 47: Therapy Wise Market Size of Gaucher Disease in France, in USD Million (2016-2026)
Figure 48: Market Size of Gaucher Disease in the United Kingdom, in USD Million (2016-2026)
Figure 49: Therapy Wise Market Size of Gaucher Disease in the United Kingdom, in USD Million (2016-2026)
Figure 50: Market Size of Gaucher Disease in Spain, in USD Million (2016-2026)
Figure 51: Therapy Wise Market Size of Gaucher Disease in Spain, in USD Million (2016-2026)
Figure 52: Market Size of Gaucher Disease in Italy, in USD Million (2016-2026)
Figure 53: Therapy Wise Market Size of Gaucher Disease in Italy, in USD Million (2016-2026)
Figure 54: Market Size of Gaucher Disease in Japan, in USD Million (2016-2026)
Figure 55: Therapy Wise Market Size of Gaucher Disease in Japan, in USD Million (2016-2026)
Figure 56: Therapeutic Approaches for Gaucher Disease
Figure 57: Mechanism of Action of Substrate Reduction Therapy and Enzyme Replacement Therapy
Figure 58: Cerdelga Sales In USD Million, (2014-2017)
Figure 59: Cerezyme Sales in USD Million (2014-2017)
Figure 60: Vpriv Sales in USD Million (2014-2017)
Figure 61: Zavesca Sales in USD Million (2014-2017)
Figure 62 Elelyso Sales in USD Million (2014-2017)
Figure 63: Marketed Drug Analysis by Molecule Type
Figure 64: Marketed Drug Analysis by Molecule Type
Figure 65: Pipeline Analysis by Stage of Development
Figure 66: Pipeline Analysis by Route of Administration
Figure 67: Pipeline Analysis by Therapeutic Class
Figure 68: Pipeline Analysis by Targets
Figure 69: Pipeline Analysis by Molecule Type
Figure 70: Total Revenues of Pfizer in USD Billion (2014-2017)
Figure 71: Segmentation of Pfizer's Revenues (2017)
Figure 72: Total Revenues of Sanofi in USD Billion (2014-2017)
Figure 73: Segmentation of Sanofi's Revenues (2017)
Figure 74: Total Revenues of Shire Pharmaceuticals in USD Billion (2014-2017)
Figure 75: Segmentation of Shire's Revenues (2017)
Figure 76: Total Revenues of Johnson & Johnson in USD Billion (2014-2017)
Figure 77 : Segmentation of Johnson & Johnson 's Revenues (2017)
Table 1: Variants of Gaucher Disease
Table 2: Therapeutic Goals of Gaucher Disease
Table 3: Enzyme Replacement Therapies for Gaucher Disease
Table 4: Substrate Reduction Therapies for Gaucher Disease
Table 5: Supportive therapies for Gaucher Disease
Table 6: Screening and Management Guidelines of Hematological Malignancy for Gaucher Disease
Table 8: Treatment Recommendation for Children with Type 1 Gaucher Disease
Table 9: Prevalent Cases of Gaucher Disease in the United States (2016-2026)
Table 10: Sub-type Specific Prevalent Cases of Gaucher Disease in the United States (2016-2026)
Table 11: Diagnosed Cases of Gaucher Disease (2016-2026)
Table 12: Age-Specific Diagnosed Cases for Gaucher Disease in the United States (2016-2026)
Table 13: Prevalent Cases of Gaucher Disease in Germany (2016-2026)
Table 14: Sub-type Specific Prevalent Cases of Gaucher Disease in Germany (2016-2026)
Table 15: Diagnosed Cases of Gaucher Disease in Germany (2016-2026)
Table 16: Age-Specific Diagnosed Cases for Gaucher Disease in Germany (2016-2026)
Table 17: Prevalent Cases of Gaucher Disease in France (2016-2026)
Table 18: Sub-type Specific Prevalent Cases of Gaucher Disease in France (2016-2026)
Table 19: Diagnosed Cases of Gaucher Disease in France (2016-2026)
Table 20: Age-Specific Diagnosed Cases for Gaucher Disease in France (2016-2026)
Table 21: Prevalent Cases of Gaucher Disease in the United Kingdom (2016-2026)
Table 22: Sub-type Specific Prevalent Cases of Gaucher Disease in the United Kingdom (2016-2026)
Table 23: Diagnosed Cases of Gaucher Disease in the United Kingdom (2016-2026)
Table 24: Age-Specific Diagnosed Cases for Gaucher Disease in the United Kingdom (2016-2026)
Table 25: Prevalent Cases of Gaucher Disease in Spain (2016-2026)
Table 26: Sub-type Specific Prevalent Cases of Gaucher Disease in Spain (2016-2026)
Table 27: Diagnosed Cases of Gaucher Disease in Spain(2016-2026)
Table 28: Age-Specific Diagnosed Cases for Gaucher Disease in Spain (2016-2026)
Table 29: Prevalent Cases of Gaucher Disease in Italy (2016-2026)
Table 30: Sub-type Specific Prevalent Cases of Gaucher Disease in Italy (2016-2026)
Table 31: Diagnosed Cases of Gaucher Disease in Italy (2016-2026)
Table 32: Age-Specific Diagnosed Cases for Gaucher Disease in Italy (2016-2026)
Table 33: Prevalent Cases of Gaucher Disease in Japan (2016-2026)
Table 34: Sub-type Specific Prevalent Cases of Gaucher Disease in Japan (2016-2026)
Table 35: Diagnosed Cases of Gaucher Disease in Japan (2016-2026)
Table 36: Age-Specific Diagnosed Cases for Gaucher Disease in Japan (2016-2026)
Table 37: Market Size of Gaucher Disease in the United States, in USD Million (2016-2026)
Table 38: Therapy Wise Market Size of Gaucher Disease in the United States, in USD Million (2016-2026)
Table 39: Market Size of Gaucher Disease in Germany, in USD Million (2016-2026)
Table 40: Therapy Wise Market Size of Gaucher Disease in Germany, in USD Million (2016-2026)
Table 41: Therapy Market Size of Gaucher Disease in France, in USD Million (2016-2026)
Table 42: Therapy Wise Market Size of Gaucher Disease in France, in USD Million (2016-2026)
Table 43: Market Size of Gaucher Disease in the United Kingdom, in USD Million (2016-2026)
Table 44: Therapy Wise Market Size of Gaucher Disease in the United Kingdom, in USD Million (2016-2026)
Table 45: Market Size of Gaucher Disease in Spain, in USD Million (2016-2026)
Table 46: Therapy Wise Market Size of Gaucher Disease in Spain, in USD Million (2016-2026)
Table 47: Market Size of Gaucher Disease in Italy, in USD Million (2016-2026)
Table 48: Therapy Wise Market Size of Gaucher Disease in Italy, in USD Million (2016-2026)
Table 49: Market Size of Gaucher Disease in Japan, in USD Million (2016-2026)
Table 50: Therapy Wise Market Size of Gaucher Disease in Japan, in USD Million (2016-2026)
Table 51: Competitive Landscape for Gaucher Disease
Table 52: Marketed Drug Analysis for Gaucher Disease
Table 53: Pipeline Product Analysis for Gaucher Disease
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