Key Features of the Reports
- The report provides granular level information about the market size, regional market share, historic market (2019-2023), and forecast (2024-2030)
- The report covers in-detail insights about the competitor’s overview, company share analysis, key market developments, and their key strategies
- The report outlines drivers, restraints, unmet needs, and trends that are currently affecting the market
- The report tracks recent innovations, key developments, and start-up details that are actively working in the market
- The report provides a plethora of information about market entry strategies, regulatory framework, and reimbursement scenario
Rare Diseases Therapeutics Market Segmentation
1. Executive Summary
2. Global Rare Diseases Therapeutics Market Introduction
2.1. Global Rare Diseases Therapeutics Market - Taxonomy
2.2. Global Rare Diseases Therapeutics Market - Definitions
2.2.1. By Disease Indication
2.2.2. By Product Class
2.2.3. By End User
2.2.4. By Region
3. Global Rare Diseases Therapeutics Market Dynamics
3.1. Drivers
3.2. Restraints
3.3. Opportunities/Unmet Needs of the Market
3.4. Trends
3.5. Product Landscape
3.6. New Product Launches
3.7. Impact of COVID 19 on Market
3.8. USP
3.9. Macro Trends
4. Global Rare Diseases Therapeutics Market Analysis, 2017-2021 and Forecast 2022-2028
4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
4.3. Market Opportunity Analysis
5. Global Rare Diseases Therapeutics Market By Disease Indication, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
5.1. Lysosomal storage diseases (LSDs)
5.1.1. Alpha-Mannosidosis
5.1.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.1.3. Market Opportunity Analysis
5.1.2. Fabry Disease
5.1.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.2.3. Market Opportunity Analysis
5.1.3. Pompe disease
5.1.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.3.3. Market Opportunity Analysis
5.1.4. Gaucher Disease
5.1.4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.4.3. Market Opportunity Analysis
5.1.5. Others
5.1.5.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.5.3. Market Opportunity Analysis
5.2. Mantle cell lymphoma leukemia
5.2.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.2.3. Market Opportunity Analysis
5.3. Glanzmann’s thrombasthenia
5.3.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.3.3. Market Opportunity Analysis
5.4. Goodpasture Syndrome
5.4.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.4.3. Market Opportunity Analysis
5.5. Others
5.5.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.5.3. Market Opportunity Analysis
6. Global Rare Diseases Therapeutics Market By Product Class, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
6.1. Enzyme Replacement Therapies
6.1.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.1.3. Market Opportunity Analysis
6.2. CNS Targeting Candidates
6.2.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.2.3. Market Opportunity Analysis
6.3. Plasma-derived factors
6.3.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.3.3. Market Opportunity Analysis
6.4. Antibody Therapy
6.4.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.4.3. Market Opportunity Analysis
6.5. Iron Chelators
6.5.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.5.3. Market Opportunity Analysis
6.6. Others
6.6.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.6.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.6.3. Market Opportunity Analysis
7. Global Rare Diseases Therapeutics Market By End User, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
7.1. Pediatric
7.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.1.3. Market Opportunity Analysis
7.2. Adult
7.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.2.3. Market Opportunity Analysis
7.3. Geriatric
7.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.3.3. Market Opportunity Analysis
7.Global Rare Diseases Therapeutics Market By By Region, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
7.1. North America
7.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.1.3. Market Opportunity Analysis
7.2. Europe
7.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.2.3. Market Opportunity Analysis
7.3. Asia Pacific (APAC)
7.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.3.3. Market Opportunity Analysis
7.4. Latin America
7.4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.4.3. Market Opportunity Analysis
7.5. Middle East and Africa (MEA)
7.5.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.5.3. Market Opportunity Analysis
7.6. Global Rare Diseases Therapeutics Market - Opportunity Analysis Index, By Product Class, Disease Indication, End User, and By Region, 2022-2028
8. North America Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
8.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.1.1. Lysosomal storage diseases (LSDs)
8.1.1.1. Alpha-Mannosidosis
8.1.1.2. Fabry Disease
8.1.1.3. Pompe disease
8.1.1.4. Gaucher Disease
8.1.1.5. Others
8.1.2. Mantle cell lymphoma leukemia
8.1.3. Glanzmann’s thrombasthenia
8.1.4. Goodpasture Syndrome
8.1.5. Others
8.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.2.1. Enzyme Replacement Therapies
8.2.2. CNS Targeting Candidates
8.2.3. Plasma-derived factors
8.2.4. Antibody Therapy
8.2.5. Iron Chelators
8.2.6. Others
8.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.3.1. Pediatric
8.3.2. Adult
8.3.3. Geriatric
8.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.4.1. United States of America (USA)
8.4.2. Canada
9. Europe Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
9.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.1.1. Lysosomal storage diseases (LSDs)
9.1.1.1. Alpha-Mannosidosis
9.1.1.2. Fabry Disease
9.1.1.3. Pompe disease
9.1.1.4. Gaucher Disease
9.1.1.5. Others
9.1.2. Mantle cell lymphoma leukemia
9.1.3. Glanzmann’s thrombasthenia
9.1.4. Goodpasture Syndrome
9.1.5. Others
9.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.2.1. Enzyme Replacement Therapies
9.2.2. CNS Targeting Candidates
9.2.3. Plasma-derived factors
9.2.4. Antibody Therapy
9.2.5. Iron Chelators
9.2.6. Others
9.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.3.1. Pediatric
9.3.2. Adult
9.3.3. Geriatric
9.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.4.1. Germany
9.4.2. France
9.4.3. Italy
9.4.4. United Kingdom (UK)
9.4.5. Spain
9.4.6. Rest of EU
10. Asia Pacific (APAC) Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
10.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.1.1. Lysosomal storage diseases (LSDs)
10.1.1.1. Alpha-Mannosidosis
10.1.1.2. Fabry Disease
10.1.1.3. Pompe disease
10.1.1.4. Gaucher Disease
10.1.1.5. Others
10.1.2. Mantle cell lymphoma leukemia
10.1.3. Glanzmann’s thrombasthenia
10.1.4. Goodpasture Syndrome
10.1.5. Others
10.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.2.1. Enzyme Replacement Therapies
10.2.2. CNS Targeting Candidates
10.2.3. Plasma-derived factors
10.2.4. Antibody Therapy
10.2.5. Iron Chelators
10.2.6. Others
10.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.3.1. Pediatric
10.3.2. Adult
10.3.3. Geriatric
10.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.4.1. China
10.4.2. India
10.4.3. Australia and New Zealand (ANZ)
10.4.4. Japan
10.4.5. Rest of APAC
11. Latin America Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
11.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.1.1. Lysosomal storage diseases (LSDs)
11.1.1.1. Alpha-Mannosidosis
11.1.1.2. Fabry Disease
11.1.1.3. Pompe disease
11.1.1.4. Gaucher Disease
11.1.1.5. Others
11.1.2. Mantle cell lymphoma leukemia
11.1.3. Glanzmann’s thrombasthenia
11.1.4. Goodpasture Syndrome
11.1.5. Others
11.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.2.1. Enzyme Replacement Therapies
11.2.2. CNS Targeting Candidates
11.2.3. Plasma-derived factors
11.2.4. Antibody Therapy
11.2.5. Iron Chelators
11.2.6. Others
11.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.3.1. Pediatric
11.3.2. Adult
11.3.3. Geriatric
11.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.4.1. Brazil
11.4.2. Mexico
11.4.3. Rest of LA
12. Middle East and Africa (MEA) Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
12.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.1.1. Lysosomal storage diseases (LSDs)
12.1.1.1. Alpha-Mannosidosis
12.1.1.2. Fabry Disease
12.1.1.3. Pompe disease
12.1.1.4. Gaucher Disease
12.1.1.5. Others
12.1.2. Mantle cell lymphoma leukemia
12.1.3. Glanzmann’s thrombasthenia
12.1.4. Goodpasture Syndrome
12.1.5. Others
12.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.2.1. Enzyme Replacement Therapies
12.2.2. CNS Targeting Candidates
12.2.3. Plasma-derived factors
12.2.4. Antibody Therapy
12.2.5. Iron Chelators
12.2.6. Others
12.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.3.1. Pediatric
12.3.2. Adult
12.3.3. Geriatric
12.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.4.1. GCC Countries
12.4.2. South Africa
12.4.3. Rest of MEA
13. Competition Landscape
13.1. Strategic Dashboard of Top Market Players
13.2. Company Profiles (Introduction, Financial Analysis, By Product Offerings, Key Developments, Strategies, and SWOT Analysis)
13.2.1. Amgen Inc.
13.2.2. Biomarin Pharmaceuticals
13.2.3. Bayer AG
13.2.4. Bristol-Myers Squibb Company
13.2.5. AstraZeneca (Alexion Pharmaceuticals Inc.)
13.2.6. Apellis.
13.2.7. Enzyvant.
13.2.8. Travere Therapeutics.
13.2.9. Pharnext.
13.2.10. Alchemab Therapeutics.
13.2.11. iOnctura.
13.2.12. PTC Therapeutics.
13.2.13. Rocket Pharmaceuticals.
14. Research Methodology
15. Key Assumptions and Acronyms
2. Global Rare Diseases Therapeutics Market Introduction
2.1. Global Rare Diseases Therapeutics Market - Taxonomy
2.2. Global Rare Diseases Therapeutics Market - Definitions
2.2.1. By Disease Indication
2.2.2. By Product Class
2.2.3. By End User
2.2.4. By Region
3. Global Rare Diseases Therapeutics Market Dynamics
3.1. Drivers
3.2. Restraints
3.3. Opportunities/Unmet Needs of the Market
3.4. Trends
3.5. Product Landscape
3.6. New Product Launches
3.7. Impact of COVID 19 on Market
3.8. USP
3.9. Macro Trends
4. Global Rare Diseases Therapeutics Market Analysis, 2017-2021 and Forecast 2022-2028
4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
4.3. Market Opportunity Analysis
5. Global Rare Diseases Therapeutics Market By Disease Indication, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
5.1. Lysosomal storage diseases (LSDs)
5.1.1. Alpha-Mannosidosis
5.1.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.1.3. Market Opportunity Analysis
5.1.2. Fabry Disease
5.1.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.2.3. Market Opportunity Analysis
5.1.3. Pompe disease
5.1.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.3.3. Market Opportunity Analysis
5.1.4. Gaucher Disease
5.1.4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.4.3. Market Opportunity Analysis
5.1.5. Others
5.1.5.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
5.1.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.1.5.3. Market Opportunity Analysis
5.2. Mantle cell lymphoma leukemia
5.2.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.2.3. Market Opportunity Analysis
5.3. Glanzmann’s thrombasthenia
5.3.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.3.3. Market Opportunity Analysis
5.4. Goodpasture Syndrome
5.4.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.4.3. Market Opportunity Analysis
5.5. Others
5.5.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
5.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
5.5.3. Market Opportunity Analysis
6. Global Rare Diseases Therapeutics Market By Product Class, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
6.1. Enzyme Replacement Therapies
6.1.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.1.3. Market Opportunity Analysis
6.2. CNS Targeting Candidates
6.2.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.2.3. Market Opportunity Analysis
6.3. Plasma-derived factors
6.3.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.3.3. Market Opportunity Analysis
6.4. Antibody Therapy
6.4.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.4.3. Market Opportunity Analysis
6.5. Iron Chelators
6.5.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.5.3. Market Opportunity Analysis
6.6. Others
6.6.1. Market Analysis, 2017-2021 and Forecast,.2022-2028, (Sales Value USD Million)
6.6.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
6.6.3. Market Opportunity Analysis
7. Global Rare Diseases Therapeutics Market By End User, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
7.1. Pediatric
7.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.1.3. Market Opportunity Analysis
7.2. Adult
7.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.2.3. Market Opportunity Analysis
7.3. Geriatric
7.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.3.3. Market Opportunity Analysis
7.Global Rare Diseases Therapeutics Market By By Region, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
7.1. North America
7.1.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.1.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.1.3. Market Opportunity Analysis
7.2. Europe
7.2.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.2.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.2.3. Market Opportunity Analysis
7.3. Asia Pacific (APAC)
7.3.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.3.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.3.3. Market Opportunity Analysis
7.4. Latin America
7.4.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.4.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.4.3. Market Opportunity Analysis
7.5. Middle East and Africa (MEA)
7.5.1. Market Analysis, 2017-2021 and Forecast, 2022-2028, (Sales Value USD Million)
7.5.2. Year-Over-Year (Y-o-Y) Growth Analysis (%) and Market Share Analysis (%)
7.5.3. Market Opportunity Analysis
7.6. Global Rare Diseases Therapeutics Market - Opportunity Analysis Index, By Product Class, Disease Indication, End User, and By Region, 2022-2028
8. North America Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
8.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.1.1. Lysosomal storage diseases (LSDs)
8.1.1.1. Alpha-Mannosidosis
8.1.1.2. Fabry Disease
8.1.1.3. Pompe disease
8.1.1.4. Gaucher Disease
8.1.1.5. Others
8.1.2. Mantle cell lymphoma leukemia
8.1.3. Glanzmann’s thrombasthenia
8.1.4. Goodpasture Syndrome
8.1.5. Others
8.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.2.1. Enzyme Replacement Therapies
8.2.2. CNS Targeting Candidates
8.2.3. Plasma-derived factors
8.2.4. Antibody Therapy
8.2.5. Iron Chelators
8.2.6. Others
8.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.3.1. Pediatric
8.3.2. Adult
8.3.3. Geriatric
8.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
8.4.1. United States of America (USA)
8.4.2. Canada
9. Europe Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
9.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.1.1. Lysosomal storage diseases (LSDs)
9.1.1.1. Alpha-Mannosidosis
9.1.1.2. Fabry Disease
9.1.1.3. Pompe disease
9.1.1.4. Gaucher Disease
9.1.1.5. Others
9.1.2. Mantle cell lymphoma leukemia
9.1.3. Glanzmann’s thrombasthenia
9.1.4. Goodpasture Syndrome
9.1.5. Others
9.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.2.1. Enzyme Replacement Therapies
9.2.2. CNS Targeting Candidates
9.2.3. Plasma-derived factors
9.2.4. Antibody Therapy
9.2.5. Iron Chelators
9.2.6. Others
9.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.3.1. Pediatric
9.3.2. Adult
9.3.3. Geriatric
9.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
9.4.1. Germany
9.4.2. France
9.4.3. Italy
9.4.4. United Kingdom (UK)
9.4.5. Spain
9.4.6. Rest of EU
10. Asia Pacific (APAC) Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
10.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.1.1. Lysosomal storage diseases (LSDs)
10.1.1.1. Alpha-Mannosidosis
10.1.1.2. Fabry Disease
10.1.1.3. Pompe disease
10.1.1.4. Gaucher Disease
10.1.1.5. Others
10.1.2. Mantle cell lymphoma leukemia
10.1.3. Glanzmann’s thrombasthenia
10.1.4. Goodpasture Syndrome
10.1.5. Others
10.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.2.1. Enzyme Replacement Therapies
10.2.2. CNS Targeting Candidates
10.2.3. Plasma-derived factors
10.2.4. Antibody Therapy
10.2.5. Iron Chelators
10.2.6. Others
10.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.3.1. Pediatric
10.3.2. Adult
10.3.3. Geriatric
10.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
10.4.1. China
10.4.2. India
10.4.3. Australia and New Zealand (ANZ)
10.4.4. Japan
10.4.5. Rest of APAC
11. Latin America Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
11.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.1.1. Lysosomal storage diseases (LSDs)
11.1.1.1. Alpha-Mannosidosis
11.1.1.2. Fabry Disease
11.1.1.3. Pompe disease
11.1.1.4. Gaucher Disease
11.1.1.5. Others
11.1.2. Mantle cell lymphoma leukemia
11.1.3. Glanzmann’s thrombasthenia
11.1.4. Goodpasture Syndrome
11.1.5. Others
11.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.2.1. Enzyme Replacement Therapies
11.2.2. CNS Targeting Candidates
11.2.3. Plasma-derived factors
11.2.4. Antibody Therapy
11.2.5. Iron Chelators
11.2.6. Others
11.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.3.1. Pediatric
11.3.2. Adult
11.3.3. Geriatric
11.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
11.4.1. Brazil
11.4.2. Mexico
11.4.3. Rest of LA
12. Middle East and Africa (MEA) Rare Diseases Therapeutics Market, 2017-2021 and Forecast 2022-2028 (Sales Value USD Million)
12.1. Disease Indication Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.1.1. Lysosomal storage diseases (LSDs)
12.1.1.1. Alpha-Mannosidosis
12.1.1.2. Fabry Disease
12.1.1.3. Pompe disease
12.1.1.4. Gaucher Disease
12.1.1.5. Others
12.1.2. Mantle cell lymphoma leukemia
12.1.3. Glanzmann’s thrombasthenia
12.1.4. Goodpasture Syndrome
12.1.5. Others
12.2. Product Class Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.2.1. Enzyme Replacement Therapies
12.2.2. CNS Targeting Candidates
12.2.3. Plasma-derived factors
12.2.4. Antibody Therapy
12.2.5. Iron Chelators
12.2.6. Others
12.3. End User Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.3.1. Pediatric
12.3.2. Adult
12.3.3. Geriatric
12.4. Country Analysis, 2017-2021 and Forecast 2022-2028 by Sales Value USD Million, Y-o-Y Growth (%), and Market Share (%)
12.4.1. GCC Countries
12.4.2. South Africa
12.4.3. Rest of MEA
13. Competition Landscape
13.1. Strategic Dashboard of Top Market Players
13.2. Company Profiles (Introduction, Financial Analysis, By Product Offerings, Key Developments, Strategies, and SWOT Analysis)
13.2.1. Amgen Inc.
13.2.2. Biomarin Pharmaceuticals
13.2.3. Bayer AG
13.2.4. Bristol-Myers Squibb Company
13.2.5. AstraZeneca (Alexion Pharmaceuticals Inc.)
13.2.6. Apellis.
13.2.7. Enzyvant.
13.2.8. Travere Therapeutics.
13.2.9. Pharnext.
13.2.10. Alchemab Therapeutics.
13.2.11. iOnctura.
13.2.12. PTC Therapeutics.
13.2.13. Rocket Pharmaceuticals.
14. Research Methodology
15. Key Assumptions and Acronyms
Amgen Inc.
Biomarin Pharmaceuticals
Bayer AG
Bristol-Myers Squibb Company
AstraZeneca (Alexion Pharmaceuticals Inc.)
Apellis.
Enzyvant.
Travere Therapeutics.
Pharnext.
Alchemab Therapeutics.
iOnctura.
PTC Therapeutics.
Rocket Pharmaceuticals.
Biomarin Pharmaceuticals
Bayer AG
Bristol-Myers Squibb Company
AstraZeneca (Alexion Pharmaceuticals Inc.)
Apellis.
Enzyvant.
Travere Therapeutics.
Pharnext.
Alchemab Therapeutics.
iOnctura.
PTC Therapeutics.
Rocket Pharmaceuticals.