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Global Orphan Drugs Market Analysis and Forecast 2025-2031

Publisher APO Research, Inc.
Published Aug 16, 2025
Length 197 Pages
SKU # APRC20361032

Description

Summary

According to APO Research, The global Orphan Drugs market is projected to grow from US$ million in 2025 to US$ million by 2031, at a Compound Annual Growth Rate (CAGR) of % during the forecast period.

The North America market for Orphan Drugs is estimated to increase from $ million in 2025 to reach $ million by 2031, at a CAGR of % during the forecast period of 2025 through 2031.

Asia-Pacific market for Orphan Drugs is estimated to increase from $ million in 2025 to reach $ million by 2031, at a CAGR of % during the forecast period of 2025 through 2031.

The China market for Orphan Drugs is estimated to increase from $ million in 2025 to reach $ million by 2031, at a CAGR of % during the forecast period of 2025 through 2031.

Europe market for Orphan Drugs is estimated to increase from $ million in 2025 to reach $ million by 2031, at a CAGR of % during the forecast period of 2025 through 2031.

The major global companies of Orphan Drugs include AstraZeneca, AbbVie, Amgen, Bristol-Myers Squibb, Biogen, Vertex Pharmaceuticals, Pfizer, Eli Lilly and Roche, etc. In 2024, the world's top three vendors accounted for approximately % of the revenue.

Report Includes

This report presents an overview of global market for Orphan Drugs, market size. Analyses of the global market trends, with historic market revenue data for 2020 - 2024, estimates for 2025, and projections of CAGR through 2031.

This report researches the key producers of Orphan Drugs, also provides the revenue of main regions and countries. Of the upcoming market potential for Orphan Drugs, and key regions or countries of focus to forecast this market into various segments and sub-segments. Country specific data and market value analysis for the U.S., Canada, Mexico, Brazil, China, Japan, South Korea, Southeast Asia, India, Germany, the U.K., Italy, Middle East, Africa, and Other Countries.

This report focuses on the Orphan Drugs revenue, market share and industry ranking of main manufacturers, data from 2020 to 2025. Identification of the major stakeholders in the global Orphan Drugs market, and analysis of their competitive landscape and market positioning based on recent developments and segmental revenues. This report will help stakeholders to understand the competitive landscape and gain more insights and position their businesses and market strategies in a better way.

This report analyzes the segments data by Type and by Application, revenue, and growth rate, from 2020 to 2031. Evaluation and forecast the market size for Orphan Drugs revenue, projected growth trends, production technology, application and end-user industry.

Orphan Drugs Segment by Company

AstraZeneca
AbbVie
Amgen
Bristol-Myers Squibb
Biogen
Vertex Pharmaceuticals
Pfizer
Eli Lilly
Roche
Novartis
Johnson & Johnson
Sanofi
Takeda
Orphan Drugs Segment by Type

Metabolic Disorders
Pulmonary
Endocrinology
Neurology
Cardio-vascular
Hematology
Oncology
Others
Orphan Drugs Segment by Application

Adult
Teenager
Baby and Child
Other
Orphan Drugs Segment by Region

North America
United States
Canada
Europe
Germany
France
U.K.
Italy
Netherlands
Nordic Countries
Asia-Pacific
China
Japan
South Korea
India
Australia
China Taiwan
Southeast Asia
South America
Mexico
Brazil
Argentina
Middle East & Africa
Turkey
Saudi Arabia
UAE

Study Objectives

1. To analyze and research the global status and future forecast, involving growth rate (CAGR), market share, historical and forecast.
2. To present the key players, revenue, market share, and Recent Developments.
3. To split the breakdown data by regions, type, manufacturers, and Application.
4. To analyze the global and key regions market potential and advantage, opportunity and challenge, restraints, and risks.
5. To identify significant trends, drivers, influence factors in global and regions.
6. To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.

Reasons to Buy This Report

1. This report will help the readers to understand the competition within the industries and strategies for the competitive environment to enhance the potential profit. The report also focuses on the competitive landscape of the global Orphan Drugs market, and introduces in detail the market share, industry ranking, competitor ecosystem, market performance, new product development, operation situation, expansion, and acquisition. etc. of the main players, which helps the readers to identify the main competitors and deeply understand the competition pattern of the market.
2. This report will help stakeholders to understand the global industry status and trends of Orphan Drugs and provides them with information on key market drivers, restraints, challenges, and opportunities.
3. This report will help stakeholders to understand competitors better and gain more insights to strengthen their position in their businesses. The competitive landscape section includes the market share and rank (in market size), competitor ecosystem, new product development, expansion, and acquisition.
4. This report stays updated with novel technology integration, features, and the latest developments in the market.
5. This report helps stakeholders to gain insights into which regions to target globally.
6. This report helps stakeholders to gain insights into the end-user perception concerning the adoption of Orphan Drugs.
7. This report helps stakeholders to identify some of the key players in the market and understand their valuable contribution.

Chapter Outline

Chapter 1: Introduces the report scope of the report, executive summary of different market segments (product type, application, etc), including the market size of each market segment, future development potential, and so on. It offers a high-level view of the current state of the market and its likely evolution in the short to mid-term, and long term.
Chapter 2: Introduces the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.
Chapter 3: Revenue of Orphan Drugs in global and regional level. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space, and capacity of each country in the world.
Chapter 4: Detailed analysis of Orphan Drugs company competitive landscape, revenue, market share and industry ranking, latest development plan, merger, and acquisition information, etc.
Chapter 5: Provides the analysis of various market segments by type, covering the revenue, and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 6: Provides the analysis of various market segments by application, covering the revenue, and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 7: Provides profiles of key companies, introducing the basic situation of the main companies in the market in detail, including product descriptions and specifications, Orphan Drugs revenue, gross margin, and recent development, etc.
Chapter 8: North America by type, by application and by country, revenue for each segment.
Chapter 9: Europe by type, by application and by country, revenue for each segment.
Chapter 10: China type, by application, revenue for each segment.
Chapter 11: Asia (excluding China) type, by application and by region, revenue for each segment.
Chapter 12: South America, Middle East and Africa by type, by application and by country, revenue for each segment.
Chapter 13: The main concluding insights of the report.

Table of Contents

197 Pages
1 Market Overview
1.1 Product Definition
1.2 Orphan Drugs Market by Type
1.2.1 Global Orphan Drugs Market Size by Type, 2020 VS 2024 VS 2031
1.2.2 Metabolic Disorders
1.2.3 Pulmonary
1.2.4 Endocrinology
1.2.5 Neurology
1.2.6 Cardio-vascular
1.2.7 Hematology
1.2.8 Oncology
1.2.9 Others
1.3 Orphan Drugs Market by Application
1.3.1 Global Orphan Drugs Market Size by Application, 2020 VS 2024 VS 2031
1.3.2 Adult
1.3.3 Teenager
1.3.4 Baby and Child
1.3.5 Other
1.4 Assumptions and Limitations
1.5 Study Goals and Objectives
2 Orphan Drugs Market Dynamics
2.1 Orphan Drugs Industry Trends
2.2 Orphan Drugs Industry Drivers
2.3 Orphan Drugs Industry Opportunities and Challenges
2.4 Orphan Drugs Industry Restraints
3 Global Growth Perspective
3.1 Global Orphan Drugs Market Perspective (2020-2031)
3.2 Global Orphan Drugs Growth Trends by Region
3.2.1 Global Orphan Drugs Market Size by Region: 2020 VS 2024 VS 2031
3.2.2 Global Orphan Drugs Market Size by Region (2020-2025)
3.2.3 Global Orphan Drugs Market Size by Region (2026-2031)
4 Competitive Landscape by Players
4.1 Global Orphan Drugs Revenue by Players
4.1.1 Global Orphan Drugs Revenue by Players (2020-2025)
4.1.2 Global Orphan Drugs Revenue Market Share by Players (2020-2025)
4.1.3 Global Orphan Drugs Players Revenue Share Top 10 and Top 5 in 2024
4.2 Global Orphan Drugs Key Players Ranking, 2023 VS 2024 VS 2025
4.3 Global Orphan Drugs Key Players Headquarters & Area Served
4.4 Global Orphan Drugs Players, Product Type & Application
4.5 Global Orphan Drugs Players Establishment Date
4.6 Market Competitive Analysis
4.6.1 Global Orphan Drugs Market CR5 and HHI
4.6.3 2024 Orphan Drugs Tier 1, Tier 2, and Tier 3
5 Orphan Drugs Market Size by Type
5.1 Global Orphan Drugs Revenue by Type (2020 VS 2024 VS 2031)
5.2 Global Orphan Drugs Revenue by Type (2020-2031)
5.3 Global Orphan Drugs Revenue Market Share by Type (2020-2031)
6 Orphan Drugs Market Size by Application
6.1 Global Orphan Drugs Revenue by Application (2020 VS 2024 VS 2031)
6.2 Global Orphan Drugs Revenue by Application (2020-2031)
6.3 Global Orphan Drugs Revenue Market Share by Application (2020-2031)
7 Company Profiles
7.1 AstraZeneca
7.1.1 AstraZeneca Comapny Information
7.1.2 AstraZeneca Business Overview
7.1.3 AstraZeneca Orphan Drugs Revenue and Gross Margin (2020-2025)
7.1.4 AstraZeneca Orphan Drugs Product Portfolio
7.1.5 AstraZeneca Recent Developments
7.2 AbbVie
7.2.1 AbbVie Comapny Information
7.2.2 AbbVie Business Overview
7.2.3 AbbVie Orphan Drugs Revenue and Gross Margin (2020-2025)
7.2.4 AbbVie Orphan Drugs Product Portfolio
7.2.5 AbbVie Recent Developments
7.3 Amgen
7.3.1 Amgen Comapny Information
7.3.2 Amgen Business Overview
7.3.3 Amgen Orphan Drugs Revenue and Gross Margin (2020-2025)
7.3.4 Amgen Orphan Drugs Product Portfolio
7.3.5 Amgen Recent Developments
7.4 Bristol-Myers Squibb
7.4.1 Bristol-Myers Squibb Comapny Information
7.4.2 Bristol-Myers Squibb Business Overview
7.4.3 Bristol-Myers Squibb Orphan Drugs Revenue and Gross Margin (2020-2025)
7.4.4 Bristol-Myers Squibb Orphan Drugs Product Portfolio
7.4.5 Bristol-Myers Squibb Recent Developments
7.5 Biogen
7.5.1 Biogen Comapny Information
7.5.2 Biogen Business Overview
7.5.3 Biogen Orphan Drugs Revenue and Gross Margin (2020-2025)
7.5.4 Biogen Orphan Drugs Product Portfolio
7.5.5 Biogen Recent Developments
7.6 Vertex Pharmaceuticals
7.6.1 Vertex Pharmaceuticals Comapny Information
7.6.2 Vertex Pharmaceuticals Business Overview
7.6.3 Vertex Pharmaceuticals Orphan Drugs Revenue and Gross Margin (2020-2025)
7.6.4 Vertex Pharmaceuticals Orphan Drugs Product Portfolio
7.6.5 Vertex Pharmaceuticals Recent Developments
7.7 Pfizer
7.7.1 Pfizer Comapny Information
7.7.2 Pfizer Business Overview
7.7.3 Pfizer Orphan Drugs Revenue and Gross Margin (2020-2025)
7.7.4 Pfizer Orphan Drugs Product Portfolio
7.7.5 Pfizer Recent Developments
7.8 Eli Lilly
7.8.1 Eli Lilly Comapny Information
7.8.2 Eli Lilly Business Overview
7.8.3 Eli Lilly Orphan Drugs Revenue and Gross Margin (2020-2025)
7.8.4 Eli Lilly Orphan Drugs Product Portfolio
7.8.5 Eli Lilly Recent Developments
7.9 Roche
7.9.1 Roche Comapny Information
7.9.2 Roche Business Overview
7.9.3 Roche Orphan Drugs Revenue and Gross Margin (2020-2025)
7.9.4 Roche Orphan Drugs Product Portfolio
7.9.5 Roche Recent Developments
7.10 Novartis
7.10.1 Novartis Comapny Information
7.10.2 Novartis Business Overview
7.10.3 Novartis Orphan Drugs Revenue and Gross Margin (2020-2025)
7.10.4 Novartis Orphan Drugs Product Portfolio
7.10.5 Novartis Recent Developments
7.11 Johnson & Johnson
7.11.1 Johnson & Johnson Comapny Information
7.11.2 Johnson & Johnson Business Overview
7.11.3 Johnson & Johnson Orphan Drugs Revenue and Gross Margin (2020-2025)
7.11.4 Johnson & Johnson Orphan Drugs Product Portfolio
7.11.5 Johnson & Johnson Recent Developments
7.12 Sanofi
7.12.1 Sanofi Comapny Information
7.12.2 Sanofi Business Overview
7.12.3 Sanofi Orphan Drugs Revenue and Gross Margin (2020-2025)
7.12.4 Sanofi Orphan Drugs Product Portfolio
7.12.5 Sanofi Recent Developments
7.13 Takeda
7.13.1 Takeda Comapny Information
7.13.2 Takeda Business Overview
7.13.3 Takeda Orphan Drugs Revenue and Gross Margin (2020-2025)
7.13.4 Takeda Orphan Drugs Product Portfolio
7.13.5 Takeda Recent Developments
8 North America
8.1 North America Orphan Drugs Revenue (2020-2031)
8.2 North America Orphan Drugs Revenue by Type (2020-2031)
8.2.1 North America Orphan Drugs Revenue by Type (2020-2025)
8.2.2 North America Orphan Drugs Revenue by Type (2026-2031)
8.3 North America Orphan Drugs Revenue Share by Type (2020-2031)
8.4 North America Orphan Drugs Revenue by Application (2020-2031)
8.4.1 North America Orphan Drugs Revenue by Application (2020-2025)
8.4.2 North America Orphan Drugs Revenue by Application (2026-2031)
8.5 North America Orphan Drugs Revenue Share by Application (2020-2031)
8.6 North America Orphan Drugs Revenue by Country
8.6.1 North America Orphan Drugs Revenue by Country (2020 VS 2024 VS 2031)
8.6.2 North America Orphan Drugs Revenue by Country (2020-2025)
8.6.3 North America Orphan Drugs Revenue by Country (2026-2031)
8.6.4 United States
8.6.5 Canada
9 Europe
9.1 Europe Orphan Drugs Revenue (2020-2031)
9.2 Europe Orphan Drugs Revenue by Type (2020-2031)
9.2.1 Europe Orphan Drugs Revenue by Type (2020-2025)
9.2.2 Europe Orphan Drugs Revenue by Type (2026-2031)
9.3 Europe Orphan Drugs Revenue Share by Type (2020-2031)
9.4 Europe Orphan Drugs Revenue by Application (2020-2031)
9.4.1 Europe Orphan Drugs Revenue by Application (2020-2025)
9.4.2 Europe Orphan Drugs Revenue by Application (2026-2031)
9.5 Europe Orphan Drugs Revenue Share by Application (2020-2031)
9.6 Europe Orphan Drugs Revenue by Country
9.6.1 Europe Orphan Drugs Revenue by Country (2020 VS 2024 VS 2031)
9.6.2 Europe Orphan Drugs Revenue by Country (2020-2025)
9.6.3 Europe Orphan Drugs Revenue by Country (2026-2031)
9.6.4 Germany
9.6.5 France
9.6.6 U.K.
9.6.7 Italy
9.6.8 Netherlands
9.6.9 Nordic Countries
10 China
10.1 China Orphan Drugs Revenue (2020-2031)
10.2 China Orphan Drugs Revenue by Type (2020-2031)
10.2.1 China Orphan Drugs Revenue by Type (2020-2025)
10.2.2 China Orphan Drugs Revenue by Type (2026-2031)
10.3 China Orphan Drugs Revenue Share by Type (2020-2031)
10.4 China Orphan Drugs Revenue by Application (2020-2031)
10.4.1 China Orphan Drugs Revenue by Application (2020-2025)
10.4.2 China Orphan Drugs Revenue by Application (2026-2031)
10.5 China Orphan Drugs Revenue Share by Application (2020-2031)
11 Asia (Excluding China)
11.1 Asia Orphan Drugs Revenue (2020-2031)
11.2 Asia Orphan Drugs Revenue by Type (2020-2031)
11.2.1 Asia Orphan Drugs Revenue by Type (2020-2025)
11.2.2 Asia Orphan Drugs Revenue by Type (2026-2031)
11.3 Asia Orphan Drugs Revenue Share by Type (2020-2031)
11.4 Asia Orphan Drugs Revenue by Application (2020-2031)
11.4.1 Asia Orphan Drugs Revenue by Application (2020-2025)
11.4.2 Asia Orphan Drugs Revenue by Application (2026-2031)
11.5 Asia Orphan Drugs Revenue Share by Application (2020-2031)
11.6 Asia Orphan Drugs Revenue by Country
11.6.1 Asia Orphan Drugs Revenue by Country (2020 VS 2024 VS 2031)
11.6.2 Asia Orphan Drugs Revenue by Country (2020-2025)
11.6.3 Asia Orphan Drugs Revenue by Country (2026-2031)
11.6.4 Japan
11.6.5 South Korea
11.6.6 India
11.6.7 Australia
11.6.8 Taiwan
11.6.9 Southeast Asia
12 South America, Middle East and Africa
12.1 SAMEA Orphan Drugs Revenue (2020-2031)
12.2 SAMEA Orphan Drugs Revenue by Type (2020-2031)
12.2.1 SAMEA Orphan Drugs Revenue by Type (2020-2025)
12.2.2 SAMEA Orphan Drugs Revenue by Type (2026-2031)
12.3 SAMEA Orphan Drugs Revenue Share by Type (2020-2031)
12.4 SAMEA Orphan Drugs Revenue by Application (2020-2031)
12.4.1 SAMEA Orphan Drugs Revenue by Application (2020-2025)
12.4.2 SAMEA Orphan Drugs Revenue by Application (2026-2031)
12.5 SAMEA Orphan Drugs Revenue Share by Application (2020-2031)
12.6 SAMEA Orphan Drugs Revenue by Country
12.6.1 SAMEA Orphan Drugs Revenue by Country (2020 VS 2024 VS 2031)
12.6.2 SAMEA Orphan Drugs Revenue by Country (2020-2025)
12.6.3 SAMEA Orphan Drugs Revenue by Country (2026-2031)
12.6.4 Brazil
12.6.5 Argentina
12.6.6 Chile
12.6.7 Colombia
12.6.8 Peru
12.6.9 Saudi Arabia
12.6.10 Israel
12.6.11 UAE
12.6.12 Turkey
12.6.13 Iran
12.6.14 Egypt
13 Concluding Insights
14 Appendix
14.1 Reasons for Doing This Study
14.2 Research Methodology
14.3 Research Process
14.4 Authors List of This Report
14.5 Data Source
14.5.1 Secondary Sources
14.5.2 Primary Sources
14.6 Disclaimer
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