Global Orphan Drugs Market Growth (Status and Outlook) 2025-2031

Global Orphan Drugs Market Growth (Status and Outlook) 2025-2031

The global Orphan Drugs market size is predicted to grow from US$ 163090 million in 2025 to US$ 292550 million in 2031; it is expected to grow at a CAGR of 10.2% from 2025 to 2031.

An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.

Bristol-Myers Squibb is one of the most important global key players of orphan drugs, holds a share of about 10%, other key players include Roche and Novartis, etc. North America is the largest market, occupied for about 40 percent, followed by Asia-Pacific. In terms of type, oncology is the largest segment, with a share of over 60%, and in terms of application, the adult segment holds a share of about 40 percent.

LPI (LP Information)' newest research report, the “Orphan Drugs Industry Forecast” looks at past sales and reviews total world Orphan Drugs sales in 2024, providing a comprehensive analysis by region and market sector of projected Orphan Drugs sales for 2025 through 2031. With Orphan Drugs sales broken down by region, market sector and sub-sector, this report provides a detailed analysis in US$ millions of the world Orphan Drugs industry.

This Insight Report provides a comprehensive analysis of the global Orphan Drugs landscape and highlights key trends related to product segmentation, company formation, revenue, and market share, latest development, and M&A activity. This report also analyses the strategies of leading global companies with a focus on Orphan Drugs portfolios and capabilities, market entry strategies, market positions, and geographic footprints, to better understand these firms’ unique position in an accelerating global Orphan Drugs market.

This Insight Report evaluates the key market trends, drivers, and affecting factors shaping the global outlook for Orphan Drugs and breaks down the forecast by Type, by Application, geography, and market size to highlight emerging pockets of opportunity. With a transparent methodology based on hundreds of bottom-up qualitative and quantitative market inputs, this study forecast offers a highly nuanced view of the current state and future trajectory in the global Orphan Drugs.

This report presents a comprehensive overview, market shares, and growth opportunities of Orphan Drugs market by product type, application, key players and key regions and countries.

Segmentation by Type:
Oncology
Pulmonary
Neurology
Hematology
Endocrinology
Cardio-vascular
Metabolic Disorders
Others

Segmentation by Application:
Baby and Child
Teenager
Adult
Other

This report also splits the market by region:
Americas
United States
Canada
Mexico
Brazil
APAC
China
Japan
Korea
Southeast Asia
India
Australia
Europe
Germany
France
UK
Italy
Russia
Middle East & Africa
Egypt
South Africa
Israel
Turkey
GCC Countries

The below companies that are profiled have been selected based on inputs gathered from primary experts and analyzing the company's coverage, product portfolio, its market penetration.
Bristol-Myers Squibb
Roche
Novartis
Johnson & Johnson
Pfizer
Amgen
Sanofi
AstraZeneca
Takeda
Vertex Pharmaceuticals
AbbVie
Biogen
Eli Lilly

Please note: The report will take approximately 2 business days to prepare and deliver.