What is the current size of the global orphan drugs market and how fast is it growing?

The market is valued at USD 230.91 billion in 2025 and is projected to reach USD 312.53 billion by 2030, reflecting a 6.24% CAGR. Read More

Which drug type holds the largest share of the orphan drugs market?

Biologics lead with 65.65% market share in 2024 and are expanding at an 8.84% CAGR through 2030. Read More

Which region is expected to grow the fastest in the orphan drugs market?

Asia-Pacific is forecast to post the strongest 11.62% CAGR between 2025 and 2030. Read More

What is the primary growth driver for the orphan drugs market?

Breakthrough gene- and cell-therapy platforms targeting ultra-rare disorders are adding about 2.1 percentage points to the market’s CAGR. Read More

What is the most significant restraint affecting market expansion?

High per-patient treatment costs—often exceeding USD 300,000 per year or USD 2 million for one-time gene therapies—are exerting downward pressure on adoption. Read More

How is artificial intelligence influencing orphan drug development?

AI-enabled adaptive clinical trials are shortening development timelines by 18–24 months, helping sponsors reach the market sooner while maintaining robust evidence standards. Read More

Orphan Drugs Companies

This report lists the top Orphan Drugs companies based on the 2023 & 2024 market share reports. Mordor Intelligence expert advisors conducted extensive research and identified these brands to be the leaders in the Orphan Drugs industry.

Global Orphan Drugs Market

Access Report

About This Report
- Market Size & Share
- Market Analysis
- Trends and Insights
- Segment Analysis
- Geography Analysis
- Competitive Landscape
- Major Players
- Recent Developments
Table of Contents
SCOPE OF THE REPORT
Market Definition