Orphan Drugs Market in the US – Size, Forecast, and Competitive Landscape: Explore Everfront Biotech’s US Potential

Market Outlook: Orphan Drugs Market in the US 2025-2029

The orphan drugs market in the US is a rapidly growing sector driven by the increasing demand for treatments for rare diseases, supported by regulatory incentives such as market exclusivity and tax breaks, while also facing challenges related to high development costs and pricing. The market is expected to grow at a CAGR of 5%-7% between 2025 and 2029. Exact values for this market can be accessed upon purchasing the report. Key companies in the US orphan drugs market include:

• Sanofi
• Amgen
• Takeda Pharmaceutical Company
• AstraZeneca
• Others

IPO Company Overview: Everfront Biotech in the US orphan drugs market

Everfront Biotech Holding Co. Ltd. has filed for an IPO with the US Securities and Exchange Commission in January 2025 under the ticker symbol EFB, creating a new opportunity for investors. Everfront Biotech is a biopharmaceutical company focused on the development of breakthrough treatments for rare and orphan diseases. Leveraging cutting-edge research and advanced biotechnology, Everfront Biotech aims to address unmet medical needs in underserved patient populations by advancing therapies that target the root causes of rare genetic disorders.

Market Segmentation: How is the US orphan drugs market segmented?

Drug Type

• Small Molecule Drugs
• Biologic Drugs
• Gene Therapy Drugs

Application

• Oncology
• Neurology
• Hematology
• Genetic Disorders
• Endocrinology

Biologic drugs are the leading drug type, driven by their effectiveness in treating complex rare diseases such as genetic disorders and certain cancers. They include monoclonal antibodies and recombinant proteins, which have shown significant therapeutic outcomes and are increasingly in demand for orphan diseases. Oncology is the leading therapeutic segment, as many orphan drugs are developed for rare cancers. The high unmet medical need, coupled with advances in cancer research and targeted therapies, has driven the growth of orphan oncology drugs, with a focus on conditions like rare blood cancers and sarcomas.

Investment Opportunities: Why explore the US orphan drugs market with Technavio?

Recent developments in the US orphan drugs market reflect a growing emphasis on innovation and regulatory support for treatments targeting rare and underserved diseases. The US Food and Drug Administration (FDA) continues to streamline approval processes for orphan drugs through initiatives such as the Orphan Drug Act, which provides incentives including market exclusivity, tax credits, and research grants to foster the development of treatments for rare diseases.

Over the past few years, the number of orphan drugs approved by the FDA has surged, with significant progress in gene therapies and biologics for rare genetic disorders, neuromuscular diseases, and rare cancers. Notably, advancements in gene editing and RNA-based therapies have paved the way for the development of personalized medicine, offering new hope for patients with conditions previously deemed untreatable.

Market Dynamics: What factors are influencing the orphan drugs market in the US?

Drivers

The orphan drugs market in the U.S. is driven by increasing awareness of rare diseases, technological advancements in biotechnology, and favorable regulatory frameworks. The Orphan Drug Act has been a significant catalyst, offering incentives such as market exclusivity, tax breaks, and grants to encourage the development of treatments for rare conditions. As advancements in gene therapies, genetic testing, and personalized medicine evolve, they have enabled the development of highly targeted treatments for rare diseases that were previously underserved. The growing understanding of genetic conditions and the rising demand for more specialized care also contribute to the expansion of the orphan drug market.

Trends

One of the most notable trends in the orphan drugs market is the increasing use of gene therapies and RNA-based treatments, which aim to address the root causes of genetic disorders at the molecular level. This includes advancements in CRISPR gene editing and therapies like RNA interference (RNAi) for diseases such as muscular dystrophy and spinal muscular atrophy. Additionally, biologic therapies such as monoclonal antibodies are gaining prominence in the treatment of rare cancers and autoimmune disorders. There is also a rise in collaborations between pharmaceutical companies, biotechnology firms, and academic institutions, accelerating the development of orphan drug candidates and bringing innovative therapies to market faster.

Opportunities

The orphan drugs market presents significant opportunities for growth, particularly as unmet medical needs remain high for patients with rare and complex diseases. Strategic partnerships between biotech and pharmaceutical companies allow for the sharing of resources, expertise, and technology, speeding up the discovery and development of new treatments. Additionally, the increased focus on genetic diseases, particularly those involving rare inherited conditions, opens up avenues for gene therapies and RNA-based treatments. Another promising opportunity is in the realm of diagnostic tools, where advancements in genetic testing and early disease detection can lead to more targeted and timely treatments, improving patient outcomes and creating new markets for orphan drugs.

Challenges

Despite its growth, the orphan drugs market faces several challenges, particularly related to the high cost of drug development and the complexity of delivering therapies to small, geographically dispersed patient populations. Developing treatments for rare diseases often involves small clinical trials, which can make it difficult to generate sufficient data for approval. Additionally, pricing pressures have been a major issue, as the high cost of orphan drugs can limit access for patients and invite scrutiny from healthcare payers and regulators. Furthermore, the regulatory hurdles in securing approval for gene therapies and biologics, as well as ensuring long-term safety and efficacy, pose challenges for companies in the orphan drugs space. Despite these challenges, the ongoing demand for novel treatments and supportive policy frameworks provide a promising outlook for the market.

Investment Questions: Topics to consider when investing in the orphan drugs market in the US

What is the company's financial stability and funding strategy?
Consider how financially secure the company is. Does it have sufficient capital to fund ongoing research, clinical trials, and commercialization efforts, or is it dependent on external funding or partnerships?

How is the company addressing potential competition?
It is important for the company to have a plan to differentiate its drug or therapy from competitors, especially if there are existing or pipeline treatments with similar mechanisms of action.

What is the company's approach to manufacturing and distribution?
Scaling manufacturing of an orphan drug can be challenging, especially for a small patient population. It is also important to consider how to ensure reliable distribution to patients, particularly those in remote or underserved areas.

What is the risk of regulatory or pricing changes?
Potential changes in healthcare policy, pricing regulations, or reimbursement policies for orphan drugs can all impact a company's operations and bottom line. Public scrutiny and political pressure on high-priced medications can also pose risks.

For a detailed analysis of the orphan drugs market in the US, buy the full report

Technavio Report Insights: How Can Technavio Reports Help Investors Make Better Business Decisions?

Key Features of a Technavio Report: 

• Comprehensive Market Analysis: Provides a detailed overview of the orphan drugs market in the US, including market size, growth rate, segmentation, and regional analysis. 

• In-Depth

  Competitive Landscape:

  Offers insights into the competitive dynamics, market share and positioning, and product offerings of key players. 

• Future Market Trends: Identifies emerging trends and opportunities that can shape the future of the market. 

• Detailed Segmentation: Breaks down the market into various segments to help investors identify specific niches and target markets. 

• Expert Analysis: Provides expert insights and forecasts from industry analysts

FAQ

1. What is the Orphan Drug Act, and how does it support drug development?
The Orphan Drug Act of 1983 provides incentives to encourage the development of treatments for rare diseases, including seven years of market exclusivity, tax credits for clinical research, and grants for research and development. This legislation aims to make it financially viable for companies to invest in the development of orphan drugs for small patient populations.

2. How large is the orphan drug market in the US?
The US orphan drug market has grown significantly in recent years, driven by advances in biotechnology and an increasing number of orphan drug approvals. It is expected to continue expanding, with revenues forecasted to grow substantially due to an increase in the number of rare diseases being targeted, along with higher demand for specialized treatments.

3. What role do biotechnology companies play in the orphan drugs market?
Biotechnology companies are key players in the orphan drugs market, often focusing on innovative treatments such as gene therapies, cell therapies, and biologic drugs. These companies leverage cutting-edge research and technology to develop treatments for rare diseases and often collaborate with larger pharmaceutical companies to bring these drugs to market.

4. What is the future outlook for the orphan drugs market?
The future of the orphan drugs market looks promising, with continued growth expected due to advancements in biotechnology, gene therapies, and personalized medicine. As the understanding of rare diseases improves and treatment options expand, more companies are likely to invest in orphan drug development. However, pricing and reimbursement challenges, along with regulatory complexities, will continue to shape the market's landscape.

Table of Contents

1 Executive Summary

• 1.1 Market overview
  • Executive Summary - Chart on Market Overview
  • Executive Summary - Data Table on Market Overview
  • Executive Summary - Chart on Global Market Characteristics
  • Executive Summary - Chart on Market by Geography
  • Executive Summary - Chart on Market Segmentation
  • Executive Summary - Chart on Incremental Growth
  • Executive Summary - Data Table on Incremental Growth
  • Executive Summary - Chart on Company Market Positioning

2 Technavio Analysis

• 2.1 Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria
  • Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria

• 2.2 Criticality of inputs and factors of differentiation
  • Overview on criticality of inputs and factors of differentiation

• 2.3 Factors of disruption
  • Overview on factors of disruption

• 2.4 Impact of drivers and challenges
  • Impact of drivers and challenges in 2024 and 2029

3 Market Landscape

• 3.1 Market ecosystem
  • Parent Market
  • Data Table on - Parent Market

• 3.2 Market characteristics
  • Market characteristics analysis

• 3.3 Value chain analysis
  • Value chain analysis

4 Market Sizing

• 4.1 Market definition
  • Offerings of companies included in the market definition

• 4.2 Market segment analysis
  • Market segments

• 4.3 Market size 2024

• 4.4 Market outlook: Forecast for 2024-2029
  • Chart on Global Market - Market size and forecast 2024-2029 ($ billion)
  • Data Table on Global Market - Market size and forecast 2024-2029 ($ billion)
  • Chart on Global Market: Year-over-year growth 2024-2029 (%)
  • Data Table on Global Market: Year-over-year growth 2024-2029 (%)

5 Historic Market Size

• 5.1 Global Market 2019 - 2023
  • Historic Market Size - Data Table on Global Market 2019 - 2023 ($ billion)

• 5.2 Segment Analysis 2019 - 2023
  • Historic Market Size – Segment Analysis 2019 - 2023 ($ billion)

• 5.4 Geography segment analysis 2019 - 2023
  • Historic Market Size - Geography Segment 2019 - 2023 ($ billion)

6 Qualitative Analysis

• 6.1 Impact of AI on the Global Market

7 Five Forces Analysis

• 7.1 Five forces summary
  • Five forces analysis - Comparison between 2024 and 2029

• 7.2 Bargaining power of buyers
  • Bargaining power of buyers - Impact of key factors 2024 and 2029

• 7.3 Bargaining power of suppliers
  • Bargaining power of suppliers - Impact of key factors in 2024 and 2029

• 7.4 Threat of new entrants
  • Threat of new entrants - Impact of key factors in 2024 and 2029

• 7.5 Threat of substitutes
  • Threat of substitutes - Impact of key factors in 2024 and 2029

• 7.6 Threat of rivalry
  • Threat of rivalry - Impact of key factors in 2024 and 2029

• 7.7 Market condition
  • Chart on market condition - Five forces 2024 and 2029

8 Market Segmentation

• 8.1 Market segments
  • Chart on Segmentation - Market share 2024-2029 (%)
  • Data Table on Segmentation - Market share 2024-2029 (%)

• 8.2 Segment Comparison
  • Chart on Comparison
  • Data Table on Comparison

• 8.3 Segment Market Opportunity
  • Market opportunity ($ billion)
  • Data Table on Market Opportunity ($ billion)

9 Customer Landscape

• 9.1 Customer landscape overview
  • Analysis of price sensitivity, lifecycle, customer purchase basket, adoption rates, and purchase criteria

10 Geographic Landscape

• 10.1 Geographic segmentation
  • Chart on market share by geography 2024-2029 (%)
  • Data Table on market share by geography 2024-2029 (%)

• 10.2 Geographic comparison
  • Chart on Geographic comparison
  • Data Table on Geographic comparison

• 10.3 Market opportunity by geography
  • Market opportunity by geography ($ billion)
  • Data Tables on Market opportunity by geography ($ billion)

11 Drivers, Challenges, and Opportunity/Restraints

• 11.1 Market drivers

• 11.2 Market challenges

• 11.3 Impact of drivers and challenges
  • Impact of drivers and challenges in 2024 and 2029

• 11.4 Market opportunities/restraints

12 Competitive Landscape

• 12.1 Overview

• 12.2 Competitive Landscape
  • Overview on criticality of inputs and factors of differentiation

• 12.3 Landscape disruption
  • Overview on factors of disruption

• 12.4 Industry risks
  • Impact of key risks on business

13 Competitive Analysis

• 13.1 Companies profiled
  • Companies covered

• 13.2 Company ranking index
  • Company ranking index

• 13.3 Market positioning of companies
  • Matrix on companies position and classification

14 Appendix

• 14.1 Scope of the report

• 14.2 Inclusions and exclusions checklist
  • Inclusions checklist
  • Exclusions checklist

• 14.3 Currency conversion rates for US$
  • Currency conversion rates for US$

• 14.4 Research methodology
  • Research methodology

• 14.5 Data procurement
  • Information sources

• 14.6 Data validation
  • Data validation

• 14.7 Validation techniques employed for market sizing
  • Validation techniques employed for market sizing

• 14.8 Data synthesis
  • Data synthesis

• 14.9 360 degree market analysis
  • 360 degree market analysis

• 14.10 List of abbreviations
  • List of abbreviations