Published: 2023 May 16

Orphan Drugs Market Trends, Demand, Growth, Size, Competitive Landscape and Market Forecast 2023-2030

SKU : PH667
180 pages
Report Summary
Table of Contents
List of Tables & Figures

Orphan Drugs Market is segmented by Product Type (Biological, Non-Biological), by Therapy Area (Oncology, Hematology Diseases, Central Nervous System, Cardiovascular, Endocrine, Respiratory, Immunomodulators, Gastro-Intestinal, Musculoskeletal, Systemic Anti-Infectives, Dermatology, Others), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2023-2030


Orphan Drugs Market Overview

The Global Orphan Drugs Market is estimated to reach at a CAGR of 11.2% during the forecast period (2023-2030). Life-threatening or persistently disabling diseases are treated, prevented from occurring, or diagnosed with an orphan drug. It is mostly used to treat rare disorders. Less than 200,000 Americans and five out of every 10,000 people in the European Union are affected by this rare sickness or condition.


Orphan Drugs Market Summary and Scope



Market CAGR


Segments Covered

By Product Type, By Therapy Area, By Distribution Channel, and By Region

Report Insights Covered

Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights.

Fastest Growing Region

Asia Pacific

Largest Market Share 

North America


Orphan Drugs Market Dynamics and Trends

The rise in research and development activities, rising adoption of orphan drugs for the treatment of diseases, and an increase in the geriatric population will drive the orphan drugs market growth.

The increasing awareness of rare diseases is expected to drive the market growth   

The rising awareness of rare diseases and treatment for them is expected to boost the market over the forecast period. As per the article published in Perspective in Clinical Research 2022, regulators like the FDA have recently developed an online platform called the "FDA Rare Disease Photo and Video Project" to record and share staff experiences and real-time patient accounts. By setting up various meetings with patients, caregivers, and FDA employees, the office of patient affairs supports patient-centricity with this goal. Several nonprofit organizations, such as the National Organization for Rare Disorders and the European Organisation for Rare Diseases, support regulatory and research activities in Europe and the United States, respectively, and promote patient advocacy through patient assistance, information sharing, and networking between various patient groups.

Furthermore, per the article published in Institute for Clinical and Economic Review, cell and gene therapies being developed as of 2020 are for rare diseases, and it is predicted that more than 70% of rare diseases are genetic. As of the middle of 2020, the FDA had authorized 599 orphan products since the Orphan Drug Act was passed. The percentage of first-in-class orphan pharmaceuticals is substantially higher than that of nonorphan drugs, with 50% of all orphan drugs authorized in the U.S. Despite the pandemic, overall clinical trial activity has continued because the industry has adjusted to the disruption and created new strategies to support ongoing research. Five thousand five hundred new planned clinical trial launches were reported in 2021, up 14% from 2020 and 19% from 2019.

The difficulty of gathering sufficient efficacy and safety data will hamper the growth of the market

However, the difficulty of gathering sufficient efficacy and safety data in small groups, the risk of financial support for both developers and healthcare systems, and the limited understanding of most diseases are barriers to developing medical treatments or cures for rare diseases. Governments' actions alone are insufficient to narrow the market gap that arises from each rare disease's generally low commercial potential. Additionally, deciding on a working definition of what defines a "rare" as opposed to an "ultra-rare" illness would be one of the major obstacles in building a policy platform to stimulate the development of ultra-rare drugs. These elements are limiting further market expansion.

COVID-19 Impact Analysis on Orphan Drugs Market

The appearance of COVID-19 considerably impacted the global orphan drugs market. As per the article published in Frontiers in Public Health 2021, there are about five to eight thousand rare diseases, particularly with a genetic basis, affecting approximately 400 million people worldwide. People with rare diseases and their caretakers report significant care insufficiencies and unmet clinical needs. The difficulty and expense of assembling large compatriots of affected individuals for study and conducting research funding is already a problem for researchers. Together with the general anxieties about health concerns everyone else has, people with rare diseases have a double burden of the question due to COVID-19. They also face a supply of medications and the accessibility of essential occupational therapies they need regularly.

On the other hand, with the prevailing COVID-19 pandemic in 2020, the FDA approved 32 orphan-designated drugs and biological products to significantly sustain progress in orphan drug research and development. The Center for Drug Evaluation and Research office created a rare disease hub to assess marketing applications for certain rare diseases. It collaborated with a rare disease team to receive support and assistance for enabling a rare disease policy. FDA launched the orphan drug modernization pilot program to keep pace with the increase in application in subsequent years. The FDA is to review all applications within a stipulated time of 90 days, irrespective of the volume and complexity of the application.

Orphan Drugs Market Segmentation Analysis

The oncology segment is expected to grow at the fastest CAGR during the forecast period (2023-2030)    

The oncology segment is expected to boost the market throughout the forecast. In July 2022, The FDA granted orphan drug designation to several therapies in development for oncology indications. MB-106 (Mustang Bio) is a CD20-targeted, autologous chimeric antigen receptor T-cell therapy. The orphan drug designation applies to the use of the agent for the treatment of Waldenstrom macroglobulinemia. Paxalisib (Kazia Therapeutics Limited) is a phosphatidylinositol 3-kinase inhibitor. The designation applies to the agent's treatment of atypical rhabdoid/teratoid tumors, an aggressive and rare childhood brain cancer. VBI-1901 (VBI Vaccines) is a bivalent gB/pp65 immunotherapeutic vaccine candidate. The designation applies to the use of this agent for the treatment of glioblastoma.

In addition, in January 2021, the U.S. Food and Medication Administration (FDA) granted PVSRIPO (stage IIB-IV) as an orphan drug for the treatment of advanced melanoma by Istari Oncology, Inc. . A patient's adaptive and innate immune systems are activated by PVSRIPO, a novel viral immunotherapy based on the Sabin type 1 polio vaccine, to promote an anti-tumor response and establish long-term immunologic memory to the prevention of cancer recurrence.

Orphan Drugs Market Geographical Share

North America region holds the largest market share of the global orphan drugs market

North America dominates the market for orphan drugs and is expected to show a similar trend over the forecast period. It is anticipated to hold a significant market size over the forecast period (2022-2029) owing to the advanced healthcare system, government initiatives and the presence of many market players. The FDA Office of Orphan Products Development approved orphan drug designation for novel drugs and biologics that are used for the safe and effective treatment, diagnosis and prevention of rare diseases or disorders that suffer fewer than 200,000 people in the United States region. The designation allows key players to qualify for various incentives, including tax credits for qualified clinical trials and, upon regulatory approval, 7 years of market exclusivity. In addition, the U.S. remains the country with the earliest and highest number of launches and among the 72 NASs launched in 2021, a record 44 (over 60%) were characterized by the FDA as first-in-class, and more than half (40) carried an orphan drug designation indicating their use for patients with rare diseases.

Moreover, in September 2020, AbbVie's experimental therapy for patients with spinal cord injury, elezanumab (ABT-555), was given Orphan drugs and Fast Track designations by the USFDA. A monoclonal antibody of the human immunoglobulin (Ig)G1 isotype called elezanumab binds only to the repellent guidance molecule A (RGMa).

Orphan Drugs Companies and Competitive Landscape

The orphan drugs market is a moderately competitive presence of local and global companies. Some of the key players which are contributing to the growth of the market are AbbVie Inc., Alexion Pharmaceuticals, Inc., Bristol-Myers Squibb Company, Celgene Corporation, Johnson & Johnson, Merck & Co., Inc., Novartis AG, Roche Holding AG, Sanofi SA, and Shire plc among others. The major players are adopting several growth strategies, such as product launches, acquisitions, and collaborations, contributing to the expansion of the orphan drugs market globally.

For instance,

  • In June 2020, Chiasma, Inc. announced that the U.S. Food and Drug Administration (FDA) approved MYCAPSSA (octreotide) capsules for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

AbbVie Inc


AbbVie is a research-based biopharmaceutical organization founded in 2013 and headquartered in the United States.  It is developing innovative and advanced therapies to meet the requirement of complex and serious diseases.

Product Portfolio:

HUMIRA (adalimumab): It is used to reduce symptoms of Moderate to severe rheumatoid arthritis (RA) in adults, Psoriatic arthritis (PsA) in adults, and ulcerative colitis in adults.

The global orphan drugs market report would provide access to approximately 65+ market data tables, 65+ figures, and in the range of 200+ (approximate) pages.​​​​​​​

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What is the Projected CAGR value of the Orphan Drugs Market?
Orphan Drugs Market is expected to grow at a high CAGR during the forecasting period 2023-2030.
Which region controlled the global market during 2023-2030?
North America region Controls the Orphan Drugs Market during 2023-2030.
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