Orphan Drugs Market Report 2025

This Orphan Drugs market report provides a comprehensive analysis of the market’s characteristics, size, and growth, including segmentation, regional and country-level breakdowns, competitive landscape, market shares, trends, and strategies. It also tracks historical and forecasted market growth across various geographies.

The orphan drugs market size has grown rapidly in recent years. It will grow from $188.45 billion in 2024 to $208.04 billion in 2025 at a compound annual growth rate (CAGR) of 10.4%. The growth in the historic period can be attributed to orphan drug act of 1983, rising interest in rare diseases, drug repurposing, advancements in genetic understanding, research funding initiatives.

The orphan drugs market size is expected to see exponential growth in the next few years. It will grow to $481.69 billion in 2029 at a compound annual growth rate (CAGR) of 23.4%. The growth in the forecast period can be attributed to evolving market access policies, value-based healthcare initiatives, global market expansion, patient-centric drug development, targeting rare cancers. Major trends in the forecast period include increasing target identification, accelerated regulatory pathways, advancements in gene therapy, expanded research collaborations, precision medicine approaches.

The growing prevalence of rare diseases is a significant factor propelling the growth of the orphan drugs market. A rare disease is defined as one that affects a small percentage of the population. Treatments for rare diseases often necessitate a cold chain - a temperature-regulated supply chain that is not universally accessible in all countries. Orphan diseases occur infrequently among people (approximately 7 out of 10,000). However, the global prevalence of rare diseases has been on the rise in recent years. For instance, in January 2022, an article from the American Cancer Society indicated that there are around 54,000 new cases of oropharyngeal or oral cancer each year in the USA, with 11,230 fatalities attributed to these cancers. The average age of diagnosis is 63, although these cancers can also affect younger individuals, with just over 20% (1 in 5) of cases occurring in patients under 55. Consequently, the increasing prevalence of rare diseases is driving the growth of the orphan drugs market.

The rising application of orphan drugs in hematology is anticipated to further drive the growth of the orphan drugs market. Hematology is a medical science branch focusing on the study, diagnosis, treatment, and management of disorders related to blood and blood-forming tissues. Orphan drugs offer therapeutic options for rare and orphan blood disorders by targeting specific genetic mutations and cellular processes, reducing the need for blood transfusions, mitigating disease complications, and enhancing the quality of life. For example, in December 2023, the Office of the Assistant Secretary for Planning and Evaluation, a US government department, reported that in 2022, the average cost of a biological product was $174 per prescription, which is 3.7 times higher than the $48 for small-molecule drugs. Notably, 43% of the 1,838 small-molecule drugs had only one manufacturer, accounting for 65% of total expenditures. Therefore, the increasing application of orphan drugs in hematology is expected to drive the growth of the orphan drugs market.

The approval of biological orphan drugs for multiple indications emerges as a significant trend propelling the growth of the orphan drugs market. Biological drugs, utilized in the treatment of rare diseases such as cancer, exhibit fewer side effects and are prevalent in the developed world where these diseases have a high incidence rate. An illustration of this trend is observed in the engagement of biological orphan drug expert Frederick D. Sancilio, Ph.D., by Cardax, Inc., to spearhead the company's biological orphan drug development program. Companies are strategically acquiring biological orphan drugs to enhance their commercial product portfolio and increase revenue.

Leading companies in the orphan drugs market are concentrating on obtaining product approvals for therapies like CAR T-cell therapy to address the urgent needs associated with rare diseases. This strategy not only improves patient outcomes but also takes advantage of regulatory incentives for innovative treatments. CAR T-cell therapy is a personalized form of immunotherapy that involves modifying a patient’s T cells to specifically target and attack cancer cells, demonstrating significant efficacy in treating certain blood cancers. For example, in March 2024, Cabaletta Bio, a US-based biotechnology firm, received FDA-granted orphan drug designation for CABA-201. CABA-201 is an investigational CAR T-cell therapy aimed at CD19-positive B-cells, intended to induce durable remission in autoimmune diseases such as systemic sclerosis. Its potential advantages include a targeted treatment approach, a favorable safety profile, and the possibility of long-lasting effects from a single infusion.

In May 2024, Sanofi S.A., a France-based pharmaceutical company that develops, discovers, and delivers medicines and vaccines, acquired Inhibrx Inc. for $1.7 billion. Through this acquisition, Sanofi aims to strengthen its rare disease pipeline by focusing on alpha-1 antitrypsin deficiency and addressing the unmet medical needs of this underserved patient population. Inhibrx is a US-based biotechnology firm dedicated to developing novel therapies, including orphan drugs, to meet the unmet medical needs associated with rare diseases.

Orphan drugs are medications specifically formulated to address uncommon medical conditions. These drugs are designed to meet public health needs that are often neglected by the pharmaceutical industry due to financial constraints. The term ""orphan"" in this context refers to medications that may have been developed for a common condition but are also effective in treating a less prevalent medical issue.

Orphan drugs come in two main types biological and non-biological. They are employed in various therapeutic areas, including oncology, hematology, neurology, cardiovascular, respiratory, and immunomodulatory applications. Hematology specifically focuses on the study of blood and blood-related disorders. The distribution of these drugs is carried out through hospital pharmacies, retail pharmacies, and online sales.

The orphan drugs market research report is one of a series of new reports that provides orphan drugs market statistics, including orphan drugs industry global market size, regional shares, competitors with an orphan drugs market share, detailed orphan drugs market segments, market trends and opportunities, and any further data you may need to thrive in the orphan drugs industry. This orphan drugs market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

Major companies operating in the orphan drugs market include Pfizer Inc., Johnson & Johnson Services Inc., F Hoffmann-La Roche Ltd., Farbenfabriken Bayer AG, Novartis AG, Sanofi SA, Bristol-Myers Squibb Co., AstraZeneca plc, GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, Eli Lilly and Company, Gilead Sciences Inc., Applied Molecular Genetics Inc., Novo Nordisk A/S, Merck & Co. KG, AbbVie Inc., Teva Pharmaceutical Industries Ltd., Regeneron Pharmaceuticals Inc., CSL Limited, Biogen Inc., Celgene Corporation, Vertex Pharmaceuticals Inc., Eisai Co. Ltd., Grifols SA, Alexion Pharmaceuticals Inc., BioMarin Pharmaceutical Inc., Aegerion Pharmaceuticals Inc., Sankyo Co. Ltd.

North America was the largest region in the global orphan drugs market in 2024. Middle East is expected to be the fastest growing region in the orphan drugs market report during the forecast period. The regions covered in the orphan drugs market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the orphan drugs market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The orphan drugs market consists of sales of ivacaftor and alglucerase. Values in this market are factory gate values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

This product will be delivered within 3-5 business days.