Orphan Drugs Global Market Report 2024

The orphan drugs market size has grown rapidly in recent years. It will grow from $188.45 billion in 2023 to $208.04 billion in 2024 at a compound annual growth rate (CAGR) of 10.4%. The growth in the historical period can be credited to factors such as the Orphan Drug Act of 1983, increasing attention to rare diseases, drug repurposing, advancements in genetic understanding, and initiatives for research funding.

The orphan drugs market size is expected to see exponential growth in the next few years. It will grow to $481.69 billion in 2028 at a compound annual growth rate (CAGR) of 23.4%. The growth anticipated in the forecast period can be linked to developments in market access policies, initiatives for value-based healthcare, global expansion of the market, patient-centric drug development, and a focus on targeting rare cancers. Notable trends in the forecast period encompass the rise in target identification, the implementation of accelerated regulatory pathways, progress in gene therapy, expanded collaborations in research, and the adoption of precision medicine approaches.

The growth of the orphan drugs market is significantly propelled by the increasing prevalence of rare diseases. Rare diseases, affecting a small percentage of the population, necessitate treatments that often require a cold chain - a temperature-regulated supply chain not universally available in all countries. Globally, there has been a rise in the prevalence of rare diseases, exemplified by approximately 54,000 new cases of oropharyngeal or oral cancer reported annually in the USA as of January 2022. With 11,230 fatalities attributed to these cancers, the increasing incidence of rare diseases, even in young individuals, is a driving force for the orphan drugs market.

The growing utilization of orphan drugs in the realm of hematology is poised to propel the orphan drugs market forward. Hematology, a medical discipline focused on blood-related disorders, benefits from orphan drugs by offering therapeutic solutions for rare and orphan blood disorders. These drugs target specific genetic mutations and cellular processes, alleviating the need for blood transfusions, minimizing disease complications, and enhancing patients' quality of life. As of June 2021, more than 110,000 individuals are coping with blood disorders, and by 2035, the number in Australia is projected to surpass 275,000. Hence, the increasing application of orphan drugs in hematology is expected to be a key driver for market growth.

The absence of supportive government policies poses a hindrance to the orphan drugs market. The lack of specific policies for orphan drugs results in a dearth of special recognition or priority for regulatory approval. Companies manufacturing orphan drugs often face challenges in foreign markets due to insufficient government funding. In India, for instance, orphan medical products (OMPs) lack clear regulations and guidelines, depriving them of tax cuts or exemptions from customs duties. Consequently, the absence of favorable government policies acts as a constraint on 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.

Major players in the orphan drugs market are strategically focusing on product approvals, including orphan drug designation (ODD), to drive revenue growth. ODD is a regulatory status granted by health agencies, such as the U.S. Food and Drug Administration (FDA), to drugs and biologics developed for the treatment, prevention, or diagnosis of rare diseases or conditions. For instance, in October 2021, Tezepelumab received orphan drug designation (ODD) from the US FDA for treating eosinophilic esophagitis (EoE). Developed through a collaboration between AstraZeneca and Amgen, Tezepelumab is a monoclonal antibody drug undergoing priority review in the US for asthma patients.

In July 2021, AstraZeneca PLC, a UK-based company specializing in orphan drugs, acquired Alexion Pharmaceuticals Inc. for an undisclosed amount. This strategic acquisition bolstered AstraZeneca's scientific presence in immunology, solidifying its position in the research and development of drugs for individuals with rare diseases. AstraZeneca's commitment to R&D investments in various therapeutic areas, including oncology, cardiology, renal, respiratory, and immunology, was further reinforced by expanding into the realm of rare illnesses where limited authorized therapy options are available. Alexion Pharmaceuticals Inc. is a US-based biopharmaceutical company renowned for manufacturing orphan drugs.


Major companies operating in the orphan drugs market report are Pfizer Inc., Johnson & Johnson Services Inc., F Hoffmann-La Roche Ltd., 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., Shire PLC, Actelion Pharmaceuticals Ltd. North America was the largest region in the global orphan drugs market in 2023. 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.

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.

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.


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