Orphan Drugs Market by product type, and disease type - Global Industry Insights, Trends, 2025

The drugs which are developed for treatment of rare diseases and conditions are referred to as orphan drugs. The rare diseases such as Tourette syndrome, Huntington’s disease, muscular dystrophy, myoclonus, and ALS (Lou Gehrig’s disease) are some of those diseases for treatment of which adequate drugs have not been developed, and hence these diseases are treated by orphan drugs. Orphan drugs are developed by the pharmaceutical companies for public health need, and the sales generated by this drugs are very minimal when compared to normal drugs. The orphan diseases manifest in patient populations representing 6-8% of the global population. Symptoms of some orphan diseases may appear at birth or in childhood including cystic fibrosis, infantile spinal muscular atrophy, familial adenomatous polyposis (FAP), and some others.

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Developing novel indications for designated orphan drugs is expected to drive the orphan drugs market

There are around 5,000 rare diseases listed globally and 80% of those are to genetic origins. The probability of getting infected by these diseases in adults is 50%.The need for the development of orphan drugs which will treat such diseases and proactive initiatives undertaken by government and regulatory authorities are the major factors driving the orphan drugs market. For instance, the authorities of U.S. FDA for orphan drug development department are providing the incentives to healthcare and biotechnology industries in order to enhance their research and development in orphan drugs. There are few drugs that have been withdrawn from the market due to certain risk developed over prolong administration of these drugs. However, these drugs have shown good results for treatment of those diseases which in particular do not have any standard drug, owing to drive the orphan drugs market. For instance, thalidomide used as a hypnotic drug was withdrawn from market when teratogenic risk was discovered, however, this drug has shown prominent result in treating leprosy.

Developing orphan drugs that are used to treat rare diseases do not have a high profit margin for the manufacturers and prolong timeline for product development and manufacturing, uncertain and expensive, are the factors hindering the growth of orphan drugs market. However, the untapped emerging market and development of novel indications for designated orphan drugs is expected to create various opportunities in the orphan drugs industry.

The increasing prevalence of rare cancer in patients owing to oncological diseases segment to dominate in the orphan drugs market

The global orphan drugs market is segmented on the basis of disease, by product type and by region

On the basis of disease type,

Hematologic and Immunologic diseases

• Infectious diseases
• Metabolic diseases
• Neurologic diseases
• Oncologic diseases
• Other orphan diseases

On the basis of product type,

• Biological orphan drugs
• Non-biological orphan drugs

Numerous collaborative research programs across North America and Europe is owing to growth of orphan drugs market

Regional segmentation of orphan drugs market by Coherent Market Insights comprises of North America, Latin America, Europe, Asia Pacific, Middle East, and Africa. North America dominates the market globally due to factors such as established regulatory and reimbursement framework and incentives given by U.S. Food and Drug Administration (FDA) for research and development in orphan drugs sector. The various collaborative research programs across United States and Europe provides research data on rare diseases to manufacturers, patients and health experts which gives timely medical care, thus boost the orphan drug market. Whereas the prevalence of genetic diseases and growth of population are some factors which might allow the orphan drugs manufacturers to invest in the Asia Pacific region.

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Initiatives taken by government to increase the investment in orphan drugs development driving the growth of orphan drugs market.

Key players operating the orphan drugs market are Acorda Therapeutics, Actelion Pharmaceuticals Ltd., Amgen, Alexion Pharmaceuticals, Baxter international, Bayer healthcare pharmaceuticals, Biomarin Pharmaceuticals, Novartis, F.Hoffmann-La Roche AG, Johnson and Johnson and many others. The increase in investments, and some initiatives taken by government helping the orphan drug manufacturers. For instance, U.S. government provides 50% of tax credit on the research and development expenses spent on the orphan drugs development by manufacturers. However the high cost of the orphan drugs and generation of small sales from it is amongst those factors which is lowering the interest of companies for market.

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