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Orphan Drugs Market Trends

ID: MRFR//1697-CR | 159 Pages | Author: Kinjoll Dey| March 2019

Orphan Drugs Market Overview


Orphan drugs market size was valued at USD 196.60 billion in 2023 and is projected to grow from USD 219.54 Billion in 2024 to USD 480.27 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 9.08% during the forecast period (2024-2032). The rising prevalence of rare diseases, growing investment in research and development of orphan drugs, and increasing awareness regarding rare disorders are driving the market growth.


Orphan Drugs Market


Orphan Drugs Market Trends



  • The growing investment in research and development of orphan drugs to boost the market growth.


The growing R&D investment efforts made by major companies for the orphan drug creation of fresh product offerings is one of the key driving factors influencing the worldwide market. A number of significant clinical-stage biopharmaceutical businesses and well-established market competitors have excellent pipeline candidates for orphan pharmaceuticals in various phases of clinical trials as a result of the growing awareness and knowledge of rare diseases. For instance, the pharmaceutical industry invested 238 billion US dollars worldwide in research and development in 2021. In contrast, R&D spending reached a total of USD 137 billion in 2012.


Furthermore, the rising interest in treating rare problems is due to the potential that significant pharmacological developments may result in the production of blockbuster medications in the field of treating uncommon ailments as opposed to typical pharmaceutical portfolios.

Orphan Drugs Market Segment Insights


Orphan Drugs Drug Type Insights


Orphan Drugs Drug Type Insightsorphan drugs market segmentation, based on drug type, includes biologics and non-biologics. The biologics segment is to hold the majority share in 2021 in the orphan drugs market revenue. This is due to the rising incidence of various rare diseases curable with biological drugs and growing investment in developing drugs for rare diseases by private companies. Furthermore, major market companies are increasing the number of biological products they are supplying, which is causing them to dominate the biological orphan medication industry.


December 2018: Merck KGaA(Germany), Darmstadt, Announces FDA orphan drug designation for Bifunctional Immunotherapy M7824 in biliary tract cancer.


Orphan Drugs Sale Insights


The orphan drugs market segmentation is based on a sale that includes, generics and prescribed. The generics segment has dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2022-2030. This is due to the low cost of generics as an alternative to branded drugs and a large number of patents expired branded drugs. In addition, Generic firms are having a harder time introducing new treatments as it gets more difficult to develop, produce, and advertise original pharmaceuticals that have lost their patent protection. Original patents worth approximately USD 217 billion that cover a wide range of products, including biologics, oral solids, injectables, inhalers, and over the counter (OTC) drugs, are about to expire. Therefore, approval is necessary for generic medications due to strict regulatory rules, which is anticipated to impede segment expansion.


ORPHAN DRUGS MARKET, BY SALE, 2021 & 2030 (USD BILLION)ORPHAN DRUGS MARKET, BY SALE, 2021 & 2030


Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review


Orphan Drugs Drug Insights


The orphan drugs market data has been bifurcated into revlimid, rituxan, opdivo, keytruda, imbruvica, soliris, jakaf, pomalyst, darzalex, and spinraza. The opdivo segment dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2024-2032. The growth of the opdivo segment in the market is mainly due to the drug works by helping the body's own immune system eliminate cancer cells. Unlike chemotherapy, Opdivo specifically targets cancer cells and often has fewer side effects than traditional chemotherapy drugs.


Orphan Drugs Therapy Class Insights


The orphan drugs market segmentation is based on the therapy class that includes Oncology, blood, central nervous systems, endocrine, cardiovascular, and respiratory. The contract research organizations segment has dominated the market in 2021 and is projected to be the faster-growing segment during the forecast period, 2022-2030. This is due to the emergence of new cancer-related diseases and increasing demand for the treatment of oncology disorders worldwide. For instance, In June 2022, ALX Oncology Holdings Inc.(US) received U.S. FDA grant as orphan drug designation (ODD) to Evorpacept, for the treatment of patients with Acute Myeloid Leukemia. As a result, the oncology section of the orphan drug industry is increasingly dominated by investors who are investing huge sums of money in oncology in order to develop solutions with uncommon medicines for patients suffering from unusual cancer conditions.


August 2022:FDA Approved of Mylan's (US)APOKYN NDA; A Novel treatment for Parkinson's Disease. The FDA approval clears the way for Bertek to market APOKYN and provides a new therapy for "off" episodes, one of the most debilitating aspects suffered by patients with Parkinson's disease.


Orphan Drugs Regional Insights


By Region, the study segments the market into North America, Europe, Asia-Pacific, and the Rest of the World. The North America orphan drugs market is expected to account for USD XX billion in 2021 and is expected to exhibit an XX% CAGR during the study period. This is attributed to the North America. Therefore, favorable regulatory policies and increasing investment by pharmaceutical companies can also be attributed to this regional growth. In addition, The United States has the greatest market share in North America. The fact that a drug with orphan drug classification in the US obtains seven years of marketing exclusivity after FDA clearance for a particular indication, tax credits, and user fee waivers is one of the factors driving market expansion.


Further, the major countries studied are the U.S., Canada, Germany, France, the UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil.


Figure 3: ORPHAN DRUGS MARKET SHARE BY REGION 2021 (%)ORPHAN DRUGS MARKET SHARE BY REGION 2021


Source: Secondary Research, Primary Research, MRFR Database, and Analyst Review


Europe orphan drugs market is expected to account for the second-largest market share due to the increase as a result of numerous pharmaceutical businesses exporting their goods outside of or inside Europe. Further, the Germany orphan drugs market is expected to hold third place for the market share, and the UK orphan drugs market is expected to fastest-growing market, and which is in the 4th place in the European region. Furthermore, France orphan drugs market will hold the 5th place for the market share.


The Asia-Pacific orphan drugs market is expected to grow at a CAGR of XX% from 2022 to 2030. This is due to the due to the presence of established market players across the region and the faster development of medical infrastructure, and higher disposable incomes across the region. Moreover, China orphan drugs market is expected to hold the largest market share, and India orphan drugs market is expected fastest-growing market in the Asia-Pacific region.


For instance, India has reportedly higher rare diseases population than the world average, but initiatives from the government side are still less, and in fact, India lacks national legislation for orphan medicines and rare diseases, in spite that these are most populated countries, making it to be the world leader in the market for the orphan drugs market.


The Rest of the World includes the Middle East, Africa, and Latin America. Nevertheless, due to a lack of awareness and low income, particularly in Africa, the Middle East and Africa held the smallest proportion of the orphan drugs market.


Orphan Drugs Key Market Players & Competitive Insights


The orphan drugs market is characterized by the presence of many global, regional, and local vendors catering to the demand created by research scientists and pharmaceutical and biotechnology companies operating in the industry. The market comprises tier-1, tier-2, and local players. The tier-1 and tier-2 players have reach across the globe with diverse product portfolios. Companies such as F. Hoffmann-La Roche AG (Switzerland), Mylan (US), Celgene Corporation (US), and Novartis AG (Switzerland) dominate the orphan drugs market due to product differentiation, financial stability, strategic developments, and diversified regional presence. The players are focused on investing in research and development. Furthermore, they adopt strategic growth initiatives, such as expansion, product launches, joint ventures, and partnerships, to strengthen their market position and capture a large customer base.


One of the primary business strategies adopted by manufacturers in the Orphan Drugs industry to benefit clients and expand the orphan drugs market sector is to manufacture locally to reduce operating costs.F. Hoffmann-La Roche Ltd (Roche) is one of the leading companies in the field of diagnostics and pharmaceuticals. It develops and markets products for the diagnosis and treatment of anemia, cancer, anticoagulation therapy, cardiovascular diseases, diabetes, central nervous system, chlamydia, dermatology, gonorrhea, gout, hepatitis B and C, hemostasis disorders, HIV/AIDS, HPV, infectious diseases, and autoimmune diseases. The firm operates in two major business segments, namely, pharmaceuticals and diagnostics. The pharmaceuticals segment offers a wide range of drugs for therapeutic areas, such as oncology, inflammation, neuroscience, infectious diseases, and ophthalmology. Furthermore, the diagnostics segment offers a wide range of tests for cancer and companion diagnostic tests. The company has a strong presence in North America, Europe, Asia, and the Middle East. For instance, In December 2019, F. Hoffmann-La Roche Ltd (Switzerland) entered licensing agreement with Sarepta Therapeutics to advance the treatment for a rare disease named Duchenne Muscular Dystrophy (DMD).


Also, Novartis AG (Novartis) is a pharmaceutical company that researches, develops, manufactures, and distributes a wide range of healthcare products globally. The company provides products that find applications in cancer, cardio-metabolic, immunology & dermatology, ophthalmology, neuroscience, and respiratory disease areas. Its Sandoz segment offers active ingredients and finished dosage forms of pharmaceuticals for cardiovascular, dermatology, central nervous system (CNS), gastrointestinal and hormonal therapy, metabolism, oncology, ophthalmic, pain, and respiratory disorders and diseases; and finished dosage forms anti-infective. It operates in the Americas, Europe, the Middle East, and Asia-Pacific.


Key Companies in the Orphan Drugs Market include



  • F. Hoffmann-La Roche AG (Switzerland)

  • Mylan (US)

  • Celgene Corporation (US)

  • Novartis AG (Switzerland)

  • Biogen (US)

  • Takeda Pharmaceutical Company Limited (Japan)

  • Merck KGaA (Germany)

  • Eli Lilly And Company (US)

  • Sanofi (France)

  • Janssen Services LLC (US)


Orphan Drugs Industry Developments


July 2021:Novartis Ag (Switzerland) received a U.S. FDA grant as Orphan Drug Designation (ODD) for NIS793 (which is a first-class novel antibody, specific for Transforming Growth Factor, Beta) in combination with standard care of chemotherapy, for the treatment of pancreatic chemotherapy.


November 2019:Celgene Corporation (US) received approval for an orphan medicine named REBLOZYL for the treatment of anemia in adults with beta-thalassemia who require regular red blood cell (RBC) transfusion.


July 2021:Novartis AG (Switzerland) received FDA orphan drug designation for NIS793 in pancreatic cancer. NIS793 is a potential first-in-class novel antibody specific for Transforming Growth Factor Beta (TGFฮฒ), which is known to have an important role in metastatic pancreatic ductal carcinoma (mPDAC) and other solid tumors.


February 2021:FDA approved for TEPMETKO Merck KGaA's (Germany)oral MET inhibitor, the first and only once-daily treatment for patients with metastatic non-small cell lung cancer with METex14 skipping mutations.


May 2020:Eli Lilly and Company (US) received U.S. FDA Approval for Retevmo (selpercatinib), the First Therapy Specifically for Patients with Advanced RET-Driven Lung and Thyroid Cancers.


Orphan Drugs Market Segmentation


Orphan Drugs Type Outlook



  • Biologics

  • Non-Biologics


Orphan Drugs Sale Outlook



  • Generics

  • Prescribed


Orphan Drugs Drug Outlook



  • Revlimid

  • Rituxan

  • Opdivo

  • Keytruda

  • Imbruvica

  • Soliris

  • Jakaf

  • Pomalyst

  • Darzalex

  • Spinraza

  • Adcetris


Orphan Drugs Therapy Class Outlook



  • Oncology

  • Blood

  • Central Nervous Systems

  • Endocrine

  • Cardiovascular

  • Respiratory


Orphan Drugs Regional Outlook



  • North America

    • US

    • Canada



  • Europe

    • Germany

    • France

    • UK

    • Italy

    • Spain

    • Rest of Europe



  • Asia-Pacific

    • China

    • Japan

    • India

    • Australia

    • South Korea

    • Rest of Asia-Pacific



  • Rest of the World

    • Middle East

    • Africa

    • Latin America



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