The global orphan drugs market size was estimated to be USD 177.01 billion in 2023 and is expected to reach at USD 638.47 billion by 2034 with a CAGR of 12.37% during the forecast period 2024-2034. Surge in research & development initiatives, rising investment for the development of innovative products, increasing awareness regarding rare diseases, surge in number of clinical trial procedures, and growing approvals of innovative orphan drugs by regulatory bodies are some of the key factors boosting the market growth.
Growing approvals of innovative orphan drugs by regulatory bodies is predicted to boost the market growth during the forecast period. The occurrence of rare diseases in the global population has been on the rise. To address this challenge, both developed and developing nations have established policies that support the advancement of medications for rare diseases and ensure their accessibility to patients. For instance, in March 2022, Ztalmy (ganaxolone) from Marinus Pharmaceuticals has been given U.S. FDA approval to treat seizures in individuals two years of age and older who have a rare cyclin-dependent kinase-like 5 (CDKL5) deficient condition (CDD).
By drug type, biologics was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to the existence of offerings labelled as biologics, rising mergers & acquisitions within market players, and increasing regulatory approvals. For instance, in June 2022, The Food and Drug Administration (FDA) has awarded orphan drug designation to evorpacept, a cutting-edge CD47 blocker developed by ALX Oncology Holdings Inc., for its potential use in treating individuals diagnosed with acute myeloid leukemia (AML). Additionally, non-biologics is predicted to grow at fastest CAGR during the forecast period owing to the growing research & development activities and increasing number of clinical trial procedures.
By therapy, oncology was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to growing research & development activities, presence of various oncology drugs, and surge in approvals of novel drugs. For instance, in February 2022, The U.S. FDA has approved CTI BioPharma's medication Pacritinib for the treatment of adult patients with myelofibrosis, a rare bone cancer that affects over 21,000 individuals in the country. Additionally, hematology is predicted to grow at fastest CAGR during the forecast period owing to the increasing number of product launches and growing approvals by regulatory bodies.
By distribution channel, hospital pharmacies was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to a large number of medications must be given intravenously by qualified medical personnel in hospitals and surge in approvals by regulatory bodies. For instance, in March 2022, The Food and Drug Administration (FDA) awarded orphan drug classification to Intellia Therapeutics' CRISPR-edited T cell receptor (TCR) T-cell therapy, which is being tested for the treatment of acute myeloid leukemia (AML). Additionally, online pharmacies is predicted to grow at fastest CAGR during the forecast period owing to the increasing numbers of individuals are purchasing these medications from internet pharmacies.
North America region is anticipated for the highest revenue share during the forecast period owing to the rising expenditures on orphan drugs, increasing focus on development of innovative products, and surge in mergers & acquisitions within market players. For instance, in November 2022, AstraZeneca subsidiary Alexion, which focuses on rare illnesses, has acquired LogicBio Therapeutics, Inc., a pioneer in the field of genomic medicine. This purchase was undertaken to hasten Alexion's advancement in genomic medicine and the treatment of uncommon illnesses. Additionally, Asia Pacific region is predicted to grow at fastest CAGR during the forecast period owing to the growing healthcare expenditures, rising awareness regarding rare diseases, and increasing partnerships within market players. For instance, in December 2022, Amgen Inc. made the announcement that it will buy Horizon Therapeutics Plc and its orphan medication portfolio for USD 27.8 billion.
Growing approvals of innovative orphan drugs by regulatory bodies is predicted to boost the market growth during the forecast period. The occurrence of rare diseases in the global population has been on the rise. To address this challenge, both developed and developing nations have established policies that support the advancement of medications for rare diseases and ensure their accessibility to patients. For instance, in March 2022, Ztalmy (ganaxolone) from Marinus Pharmaceuticals has been given U.S. FDA approval to treat seizures in individuals two years of age and older who have a rare cyclin-dependent kinase-like 5 (CDKL5) deficient condition (CDD).
By drug type, biologics was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to the existence of offerings labelled as biologics, rising mergers & acquisitions within market players, and increasing regulatory approvals. For instance, in June 2022, The Food and Drug Administration (FDA) has awarded orphan drug designation to evorpacept, a cutting-edge CD47 blocker developed by ALX Oncology Holdings Inc., for its potential use in treating individuals diagnosed with acute myeloid leukemia (AML). Additionally, non-biologics is predicted to grow at fastest CAGR during the forecast period owing to the growing research & development activities and increasing number of clinical trial procedures.
By therapy, oncology was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to growing research & development activities, presence of various oncology drugs, and surge in approvals of novel drugs. For instance, in February 2022, The U.S. FDA has approved CTI BioPharma's medication Pacritinib for the treatment of adult patients with myelofibrosis, a rare bone cancer that affects over 21,000 individuals in the country. Additionally, hematology is predicted to grow at fastest CAGR during the forecast period owing to the increasing number of product launches and growing approvals by regulatory bodies.
By distribution channel, hospital pharmacies was the highest revenue-grossing segment in the global orphan drugs market in 2023 owing to a large number of medications must be given intravenously by qualified medical personnel in hospitals and surge in approvals by regulatory bodies. For instance, in March 2022, The Food and Drug Administration (FDA) awarded orphan drug classification to Intellia Therapeutics' CRISPR-edited T cell receptor (TCR) T-cell therapy, which is being tested for the treatment of acute myeloid leukemia (AML). Additionally, online pharmacies is predicted to grow at fastest CAGR during the forecast period owing to the increasing numbers of individuals are purchasing these medications from internet pharmacies.
North America region is anticipated for the highest revenue share during the forecast period owing to the rising expenditures on orphan drugs, increasing focus on development of innovative products, and surge in mergers & acquisitions within market players. For instance, in November 2022, AstraZeneca subsidiary Alexion, which focuses on rare illnesses, has acquired LogicBio Therapeutics, Inc., a pioneer in the field of genomic medicine. This purchase was undertaken to hasten Alexion's advancement in genomic medicine and the treatment of uncommon illnesses. Additionally, Asia Pacific region is predicted to grow at fastest CAGR during the forecast period owing to the growing healthcare expenditures, rising awareness regarding rare diseases, and increasing partnerships within market players. For instance, in December 2022, Amgen Inc. made the announcement that it will buy Horizon Therapeutics Plc and its orphan medication portfolio for USD 27.8 billion.
Segmentation: Orphan Drugs Market Report 2022 - 2033
Orphan Drugs Market Analysis & Forecast by Drug Type 2023 - 2034 (Revenue USD Bn)
- Non-Biologics
- Biologics
Orphan Drugs Market Analysis & Forecast by Therapy 2023 - 2034 (Revenue USD Bn)
- Neurology
- Cardiovascular
- Oncology
- Hematology
- Endocrinology
- Immunotherapy
- Infectious Diseases
- Respiratory
- Others
Orphan Drugs Market Analysis & Forecast by Distribution Channel 2023 - 2034 (Revenue USD Bn)
- Online Pharmacies
- Hospital Pharmacies
- Retail Pharmacies
Orphan Drugs Market Analysis & Forecast by Region 2023 - 2034 (Revenue USD Bn)
North America
- U.S.
- Canada
Europe
- Germany
- France
- UK
- Spain
- Italy
- Rest of Europe
Asia Pacific
- China
- Japan
- India
- Australia
- South Korea
- Rest of APAC
Latin America
- Brazil
- Mexico
- Argentina
- Rest of LATAM
Middle East & Africa
- South Africa
- GCC
- Rest of MEA
Table of Contents
1. Research Methodology
2. Introduction
4. Market Environment Analysis
5. Market Dynamics
7. Orphan Drugs Market: Drug Type Estimates & Trend Analysis
8. Orphan Drugs Market: Therapy Estimates & Trend Analysis
9. Orphan Drugs Market: Distribution Channel Estimates & Trend Analysis
10. Regional Market Analysis
11. North America Orphan Drugs Market
12. Europe Global Orphan Drugs Market
13. Asia Pacific Global Orphan Drugs Market
14. Latin America Global Orphan Drugs Market
15. MEA Global Orphan Drugs Market
16. Competitor Analysis
17. Company Profiles
Companies Mentioned
- Bristol-Myers Squibb Company
- Amryt Pharma Plc.
- Glaxosmithkline Plc (Gsk)
- Johnson & Johnson (Janssen Global Services LLC)
- Sanofi S.A
- Novartis International Ag (Sandoz)
- Pfizer Inc.
- Abbvie Inc.
- F. Hoffmann-La Roche Ltd.
- Amgen Inc.
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 145 |
| Published | October 2023 |
| Forecast Period | 2023 - 2034 |
| Estimated Market Value ( USD | $ 177.01 Billion |
| Forecasted Market Value ( USD | $ 638.47 Billion |
| Compound Annual Growth Rate | 12.3% |
| Regions Covered | Global |
| No. of Companies Mentioned | 10 |
