Japan Orphan Drugs Market

Market Overview:

Japan orphan drugs market size reached USD 14,034 Million in 2024. Looking forward, IMARC Group expects the market to reach USD 32,687 Million by 2033, exhibiting a growth rate (CAGR) of 9.8% during 2025-2033. The rising number of rare diseases lacking treatment, along with improving access to healthcare, represent some of the key factors driving the market.

Orphan drugs are pharmaceutical products designed for the diagnosis, prevention, and treatment of rare medical conditions. These medications are developed to address specific healthcare needs and typically have a limited market because they are intended for a small number of patients. They prove effective against a wide range of medical conditions, including but not limited to oncological, metabolic, hematologic, immunologic, infectious, and neurological diseases. In addition to this, many of these conditions, such as lymphoma, leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, and renal cell carcinoma, are often life-threatening, chronic, progressive, degenerative, and disabling. They necessitate specialized treatment options that are tailored to address their unique symptoms. Consequently, orphan drugs are widely utilized in hospitals across Japan.

Japan Orphan Drugs Market Trends:

In the context of the Japan market, several factors are fueling market growth. One significant driver is the increasing prevalence of cancer and rare genetic disorders. Pharmaceutical manufacturers are actively developing innovative orphan drugs to offer personalized therapy options to patients in response to this growing demand, thereby positively influencing the regional market. Furthermore, the market is benefiting from heightened public awareness of the advantages of orphan drugs. Besides this, the emergence of new pharmaceutical companies in the market and the implementation of favorable government policies aimed at curbing the spread of contagious diseases are also contributing to this positive growth trajectory. In addition to these factors, various product innovations, such as the creation of biological orphan drugs, are serving as additional growth catalysts. These drugs have the capacity to treat diseases like cancer and reverse the damage inflicted on stem cells, significantly boosting their demand, which is acting as another significant growth-inducing factor. Moreover, improvements in healthcare infrastructure and a substantial focus on research and development (R&D) activities are expected to further propel the market's expansion.

Japan Orphan Drugs Market Segmentation:

IMARC Group provides an analysis of the key trends in each segment of the market, along with forecasts at the country level for 2025-2033. Our report has categorized the market based on drug type, disease type, phase, top selling drugs, and distribution channel.

Drug Type Insights:

• Biological
• Non-Biological

The report has provided a detailed breakup and analysis of the market based on the drug type. This includes biological and non-biological.

Disease Type Insights:

• Oncology
• Hematology
• Neurology
• Cardiovascular
• Others

A detailed breakup and analysis of the market based on the disease type have also been provided in the report. This includes oncology, hematology, neurology, cardiovascular, and others.

Phase Insights:

• Phase I
• Phase II
• Phase III
• Phase IV

The report has provided a detailed breakup and analysis of the market based on the phase. This includes phase I, phase II, phase III, and phase IV.

Top Selling Drugs Insights:

• Revlimid
• Rituxan
• Copaxone
• Opdivo
• Keytruda
• Imbruvica
• Avonex
• Sensipar
• Soliris
• Others

A detailed breakup and analysis of the market based on the top selling drugs have also been provided in the report. This includes revlimid, rituxan, copaxone, opdivo, keytruda, imbruvica, avonex, sensipar, soliris, and others.

Distribution Channel Insights:

• Hospital Pharmacies
• Retail Pharmacies
• Online Stores
• Others

The report has provided a detailed breakup and analysis of the market based on the distribution channel. This includes hospital pharmacies, retail pharmacies, online stores, and others.

Regional Insights:

• Kanto Region
• Kansai/Kinki Region
• Central/ Chubu Region
• Kyushu-Okinawa Region
• Tohoku Region
• Chugoku Region
• Hokkaido Region
• Shikoku Region

The report has also provided a comprehensive analysis of all the major regional markets, which include Kanto Region, Kansai/Kinki Region, Central/ Chubu Region, Kyushu-Okinawa Region, Tohoku Region, Chugoku Region, Hokkaido Region, and Shikoku Region.

Competitive Landscape:

The market research report has also provided a comprehensive analysis of the competitive landscape in the market. Competitive analysis such as market structure, key player positioning, top winning strategies, competitive dashboard, and company evaluation quadrant has been covered in the report. Also, detailed profiles of all major companies have been provided.

Japan Orphan Drugs Market:

• April 2025: Avidity Biosciences, Inc., a biopharmaceutical business dedicated to the development of a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs™), announced that Japan Ministry of Health, Labour and Welfare (MHLW) has designated delpacibart etedesiran (del-desiran) as an Orphan Drug designation (ODD) for myotonic dystrophy type 1 (DM1), an investigational therapy aimed at the root cause of DM1, an underdiagnosed, progressive and frequently lethal neuromuscular disease without approved treatments.
• March 2025: HanAll Biopharma Co., Ltd., a biopharmaceutical company with global operations dedicated to the discovery and development of novel medicines for patients, reported that Batoclimab, an anti-FcRn therapy in development for a variety of autoimmune conditions, has been granted Orphan Drug Designation (ODD) by the Ministry of Health, Labor and Welfare (MHLW) in Japan for active Thyroid Eye Disease (TED).
• February 2025: Essential Pharma and Clinigen, the global specialist pharmaceutical services group, announced an extension of their JAPAC partnership, to further drive patient access to four therapies in Japan and Korea, and enhance their combined market presence within the region. Essential Pharma CEO Emma Johnson and Clinigen CEO Jerome Charton hosted a ceremonial signing ceremony yesterday. While initially concentrated on maintaining continuity of patient access in the JAPAC region, the two organizations see further strategic collaboration, advancing Essential Pharma's aspiration to expand on its expanded portfolio and late-stage pipeline with central nervous system, gastroenterology, ophthalmology, and rare disease focus and drawing on Clinigen's rare disease and orphan drug capabilities, to enhance access to novel medicines for patients worldwide.
• January 2025: KalVista Pharmaceuticals is granted Orphan Drug Designation and Submits New Drug Application for Sebetralstat in Japan for Hereditary Angioedema. Upon approval, sebetralstat would be the first oral on-demand therapy for HAE in Japan.

Japan Orphan Drugs Market Report Coverage:

Key Benefits for Stakeholders:

• IMARC’s industry report offers a comprehensive quantitative analysis of various market segments, historical and current market trends, market forecasts, and dynamics of the Japan orphan drugs market from 2019-2033.
• The research report provides the latest information on the market drivers, challenges, and opportunities in the Japan orphan drugs market.
• Porter's five forces analysis assist stakeholders in assessing the impact of new entrants, competitive rivalry, supplier power, buyer power, and the threat of substitution. It helps stakeholders to analyze the level of competition within the Japan orphan drugs industry and its attractiveness.
• Competitive landscape allows stakeholders to understand their competitive environment and provides an insight into the current positions of key players in the market.