Japan Rare Disease Treatment Market: By Molecule/Product (Selumetinib Eculizumab, Asfostatse Alfa, Ravulizumab-cwvz, Velaglucerase alfa, Imiglucerase, Elosulfasealfa, Agalsidase Beta BS, Lanadelumab, ADYNOVATE, and Others), By Disease (Hemophilia, Gaucher Disease, Morquio Syndrome, Fabry disease, Atypical Hemolytic Uremic Syndrome, Lysosomal Acid Lipase Deficiency, Neurofibromatosis Type 1 Plexiform Neurofibromas, Neuromyelitis Optica Spectrum Disorder, Paroxysmal Nocturnal, Hemoglobinuria, Hereditary Angioedema (HAE), and Others) 2019-2030

Japan Rare Disease Treatment Market size was valued at US$ 4,786.3 million in 2023 and is growing at a CAGR of 12.6% from 2024 - 2030. The rare disease treatment market is primarily driven by several key factors such as advancements in genetic research and diagnostic technologies which have improved the understanding and identification of rare devices facilitating more accurate diagnosis. In addition, the market has significant incentives that play a crucial role in encouraging pharmaceutical companies to invest in the development of rare disease therapeutics with regulatory incentives and orphan drug designations. Furthermore, advances in biotechnology and drug delivery systems, patient-centred approaches, and personalized medicine will enhance market growth.

However, the limited patient population fragmented market, and high costs and financial burdens are the challenges of Japan's rare diseases treatment market. For instance, according to the article “Evaluation of Clinical Practice Guidelines for rare diseases in Japan in 2018, out of 331 designated rare diseases, 327 were categorized as rare diseases because they affected fewer than 50,000 patients in Japan. Diseases affecting less than 50,000 patients qualify for orphan designation under pharmaceutical regulations, equating to a prevalence rate of approximately 4 in 10,000 people. By comparison, in the EU, diseases affecting fewer than 50in 10,000 people are classified as rare.

Japan Rare Disease Treatment Market Key Developments:

• In April 2024, NS pharma, Inc. announced that its parent company, Nippon Shinyaku Co., headquartered in Kyoto, Japan has entered into a collaborative research agreement with Mina therapeutics, based in London, United Kingdom aimed to develop nucleic acid drugs for treating challenging and rare central nervous systems diseases.
• In May 2024, Asahi Kasei, a Japanese pharmaceutical company announced its expansion of rare diseases sector through a proposed acquisition of Swedish Biotech firm Calliditas Therapeutics.