Rare Gastrointestinal Diseases Treatment Market Report 2025

The rare gastrointestinal diseases treatment market size has grown strongly in recent years. It will grow from $1.36 billion in 2024 to $1.45 billion in 2025 at a compound annual growth rate (CAGR) of 6.1%. The growth during the historic period is linked to increasing patient advocacy initiatives, a rising number of clinical trials for rare gastrointestinal (GI) therapies, greater early diagnosis through advanced screening, expanding collaboration between pharmaceutical companies and research institutes, and growing demand for targeted treatment options.

The rare gastrointestinal diseases treatment market size is expected to see strong growth in the next few years. It will grow to $1.81 billion in 2029 at a compound annual growth rate (CAGR) of 5.7%. The growth projected for the forecast period is driven by increasing investment in novel drug development, rising adoption of personalized medicine, expansion of precision medicine, enhanced awareness and education among healthcare providers, and growth of rare disease patient registries. Primary trends during the forecast period include advancements in genomic and molecular diagnostics, development of targeted biologic therapies, innovations in drug delivery systems, progress in telehealth and remote patient monitoring, and development of AI-driven clinical decision support tools.

The increasing use of diagnostic testing is expected to drive growth in the rare gastrointestinal diseases treatment market in the coming years. Diagnostic tests are medical procedures performed to detect, diagnose, or monitor diseases, conditions, or infections in patients. Growing awareness about the importance of early disease detection is fueling demand for these tests, as timely diagnosis allows for prompt intervention and improved patient outcomes. Diagnostic testing plays a crucial role in treating rare gastrointestinal diseases by enabling early and accurate identification of the underlying conditions. It helps pinpoint specific disease pathways, supports personalized treatment planning, and improves overall patient care through timely medical responses. For example, in December 2024, the UK’s National Health Service reported that the number of diagnostic tests conducted reached 2,544,809, up from 2,312,030 in 2023. This rise in diagnostic testing is therefore driving the market for rare gastrointestinal disease treatments.

Leading companies in this market are developing innovative therapies such as fully human monoclonal antibodies that target disease pathways to enhance patient outcomes. These antibodies are lab-engineered proteins derived entirely from human sources, designed to precisely target molecules responsible for disease, improving treatment effectiveness while minimizing side effects. For instance, in August 2023, Regeneron Pharmaceuticals, a US biotechnology firm, gained FDA approval for Veopoz (pozelimab-bbfg), a treatment for CHAPLE disease - a rare, hereditary gastrointestinal disorder. Veopoz is a fully human monoclonal antibody that targets complement factor C5 to regulate the overactive complement system responsible for protein loss and gastrointestinal damage in CHAPLE patients. This targeted approach addresses the root cause of the disease, preventing severe symptoms and potentially life-threatening complications.

In May 2023, Ironwood Pharmaceuticals, a US-based company specializing in gastrointestinal healthcare, acquired VectivBio Holding AG, a Switzerland-based clinical-stage biotechnology firm, for about $1 billion. Through this acquisition, Ironwood aims to broaden its gastrointestinal product portfolio by adding VectivBio’s late-stage drug candidate, apraglutide, which is being developed to treat short bowel syndrome with intestinal failure. The deal leverages Ironwood’s commercial and clinical expertise to accelerate growth, introduce innovative therapies, and enhance long-term shareholder value.

Major players in the rare gastrointestinal diseases treatment market are Johnson & Johnson, Merck & Co. Inc., Pfizer Inc., Bayer AG, Sanofi S.A., Zealand Pharma A/S, Novartis AG, AstraZeneca plc, GlaxoSmithKline plc, Eli Lilly and Company, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., Regeneron Pharmaceuticals Inc., Ipsen S.A., PTC Therapeutics Inc., Ironwood Pharmaceuticals Inc., Mirum Pharmaceuticals Inc., Albireo Pharma Inc., Orphalan S.A., and Ellodi Pharmaceuticals Inc.

North America was the largest region in the rare gastrointestinal diseases treatment market in 2024. The regions covered in rare gastrointestinal diseases treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the rare gastrointestinal diseases treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the pharmaceutical companies contend with tariffs on APIs, glass vials, and lab equipment inputs with few alternative sources. Generic drug makers, operating on razor-thin margins, are especially vulnerable, with some reducing production of low-profit medicines. Biotech firms face delays in clinical trials due to tariff-related shortages of specialized reagents. In response, the industry is expanding API production in India and Europe, increasing inventory stockpiles, and pushing for trade exemptions for essential medicines.

Rare gastrointestinal (GI) diseases treatment refers to specialized medical care, therapies, and interventions aimed at managing uncommon digestive system disorders that affect a small segment of the population. Its primary purpose is to alleviate symptoms, prevent complications, improve quality of life, and, when possible, achieve long-term disease control or remission.

The primary types of rare gastrointestinal diseases treatment include drugs and devices. Drugs are substances used to prevent, treat, or manage diseases and medical conditions, including specialized therapies such as biologics and small molecules. They target specific symptoms, reduce inflammation, and improve overall patient health and quality of life. These treatments can be administered via various routes, including oral, intravenous (IV), subcutaneous, and others. Applications include eosinophilic gastroenteritis, Hirschsprung's disease, and chronic intestinal pseudo-obstruction. End users include hospitals, research institutes, and other healthcare providers.

The rare gastrointestinal diseases treatment market research report is one of a series of new reports that provides rare gastrointestinal diseases treatment market statistics, including rare gastrointestinal diseases treatment industry global market size, regional shares, competitors with the rare gastrointestinal diseases treatment market share, rare gastrointestinal diseases treatment market segments, market trends, and opportunities, and any further data you may need to thrive in the rare gastrointestinal diseases treatment industry. This rare gastrointestinal diseases treatment market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The rare gastrointestinal diseases treatment market consists of revenues earned by entities by providing services such as diagnosis of rare gastrointestinal disorders, prescription of specialized therapies and medications, management and monitoring of patient treatment plans, and support programs for patients with rare digestive conditions. The market value includes the value of related goods sold by the service provider or included within the service offering. The rare gastrointestinal diseases treatment market also includes sales of specialized drugs, biologics, nutritional therapies, and diagnostic kits. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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