Rare Endocrine Disease Treatment Market Report 2025

The rare endocrine disease treatment market size has grown strongly in recent years. It will grow from $11.74 billion in 2024 to $12.58 billion in 2025 at a compound annual growth rate (CAGR) of 7.2%. The growth during the historic period was driven by increasing awareness of rare endocrine diseases, rising demand for hormone replacement therapies, growing prevalence of genetic and hormonal disorders, greater focus on specialized treatment centers, and expanding funding for rare disease research.

The rare endocrine disease treatment market size is expected to see strong growth in the next few years. It will grow to $16.41 billion in 2029 at a compound annual growth rate (CAGR) of 6.9%. The growth during the forecast period is fueled by the development of biologics and targeted therapies, rising emphasis on patient centric care, increasing investments from pharmaceutical companies, growing demand for digital health and telemedicine in rare disease management, and expanding support programs from governments and non profit organizations. The primary trends in the forecast period include integration of artificial intelligence in diagnosis and treatment planning, advancement of telemedicine platforms for chronic disease care, innovation in biologic and peptide based therapies, progress in genetic and molecular research, and improvements in patient monitoring technologies.

The growing focus on precision medicine is expected to drive the expansion of the rare endocrine disease treatment market. Precision medicine tailors prevention, diagnosis, and treatment plans to an individual’s genetic, environmental, and lifestyle factors. This approach is gaining traction due to advancements in genomics, which have improved the identification of disease mechanisms and facilitated the development of personalized treatment strategies. Precision medicine is especially beneficial in treating rare endocrine diseases by customizing therapies based on a patient’s genetic, molecular, and hormonal profiles. This leads to more precise diagnoses and targeted treatments that increase effectiveness while minimizing side effects. For example, in February 2024, the Personalized Medicine Coalition reported that the FDA approved 16 new personalized treatments for rare diseases in 2023, up from six in 2022. These treatments included seven cancer drugs and three treatments for other conditions, highlighting the growing role of precision medicine in addressing rare diseases. As a result, the increasing focus on precision medicine is driving the growth of the rare endocrine disease treatment market.

Companies in the rare endocrine disease treatment market are focusing on advancing therapies such as parathyroid hormone (PTH) prodrugs to improve treatment efficacy and prolong therapeutic effects for patients. PTH prodrugs are hormone therapies that involve attaching polyethylene glycol (PEG) to PTH, enhancing stability, extending circulation time, and providing longer-lasting therapeutic benefits for endocrine disorders. For example, in August 2024, Ascendis Pharma A/S, a Denmark-based biopharmaceutical company, received FDA approval for YORVIPATH, the first and only treatment for adult hypoparathyroidism, a rare endocrine disorder that affects around 70,000 to 90,000 people in the U.S. YORVIPATH uses Ascendis’ TransCon prodrug technology to provide continuous PTH exposure, reduce dosing frequency, and normalize serum calcium levels. Phase 3 trial results showed that many patients achieved normal calcium levels without needing additional vitamin D or high doses of calcium, marking a significant improvement over standard therapies. This innovation enhances clinical outcomes and improves quality of life for adults with hypoparathyroidism by addressing both short- and long-term complications.

In July 2024, AstraZeneca PLC, a UK-based biopharmaceutical company, acquired Amolyt Pharma SAS for $1.05 billion. The acquisition aims to strengthen AstraZeneca’s late-stage rare disease pipeline and expand its bone metabolism franchise, particularly targeting rare endocrine conditions such as hypoparathyroidism. Amolyt Pharma SAS, based in France, is a clinical-stage biotechnology company focused on developing innovative therapies for rare endocrine diseases. This move positions AstraZeneca to address significant unmet needs in the rare endocrine disease treatment space.

Major players in the rare endocrine disease treatment market are Pfizer Inc., Bayer AG, AstraZeneca PLC, Eli Lilly and Company, Novo Nordisk A/S, Sandoz Group AG, Alexion Pharmaceuticals Inc., Ipsen S.A., Kyowa Kirin Co. Ltd., BioMarin Pharmaceutical Inc., Recordati S.p.A., Camurus AB, EMD Serono Inc., Corcept Therapeutics Inc., Ascendis Pharma A/S, Inozyme Pharma Inc., Spruce Biosciences Inc., MBX Biosciences Inc., Crinetics Pharmaceuticals Inc., Tiburio Therapeutics Inc.

North America was the largest region in the rare endocrine disease treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in rare endocrine disease treatment report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the rare endocrine disease 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 healthcare sector, particularly in the supply of critical medical devices, diagnostic equipment, and pharmaceuticals. Hospitals and healthcare providers are facing higher costs for imported surgical instruments, imaging equipment, and consumables such as syringes and catheters, many of which have limited domestic alternatives. These increased costs are straining healthcare budgets, leading some providers to delay equipment upgrades or pass on expenses to patients. Additionally, tariffs on raw materials and components are disrupting the production of essential drugs and devices, causing supply chain bottlenecks. In response, the industry is diversifying sourcing strategies, boosting local manufacturing where possible, and advocating for tariff exemptions on life-saving medical products.

Rare endocrine disease treatment refers to the medical management of uncommon disorders that affect the hormone producing glands of the body. These treatments aim to restore hormonal balance, relieve symptoms, and prevent complications in order to improve patients’ quality of life.

The primary types of rare endocrine disease treatment are biologics and organic compounds. Biologics are therapeutic products derived from living organisms or their components, such as proteins, cells, or nucleic acids, and are used to prevent, treat, or manage diseases. Treatment approaches include hormone replacement therapy, radiotherapy, surgery, targeted therapy, and immunotherapy, which are applied to conditions such as Cushing's syndrome, Addison's disease, acromegaly, and others. Distribution channels include hospital pharmacies, retail pharmacies, online pharmacies, direct sales, and others, while end users encompass hospitals, specialty clinics, research institutes, and others.

The rare endocrine disease treatment market research report is one of a series of new reports that provides rare endocrine disease treatment market statistics, including rare endocrine disease treatment industry global market size, regional shares, competitors with a rare endocrine disease treatment market share, detailed rare endocrine disease treatment market segments, market trends and opportunities, and any further data you may need to thrive in the rare endocrine disease treatment industry. This rare endocrine disease 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 endocrine disease treatment market consists of revenues earned by entities by providing services such as multidisciplinary clinical care, personalized treatment options, clinical trials and research care coordination, and long-term support. The market value includes the value of related goods sold by the service provider or included within the service offering. The rare endocrine disease treatment market also includes sales of diagnostic imaging tools, laboratory and molecular diagnostic devices, monitoring & therapeutic devices, and Digital and Telemedicine Infrastructure. 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.

This product will be delivered within 3-5 business days.