Myelodysplastic Syndrome Drugs Market Report 2025

The myelodysplastic syndrome drugs market size has grown strongly in recent years. It will grow from $2.25 billion in 2024 to $2.44 billion in 2025 at a compound annual growth rate (CAGR) of 8.3%. The growth in the historic period can be attributed to aging population, advancements in diagnostics, chemotherapy-related MDS cases, increased cancer survivors, clinical research and drug development.

The myelodysplastic syndrome drugs market size is expected to see strong growth in the next few years. It will grow to $3.48 billion in 2029 at a compound annual growth rate (CAGR) of 9.3%. The growth in the forecast period can be attributed to growing geriatric population, advancements in targeted therapies, increased understanding of disease mechanisms, rising awareness and early diagnosis, expansion of oncology research. Major trends in the forecast period include erythropoiesis-stimulating agents optimization, combination therapies, increasing focus on iron chelation therapy, regulatory approvals and market access, patient-centric care models.

The forecast of 9.3% growth over the next five years reflects a modest reduction of 0.4% from the previous estimate for this market. This reduction is primarily due to the impact of tariffs between the US and other countries. Tariff barriers are expected to hamper U.S. hematology clinics by increasing the cost of hypomethylating agents and growth factor therapies sourced from Sweden and Singapore, thereby delaying bone marrow disorder treatments and elevating specialized oncology care expenses. The effect will also be felt more widely due to reciprocal tariffs and the negative effect on the global economy and trade due to increased trade tensions and restrictions.

The anticipated rise in myelodysplastic syndrome cases is set to drive growth in the market for drugs treating this condition. Myelodysplastic syndrome represents a group of disorders impacting blood cell development in the bone marrow, categorized as a form of blood cancer. Medications for this syndrome aim to modulate the immune system, reducing the need for red blood cell transfusions. Notably, estimates from the American Journal of Managed Care in October 2022 place the prevalence of myelodysplastic syndrome in the USA between 60,000 to 175,000 cases, substantiating the market's growth due to increased incidences.

The burgeoning demand for personalized medicine is expected to be a driving force behind the expansion of the myelodysplastic syndrome drugs market. Personalized medicine involves tailoring treatments based on an individual's unique genetic and medical characteristics. This approach optimizes treatment outcomes and minimizes adverse effects, especially relevant in managing the heterogeneous nature of myelodysplastic syndrome. Notably, in 2022, the FDA's Center for Drug Evaluation and Research greenlit 37 new molecular entities, with the Personalized Medicine Coalition categorizing 12, around 34%, as personalized medicines, emphasizing the market's growth trajectory.

Leading companies in the myelodysplastic syndrome drugs market are focusing on developing telomerase inhibitors to target the enzyme telomerase, with the aim of slowing disease progression by limiting the uncontrolled division of abnormal cells. These inhibitors are beneficial in treating myelodysplastic syndrome as they function by inhibiting telomerase, which can reduce the proliferation of abnormal cells and potentially enhance patient outcomes. For example, in June 2024, Geron Corporation, a biotechnology firm based in the U.S., launched Rytelo (imetelstat), an FDA-approved intravenous telomerase inhibitor designed for adults with low to intermediate-1 risk myelodysplastic syndromes (LR-MDS) and transfusion-dependent anemia that is unresponsive to and ineligible for erythropoiesis-stimulating agents (ESA). Rytelo provides durable transfusion independence, improves hemoglobin levels, and addresses a significant unmet need in the myelodysplastic syndrome drugs market while maintaining a manageable safety profile.

Prominent companies operating in the myelodysplastic syndrome drugs sector are intensifying efforts to introduce novel medications, aiming to gain a competitive edge. TIBSOVO, an oral targeted therapy inhibiting the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, is a prime example. Approved for treating adult patients with relapsed or refractory myelodysplastic syndromes (MDS), TIBSOVO represents a significant milestone in the industry. Notably, Servier Laboratories' approval of TIBSOVO by the US FDA in October 2023 marks the first targeted therapy sanctioned for this indication. The approval stems from a pivotal Phase I study evaluating its efficacy in this new application.

In January 2023, Merck, a pharmaceutical company based in the U.S., acquired Imago Biosciences, Inc. for an undisclosed amount. This acquisition is intended to strengthen Merck's hematology capabilities by incorporating Imago Biosciences' innovative therapies. Imago Biosciences, Inc. specializes in the development of cutting-edge therapies for hematologic malignancies, including myelodysplastic syndromes (MDS).

Major companies operating in the myelodysplastic syndrome drugs market include Amgen Inc., Celgene Corporation, Otsuka Pharmaceutical Co. Ltd., Takeda Pharmaceutical Company Limited, Cipla Limited, Dr. Reddy's Laboratories Ltd., Lupin Ltd., Onconova Therapeutics Inc., Sun Pharmaceutical Industries Limited, FibroGen Inc., Aprea Therapeutics, Bayer AG, Bristol Myers Squibb, Celator Pharmaceuticals, Crystal Genomics Inc., AbbVie Inc., Astex Pharmaceuticals Inc., Geron Co., Hikma Pharmaceuticals plc, Jazz Pharmaceuticals Inc., Mylan NV, Novartis AG, Sandoz Inc., Syros Pharmaceuticals, Teva Pharmaceutical Industries Ltd., Acceleron Pharma Inc., Agios Pharmaceuticals Inc., Incyte Corporation, Array Biopharma Inc., MEI Pharma Inc.

North America was the largest region in the Myelodysplastic syndrome drugs market in 2024. Asia-Pacific is expected to be the fastest growing region in the global myelodysplastic syndrome drugs market report during the forecast period. The regions covered in the myelodysplastic syndrome drugs market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the myelodysplastic syndrome drugs 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 resulting trade tensions in spring 2025 are having a significant impact on the pharmaceutical sector. Companies are grappling with higher costs on imported active pharmaceutical ingredients (APIs), glass vials, and laboratory equipment - many of which have limited alternative sources. Generic drug manufacturers, already operating with minimal profit margins, are particularly affected, with some scaling back production of low-margin medications. Biotech firms are also experiencing delays in clinical trials due to shortages of specialized reagents linked to tariffs. In response, the industry is shifting API production to regions like India and Europe, building up inventory reserves, and advocating for tariff exemptions on essential medicines.

The myelodysplastic syndrome drugs market research report is one of a series of new reports that provides myelodysplastic syndrome drugs market statistics, including myelodysplastic syndrome drugs industry global market size, regional shares, competitors with a myelodysplastic syndrome drugs market share, detailed myelodysplastic syndrome drugs market segments, market trends and opportunities, and any further data you may need to thrive in the myelodysplastic syndrome drugs industry. This myelodysplastic syndrome drugs 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.

Myelodysplastic syndrome (MDS) covers a spectrum of conditions where the bone marrow struggles to produce enough healthy blood cells - red blood cells, white blood cells, and platelets due to irregular cell formation and maturation. This deficiency arises from uncontrolled cell growth and division, causing tissue damage. Medications for myelodysplastic syndrome encompass epoetin alfa, darbepoetin alfa, filgrastim, and other drugs tailored for MDS treatment.

The primary types of drug therapies for myelodysplastic syndrome fall into three categories immunomodulatory drugs, hypomethylating agents, and anti-anemics. Immunomodulatory drugs (IMiDs) constitute a medication class that adjusts or regulates the immune system's response while directly impacting bone marrow cells. The spectrum of syndromes includes refractory cytopenia with multilineage dysplasia, refractory anemia, refractory anemia with excess blasts, and refractory anemia with ringed sideroblasts. These drugs can be administered through various routes such as oral and parenteral methods and serve multiple applications, including original and generic treatments. They are utilized by various end-users, including hospitals, clinics, and ambulatory surgical centers, to address the diverse needs of individuals diagnosed with MDS.

The myelodysplastic syndrome drug market consists of sales of drugs such as azacytidine, decitabine, and lenalidomide. 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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