Small Molecule Targeted Therapy Market Report 2025

The small molecule targeted therapy market size has grown strongly in recent years. It will grow from $70.45 billion in 2024 to $77.29 billion in 2025 at a compound annual growth rate (CAGR) of 9.7%. The growth during the historic period was driven by the expanding availability of advanced diagnostic tools, increasing number of clinical trials, heightened focus on oncology and rare disease treatments, growing regulatory approvals for innovative small molecule drugs, and rising emphasis on combination therapies.

The small molecule targeted therapy market size is expected to see strong growth in the next few years. It will grow to $110.52 billion in 2029 at a compound annual growth rate (CAGR) of 9.4%. The growth during the forecast period is fueled by increasing prevalence of chronic and genetic diseases, greater adoption of personalized medicine, growing investment in pharmaceutical research and development, rising awareness among healthcare professionals and patients, and higher government support and funding. The primary trends in the forecast period include technological advancements in drug design, progress in high-throughput screening techniques, development of precision medicine platforms, technology-driven improvements in drug delivery systems, and integration of genomics and proteomics data.

The increasing focus on personalized medicine is anticipated to drive growth in the small molecule targeted therapy market. Personalized medicine is a healthcare strategy that customizes prevention, diagnosis, and treatment based on an individual’s genetic, environmental, and lifestyle factors. This growing shift toward personalized approaches is largely fueled by advancements in genomics, which have enabled a deeper understanding of genetic variations and facilitated the development of more precise, targeted treatments that enhance patient outcomes and minimize trial-and-error in therapies. Small molecule targeted therapy contributes to personalized medicine by specifically targeting a patient’s unique molecular or genetic disease profile, improving treatment efficacy and reducing side effects. For example, in February 2024, the Personalized Medicine Coalition, a non-profit organization based in the US, reported that in 2023, the FDA approved 16 new personalized treatments for rare disease patients, a significant increase from six in 2022. These approvals included seven cancer drugs and three for other conditions, underscoring the role of personalized medicine in driving growth in the small molecule targeted therapy market.

Companies in the small molecule targeted therapy market are focusing on creating innovative oral solutions to improve targeted drug delivery, enhance patient compliance, and address specific molecular pathways, especially in oncology and chronic diseases. Oral small molecules are low-molecular-weight drugs designed to be taken by mouth, which can easily enter cells and target disease-related proteins or pathways. For instance, in June 2025, Kura Oncology Inc., a biotech company based in the US, received FDA approval for its New Drug Application (NDA) for ziftomenib, an oral treatment for adult patients with relapsed or refractory acute myeloid leukemia (AML) with nucleophosmin 1 (NPM1) mutations. This oral targeted therapy addresses a critical unmet need for adult AML patients with NPM1 mutations, offering a favorable safety profile and promising efficacy, including significant complete remission rates in clinical trials. Ziftomenib works by selectively inhibiting the menin-KMT2A interaction, disrupting leukemic cell growth and providing a new treatment option for this difficult-to-treat subtype of AML.

In May 2025, Eli Lilly and Company, a US-based pharmaceutical firm, acquired SiteOne Therapeutics Inc. for an undisclosed amount. The acquisition strengthens Eli Lilly's chronic pain treatment portfolio by adding innovative, non-addictive therapies targeting chronic pain through advanced sodium channel inhibitors. SiteOne Therapeutics Inc., also based in the US, specializes in small molecule targeted therapies.

Major players in the small molecule targeted therapy market are F. Hoffmann-La Roche AG (Roche), Merck KGaA, Pfizer Inc., Bayer AG, Sanofi S.A., Novartis International AG, AstraZeneca PLC, Bristol-Myers Squibb Company, GlaxoSmithKline plc (GSK), Eli Lilly and Company, Amgen Inc., Regeneron Pharmaceuticals Inc., Astellas Pharma Inc., Lonza Group AG, Blueprint Medicines Corporation, Kura Oncology Inc., G1 Therapeutics Inc., Ascentage Pharma Group International Inc., Rgenta Therapeutics Inc., Verastem Inc.

North America was the largest region in the small molecule targeted therapy market in 2024. Asia Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in small molecule targeted therapy report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the small molecule targeted therapy 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.

Small molecule targeted therapy consists of low-molecular-weight drugs that specifically interact with disease-related targets such as enzymes, receptors, or signaling proteins. These therapies selectively inhibit or modulate disease pathways, enabling precise treatment while minimizing effects on healthy cells.

The primary therapy types of small molecule targeted therapy are chemotherapy, targeted therapy, and immunotherapy. Chemotherapy involves the use of chemical drugs to kill rapidly dividing cells in the body. Key drug classes, including monoclonal antibodies, small molecule inhibitors, and combination therapies, act through mechanisms such as kinase inhibition, proteasome inhibition, aromatic hydrocarbon receptor modulation, and cell cycle inhibition. This therapy is applied in conditions such as cancer, autoimmune diseases, and infectious diseases and can be administered orally, intravenously, or subcutaneously.

The small molecule targeted therapy market research report is one of a series of new reports that provides small molecule targeted therapy market statistics, including small molecule targeted therapy industry global market size, regional shares, competitors with a small molecule targeted therapy market share, detailed small molecule targeted therapy market segments, market trends and opportunities, and any further data you may need to thrive in the small molecule targeted therapy industry. This small molecule targeted therapy 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 small molecule targeted therapy market consists of sales of tyrosine kinase inhibitors, proteasome inhibitors, and BCL-2 inhibitors. 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 and 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.