KIT Inhibitor - Target Population, Competitive Landscape, and Market Forecast - 2034

Key Highlights:

• KIT inhibitors block the overactive KIT receptor tyrosine kinase, a key driver in cancers like gastrointestinal stromal tumor (GIST), systemic mastocytosis, and some leukemias. By disrupting KIT signaling, they target abnormal cell growth and survival linked to KIT mutations and resistance pathways.
• Multiple KIT inhibitors have received regulatory approval across various indications, highlighting their therapeutic relevance. These include ROMVIMZA (vimseltinib) for tenosynovial giant cell tumor (TGCT); AYVAKIT/AYVAKYT (avapritinib) for GIST, advanced systemic mastocytosis (AdvSM), and indolent systemic mastocytosis (ISM); and QINLOCK (ripretinib) for advanced GIST, among others.
• The KIT inhibitors market remains highly competitive, driven by established players such as ONO Pharmaceutical and Blueprint Medicines, among others. At the same time, companies like Celldex Therapeutics, Cogent Biosciences, and Blueprint Medicines, among others are actively advancing pipeline candidates across various stages of development. This sustained R\&D activity reflects ongoing innovation and intensifying competition within the KIT-targeted therapy landscape.
• Emerging KIT inhibitors in the pipeline include barzolvolimab (CDX-0159), bezuclastinib (CGT9486), and elenestinib (BLU-263), among others, each demonstrating encouraging potential across a range of indications. These candidates are poised to diversify and strengthen the therapeutic arsenal, potentially addressing unmet needs in both oncology and rare disease settings.
• In February 2025, the US FDA approved ROMVIMZA (vimseltinib) for symptomatic TGCT in adults where surgery poses high risk of functional impairment, addressing a critical unmet need in this population.

DelveInsight's "KIT Inhibitor" - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the KIT inhibitor, historical and projected epidemiological data, competitive landscape as well as the KIT inhibitor market trends in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.

The KIT inhibitor market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM KIT inhibitor market size from 2020 to 2034. The report also covers current KIT inhibitor treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

KIT Inhibitor Understanding and Treatment Algorithm

KIT Inhibitor Overview

KIT inhibitors are targeted therapies designed to block the aberrant activity of the KIT receptor tyrosine kinase, which is frequently mutated or overexpressed in malignancies such as GIST, systemic mastocytosis, and certain leukemias.

By inhibiting KIT-driven signaling, these agents disrupt key pathways involved in tumor growth and survival. Imatinib was the first KIT inhibitor to achieve clinical success, targeting both wild-type and select mutant forms of KIT, while ripretinib represents a next-generation agent engineered to overcome resistance by broadly inhibiting multiple domains of the KIT protein.

KIT Inhibitor Clinical Relevance

The limited number of approved KIT inhibitors compared to other targeted therapies reflects the complex scientific and clinical landscape surrounding KIT-driven diseases. Resistance remains a major hurdle, as secondary mutations in the KIT gene often emerge during treatment, reducing the long-term efficacy of existing inhibitors. Moreover, the therapeutic relevance of KIT inhibition is largely confined to a narrow range of malignancies, primarily systemic mastocytosis and GIST, among others, where activating KIT mutations serve as key oncogenic drivers. This constrained disease spectrum and the evolving nature of resistance mechanisms continue to challenge the broader clinical adoption of KIT-targeted therapies.

The emerging pipeline of KIT inhibitors is expanding beyond traditional indications and is now being investigated across a broader range of diseases, including CSU, NonAdvSM, AdvSM, and GvHD, among others. This shift reflects growing interest in targeting KIT-driven pathways in immune-mediated and inflammatory conditions, potentially unlocking new therapeutic avenues beyond oncology.

KIT Inhibitor Epidemiology

The KIT inhibitor epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total cases in selected indications for KIT Inhibitor, total eligible patient pool in selected indications for KIT Inhibitor, and total treated cases in selected indications for KIT Inhibitor in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034.

GIST

• According to DelveInsight's assessment, the estimated number of GIST incident cases across the 7MM reached ~17 thousand in 2024.
• In 2024, the US reported the highest number of incident GIST cases among the 7MM, accounting for ~43% of the total. This figure is expected to rise significantly in the coming years, driven by improvements in diagnostic accuracy and advancements in genetic testing.

CSU

• In 2024, there were ~600 thousand diagnosed prevalent cases of CSU in the US, a number projected to rise by 2034 due to growing disease awareness and improved diagnostic practices.
• In 2024, among the severity-specific diagnosed prevalent cases of CSU in EU4 and the UK, severe cases accounted for ~640 thousand, followed by moderate cases with ~530 thousand, and mild cases with ~250 thousand.

GvHD

• In 2024, the 7MM reported ~10 thousand cases of acute GvHD and ~42 thousand five-year prevalent cases of chronic GvHD.
• In 2024, among chronic GvHD cases by organ involvement in Germany, the oral mucosa was the most affected site, accounting for ~3 thousand cases.
• In 2024, among acute GvHD cases in Japan, Grade B[II] accounted for the highest proportion, representing ~60% of the total cases.

KIT Inhibitor Drug Chapters

The drug chapter segment of the KIT inhibitor reports includes a detailed analysis of KIT inhibitor early-, mid-, and late-stage (Phase I, Phase II and Phase III) pipeline drugs. It also helps understand the KIT inhibitor's clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug the latest news and press releases.

Marketed Drugs

Currently, the landscape of US FDA-approved KIT inhibitors remains limited, with only a few branded options available-such as ROMVIMZA (vimseltinib), AYVAKIT (avapritinib) and QINLOCK (ripretinib), among others-and no generics on the market. This lack of competition underscores the clinical complexity of targeting KIT mutations.

ROMVIMZA (vimseltinib): ONO Pharmaceutical (Deciphera Pharmaceuticals)

ROMVIMZA is an oral kinase inhibitor that selectively targets colony-stimulating factor 1 receptor (CSF1R). Preclinical studies demonstrated that vimseltinib inhibits CSF1R autophosphorylation, blocks ligand-induced signaling, and suppresses the proliferation of CSF1R-expressing cells. It is indicated for the treatment of adult patients with symptomatic TGCT when surgical resection is likely to result in significant functional impairment or severe morbidity.

In February 2025, the US FDA approved ROMVIMZA for the treatment of adult patients with symptomatic TGCT when surgical resection is expected to cause substantial functional impairment or severe morbidity. Earlier, in July 2024, the EMA accepted the marketing authorization application (MAA) for vimseltinib for the treatment of TGCT. Additionally, vimseltinib is being investigated in a Phase II clinical trial for the treatment of chronic GvHD.

QINLOCK (ripretinib): ONO Pharmaceutical (Deciphera Pharmaceuticals)

QINLOCK is a broad-spectrum tyrosine kinase inhibitor designed to target KIT and PDGFRA, including wild-type forms as well as primary and secondary mutations that commonly drive resistance in GIST. It also exhibits in vitro activity against other kinases such as PDGFRB, TIE2, VEGFR2, and BRAF.

Clinically, QINLOCK is approved for the treatment of adult patients with advanced GIST who have progressed after treatment with three or more kinase inhibitors, including imatinib, addressing a critical need in later-line settings.

QINLOCK was first approved by the US FDA in May 2020, followed by European Commission approval in November 2021.

AYVAKIT/AYVAKYT (avapritinib): Blueprint Medicines

AYVAKIT is a potent tyrosine kinase inhibitor that selectively targets key activating mutations in KIT and PDGFRA, including KIT D816V and PDGFRA D842V, with sub-nanomolar to low nanomolar potency in preclinical assays. It has demonstrated strong inhibitory activity against autophosphorylation and cell proliferation in mutant KIT-driven models and resistance-associated Platelet-derived Growth Factor Receptor Alpha (PDGFRA) mutations.

Clinically, AYVAKIT is approved for unresectable or metastatic GIST with PDGFRA exon 18 mutations (including D842V), AdvSM, and ISM, though its use is limited in patients with platelet counts below 50X10?/L.

AYVAKIT received its first US FDA approval in January 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including the D842V variant-marking it as the first approved therapy specifically targeting this mutation. Later, in September 2020, the European Commission granted conditional marketing authorization under the brand name AYVAKYT for use as monotherapy in adult patients with unresectable or metastatic GIST carrying the PDGFRA D842V mutation.

Emerging Drugs

A growing pipeline of KIT inhibitors is being investigated across a diverse set of indications beyond oncology, including CSU, multiple sclerosis, and GvHD. Prominent candidates such as barzolvolimab (CDX-0159), bezuclastinib (CGT9486), and elenestinib (BLU-263), among others reflect this expanding therapeutic interest, signaling the potential of KIT-targeted therapies in addressing immune-mediated and inflammatory conditions.

Barzolvolimab (CDX-0159): Celldex Therapeutics

Barzolvolimab is a humanized monoclonal antibody that targets the receptor tyrosine kinase KIT with high specificity, effectively inhibiting its activity. By disrupting KIT signaling, which governs mast cell development, survival, and function, barzolvolimab addresses a key driver of mast cell-mediated inflammatory responses. Its mechanism is particularly relevant in conditions like chronic urticaria, where aberrant mast cell activation plays a central role in disease pathogenesis.

Barzolvolimab is currently in various stages of clinical development for multiple inflammatory conditions, including CSU, CIndU, EoE, prurigo nodularis, and atopic dermatitis, with CSU being the most advanced, now in Phase III. In June 2025, Celldex Therapeutics presented compelling Phase II data at the EAACI Congress, demonstrating sustained complete responses and significant quality-of-life improvements at 76 weeks, well beyond the dosing period. Additional findings highlighted marked reductions in angioedema at 52 weeks, further underscoring barzolvolimab's potential as a transformative therapy in CSU.

Bezuclastinib (CGT9486): Cogent Biosciences

Bezuclastinib (CGT9486) is a highly selective and potent tyrosine kinase inhibitor targeting the KIT D816V mutation, a key driver in the majority of systemic mastocytosis cases, as well as other KIT exon 17 mutations commonly seen in advanced GIST. It is being developed as a potential treatment option for patients with NonAdvSM, AdvSM, and GIST.

In February 2025, Cogent Biosciences announced a poster presentation at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting, held from February 28 to March 3. The presentation highlighted efficacy and safety data from adult patients with Non-Advanced Systemic Mastocytosis treated with bezuclastinib in the Phase II SUMMIT trial.

In 2025, the company aims to reach several key milestones for bezuclastinib: report top-line data from the Phase II SUMMIT trial in NonAdvSM in July; announce top-line results from the Phase II APEX trial in AdvSM in the second half of the year; submit the first New Drug Application (NDA) for bezuclastinib by year-end; and deliver top-line results from the pivotal Phase III PEAK trial in patients with imatinib-resistant GIST by the end of 2025.

Elenestinib (BLU-263): Blueprint Medicines

Elenestinib (BLU-263) is an investigational, orally administered, potent, and highly selective KIT D816V inhibitor under development for the treatment of ISM and other mast cell disorders. It is engineered to deliver potency comparable to avapritinib, with reduced off-target activity and minimal central nervous system penetration. Elenestinib is currently being evaluated in the ongoing registration-enabling Phase II/III HARBOR trial in ISM. The trial consists of multiple parts, including a dose-finding Part 1, a registration-enabling Part 2, and a long-term treatment Part 3.

In December 2022, top-line 12-week data from the dose-finding Part 1 of the HARBOR trial were announced, followed by a presentation of these data at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023.

KIT Inhibitor Market Outlook

The KIT inhibitor market is undergoing rapid expansion, driven by progress in precision oncology and a growing focus on KIT mutations as key therapeutic targets. Currently approved therapies include ROMVIMZA (vimseltinib) for TGCT; AYVAKIT/AYVAKYT (avapritinib) for GIST, AdvSM, and ISM; and QINLOCK (ripretinib) for advanced GIST, among others. Emerging therapies are addressing resistance pathways, resulting in next-generation inhibitors with improved efficacy. Notable pipeline developments include barzolvolimab (CDX-0159) for CSU, CIndU, EoE, prurigo nodularis, and atopic dermatitis; bezuclastinib (CGT9486) for AdvSM, NonAdvSM, and GIST; and elenestinib (BLU-263) for ISM, among others. As research expands the use of KIT inhibitors beyond conventional oncology and combination regimens advance to counter resistance, companies offering distinct clinical advantages and navigating regulatory complexities effectively are likely to gain a competitive edge.

KIT Inhibitor Uptake

This section focuses on the uptake rate of potential approved and emerging KIT inhibitor expected to be launched in the market during 2020-2034.

KIT Inhibitor Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II and Phase I. It also analyzes key players involved in developing targeted therapeutics.

The presence of numerous drugs under different stages is expected to generate immense opportunity for KIT inhibitor market growth over the forecasted period.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for KIT inhibitor emerging therapies.

KOL Views

To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on KIT inhibitor's evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.

DelveInsight's analysts connected with 25+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM.

Their opinion helps understand and validate current and emerging therapy treatment patterns or KIT inhibitors market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Market Access and Reimbursement

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, and a descriptive overview of KIT inhibitor, explaining its mechanism, and therapies (current and emerging).
• Comprehensive insight into the Competitive Landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current landscape.
• A detailed review of the KIT inhibitor market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis expert insights/KOL views, and treatment preferences that help shape and drive the 7MM KIT inhibitor market.

KIT Inhibitor Report Insights

• KIT Inhibitor Targeted Patient Pool
• KIT Inhibitor Therapeutic Approaches
• KIT Inhibitor Pipeline Analysis
• KIT Inhibitor Market Size and Trends
• Existing and Future Market Opportunity

KIT Inhibitor Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

KIT Inhibitor Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT)

Key Questions:

• What was the KIT inhibitor total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors for this growth?
• Which drug is going to be the largest contributor in 2034?
• Which is the most lucrative market for KIT inhibitor?
• Which drug accounts for maximum sales among KIT inhibitors?
• What are the pricing variations among different geographies for approved therapies?
• How has the reimbursement landscape for KIT inhibitors evolved since the first one was approved? Do patients face any access issues driven by reimbursement decisions?
• What are the risks, burdens, and unmet needs of treatment with KIT inhibitor? What will be the growth opportunities across the 7MM for the patient population on KIT inhibitor?
• What are the key factors hampering the growth of the KIT inhibitor market?
• What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
• What key designations have been granted to the therapies for KIT inhibitor?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred therapy options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy:

• The report will help develop business strategies by understanding the latest trends and changing dynamics driving the KIT inhibitor market.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) the UK, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of indication-wise current and emerging therapies under the attribute analysis section to provide visibility around leading indications.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.