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Add to CartSystemic Mastocytosis Treatment Market
By Type;
AK-002, BLU-285, Brentuximab Vedotin, Crenolanib Besylate and OthersBy Route Of Administration;
Oral, Injectables and TopicalBy End User;
Hospitals, Specialty Clinics, Research & Academic Institutes and OthersBy Geography;
North America, Europe, Asia Pacific, Middle East & Africa and Latin America - Report Timeline (2021 - 2031)Systemic Mastocytosis Treatment Market Overview
Systemic Mastocytosis Treatment Market (USD Million)
Systemic Mastocytosis Treatment Market was valued at USD 301.26 million in the year 2024. The size of this market is expected to increase to USD 516.30 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 8.0%.
Systemic Mastocytosis Treatment Market
*Market size in USD million
CAGR 8.0 %
| Study Period | 2025 - 2031 |
|---|---|
| Base Year | 2024 |
| CAGR (%) | 8.0 % |
| Market Size (2024) | USD 301.26 Million |
| Market Size (2031) | USD 516.30 Million |
| Market Concentration | Medium |
| Report Pages | 396 |
Major Players
- Bausch Health Companies Inc
- Pfizer Inc
- Mylan NV
- Teva Pharmaceutical Industries Ltd
- Sanofi S.A.
- Johnson & Johnson
- Novartis AG
- Bayer AG
- The USA Food and Drug Administration
Market Concentration
Consolidated - Market dominated by 1 - 5 major players
Systemic Mastocytosis Treatment Market
Fragmented - Highly competitive market without dominant players
The Systemic Mastocytosis Treatment Market is evolving rapidly as hematologists and allergists increasingly adopt targeted therapies and biologics to manage chronic symptoms like itching, gastrointestinal distress, and anaphylaxis. Approximately 65% of treatment centers now combine tyrosine kinase inhibitors with mast cell stabilizers to enhance patient outcomes. This comprehensive treatment model is accelerating innovation and market expansion, enabled by close collaboration between clinical researchers and pharmaceutical developers.
Advanced Drug Formulations Enhancing Treatment Profiles
About 62% of drug manufacturers are launching enhanced treatment options featuring next-generation TKIs, antibody–drug conjugates, oral bioavailability improvements, and personalized dosing algorithms. These technological advancements improve efficacy, reduce side‑effects, and optimize patient compliance. Partnerships between biotech firms and academic centers are driving rapid development and regulatory submission pipelines.
Clinical Trials Paving the Way for Combination Regimens
Coordinated clinical studies and multicenter trials involving specialist clinics, pharma partners, and patient advocacy groups have resulted in a 59% increase in utilization of combination regimens and maintenance therapies. These cross-functional collaborations support standardized protocols, comprehensive patient monitoring, and evidence-based care—driving growth through integrated treatment pathways.
Smart & Personalized Treatment Approaches Ahead
Looking ahead, more than 70% of future treatment approaches are expected to incorporate biomarker-guided therapy selection, smart adherence-tracking systems, and next-gen oral biologics designed for less frequent dosing. These advances mark a shift toward precision management of systemic mastocytosis, offering tailored care plans that enhance long-term disease control and patient quality of life.
Systemic Mastocytosis Treatment Market Key Takeaways
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Market poised for robust growth—the global systemic mastocytosis treatment market was valued at approximately USD 310.5 million in 2023 and is projected to reach USD 482.3 million by 2032, reflecting a compound annual growth rate (CAGR) of 5.1% during the forecast period.
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North America leads regional demand&mdash>accounting for USD 127.3 million in revenue in 2023, driven by advanced healthcare infrastructure and high disease awareness.
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AK-002 dominates product segment&mdash>holding a 45% market share in 2023, highlighting its efficacy in treating indolent systemic mastocytosis.
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Oral administration preferred&mdash>with 54.2% of treatments delivered orally in 2023, due to patient convenience and adherence benefits.
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Hospital pharmacies are primary distribution channel&mdash>accounting for 40.5% of market share in 2023, reflecting their role in dispensing specialty treatments.
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Asia-Pacific exhibits rapid growth&mdash>projected to grow at a CAGR of 5.9% from 2024 to 2032, driven by improving healthcare access and rising disease awareness.
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Strategic acquisitions enhance market dynamics&mdash>Sanofi's acquisition of Blueprint Medicines in 2025 strengthens its position in rare immunology diseases, adding Ayvakit/Ayvakyt to its portfolio, the only approved treatment for advanced and indolent systemic mastocytosis in the U.S. and EU.
Systemic Mastocytosis Treatment Market Recent Developments
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In May 2023, the US Food and Drug Administration (FDA) approved AYVAKIT® (avapritinib) by Blueprint Medicines Corporation for treating indolent systemic mastocytosis (ISM). It is the first and only FDA-licensed therapy for ISM, now available to patients across the United States.
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In June 2021, the US FDA approved Ayvakit® (avapritinib) from Blueprint Medicines Corporation for adult patients with advanced systemic mastocytosis, including mast cell leukemia, systemic mastocytosis with an associated hematological neoplasm, and aggressive systemic mastocytosis.
Systemic Mastocytosis Treatment Market Segment Analysis
In this report, the Systemic Mastocytosis Treatment Market has been segmented by Type, Route Of Administration, End User and Geography.
Systemic Mastocytosis Treatment Market, Segmentation by Type
The Type segmentation captures the evolving portfolio of targeted and supportive therapies being developed for systemic mastocytosis, a rare hematologic disorder marked by aberrant mast-cell proliferation. Pipeline strategies emphasize precision medicine, particularly agents aimed at KIT and downstream signaling, while supportive agents address symptom burden and quality of life. Partnerships between biotech innovators, academic centers, and specialty clinics are accelerating trials, companion diagnostics, and post-approval evidence generation to support broader market access.
AK-002
AK-002 (anti-Siglec-8 approach) is investigated to reduce mast-cell and eosinophil activity, positioning as a potential option for refractory symptoms and overlapping inflammatory manifestations. Developers focus on clinical differentiation through symptom control, steroid-sparing potential, and durability of response. As data mature, payer engagement and real-world registries will be central to demonstrating value and informing treatment algorithms.
BLU-285
BLU-285 (avapritinib) targets mutant KIT signaling central to advanced disease biology, anchoring a wave of targeted therapies designed for higher response depth. Commercial priorities include optimized sequencing with other agents, management of adverse events, and expansion to earlier lines where appropriate. Collaboration with diagnostic providers to standardize mutation testing underpins uptake across regions and care settings.
Brentuximab Vedotin
Brentuximab Vedotin, an antibody-drug conjugate, is assessed in select mast-cell–related contexts where CD markers and comorbid hematologic profiles warrant exploration. While not a foundational therapy in most algorithms, it represents a strategic option in niche phenotypes and combination research. Evidence development focuses on biomarker-driven selection and safety management to refine its role in specialized care pathways.
Crenolanib Besylate
Crenolanib Besylate, a kinase inhibitor under investigation, targets pathways implicated in aberrant mast-cell survival and proliferation. Sponsors are prioritizing precise patient selection, pharmacodynamic readouts, and combination strategies that may extend benefit in advanced disease. Regulatory interactions, orphan-drug frameworks, and multicenter trial networks are critical to accelerate clinical development.
Others
Others encompass symptomatic treatments (e.g., antihistamines, leukotriene inhibitors), adjunctive corticosteroids, and additional targeted candidates at earlier clinical stages. This heterogeneous group remains essential for individualized regimens, bridging gaps in access and tolerability. Future growth will rely on integrated care models, digital monitoring of patient-reported outcomes, and global expansion of specialist centers.
Systemic Mastocytosis Treatment Market, Segmentation by Route Of Administration
The Route Of Administration segmentation reflects practical considerations that shape adherence, monitoring intensity, and resource planning across health systems. Oral targeted agents enable decentralized care and long-term management, while injectables are common in controlled settings for induction or combination strategies. Topical formulations complement systemic therapy for cutaneous symptoms, rounding out comprehensive, stepwise disease management.
Oral
Oral therapies support sustained exposure to targeted agents with convenient dosing that favors adherence and quality of life. Market expansion is tied to mutation testing workflows, treatment sequencing guidance, and proactive toxicity management. Payers and providers emphasize outpatient pathways, remote monitoring, and patient education to optimize long-term outcomes.
Injectables
Injectables are administered in specialty clinics and hospitals where closer observation, dose titration, or infusion infrastructure is required. Their role spans induction, rescue, and select combination regimens informed by disease severity and comorbidities. Vendor strategies center on administration support programs, cold-chain reliability, and real-world evidence to validate cost-effectiveness.
Topical
Topical approaches provide localized relief for cutaneous manifestations that significantly impact daily functioning. They are frequently integrated with systemic therapy to achieve multimodal symptom control and flare prevention. Innovation focuses on patient-friendly vehicles, safety in long-term use, and alignment with dermatology-hematology co-management protocols.
Systemic Mastocytosis Treatment Market, Segmentation by End User
The End User segmentation underscores where diagnostic capabilities, multidisciplinary expertise, and supportive services converge to deliver specialized care. Hospitals anchor intensive management and access to advanced therapeutics, while specialty clinics streamline follow-up and chronic care. Academic & research institutes drive trial enrollment and biomarker discovery, as pharma-biotech partnerships expand access and education across networks.
Hospitals
Hospitals manage complex and advanced cases requiring rapid diagnostics, inpatient stabilization, and integrated subspecialty input. They coordinate molecular testing, initiate targeted therapy, and manage complications, ensuring guideline-consistent care. Hospital-based centers also serve as referral hubs for regional networks, strengthening care continuity from diagnosis through survivorship.
Specialty Clinics
Specialty Clinics offer concentrated expertise in mast-cell disorders, enabling efficient monitoring, dose optimization, and adverse-event management. Their nimble operations support timely access to symptom-directed therapies and patient education. Collaboration with pharmacies and diagnostic labs enhances care coordination and real-world data capture.
Research & Academic Institutes
Research & Academic Institutes underpin innovation through translational studies, registries, and investigator-initiated trials that refine biomarker-guided care. They shape treatment algorithms, publish practice-changing evidence, and train the next generation of specialists. International consortia accelerate enrollment in rare disease studies and promote standardization of outcome measures.
Others
Others include comprehensive care centers, integrated delivery networks, and qualified community practices participating in shared-care models. These settings extend access to specialist oversight via telehealth, e-consults, and coordinated referral pathways. Growth depends on payer alignment, value-based contracts, and scalable patient-support programs.
Systemic Mastocytosis Treatment Market, Segmentation by Geography
In this report, the Systemic Mastocytosis Treatment Market has been segmented by Geography into five regions: North America, Europe, Asia Pacific, Middle East and Africa and Latin America.
Regions and Countries Analyzed in this Report
North America
North America benefits from established rare-disease infrastructure, comprehensive molecular diagnostics, and rapid access pathways for innovative therapies. Academic-industry collaborations and robust patient advocacy groups strengthen clinical trial enrollment and real-world evidence generation. Payer frameworks increasingly recognize the value of targeted treatments, supporting sustainable adoption in specialty centers.
Europe
Europe advances coordinated care through reference networks, harmonized orphan-drug policies, and national registries that standardize outcomes. Emphasis on health-technology assessment and budget impact modeling shapes market access and long-term planning. Cross-border research initiatives and clinician training programs further expand specialist capacity and equitable access.
Asia Pacific
Asia Pacific is expanding diagnostic capabilities and specialist clinics in major urban hubs, improving early identification and referral. Regional opportunities include local manufacturing partnerships, clinical trial participation, and telemedicine to extend expertise to underserved areas. As reimbursement frameworks evolve, stakeholders prioritize affordability and patient-support services to increase uptake.
Middle East & Africa
Middle East & Africa show gradual growth driven by investments in tertiary care, adoption of molecular testing, and partnerships with international centers. Capacity building through clinician education and shared-care models supports earlier diagnosis and safer therapy administration. Expanding access programs and regional procurement initiatives are key to improving treatment availability.
Latin America
Latin America focuses on strengthening referral pathways, enhancing diagnostic access, and negotiating affordability through public tenders and patient-assistance programs. Collaborations with academic institutions foster registry development and local evidence to guide policy. As specialized centers scale, integrated pathways will support consistent follow-up and long-term outcomes.
Market Trends
This report provides an in depth analysis of various factors that impact the dynamics of Global Systemic Mastocytosis Treatment Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.
Drivers, Restraints and Opportunity Analysis
Drivers
- Therapeutic interventions
- Tyrosine kinase inhibitors
- Monoclonal antibodies
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Personalized treatment -Personalized treatment in the global systemic mastocytosis market represents a significant advancement, offering tailored approaches that consider individual patient characteristics, disease subtype, and treatment response. This approach acknowledges the heterogeneity of systemic mastocytosis and aims to optimize therapeutic outcomes while minimizing adverse effects. Several key elements contribute to personalized treatment strategies in this market: Advances in molecular profiling techniques have enabled a deeper understanding of the genetic alterations driving systemic mastocytosis. Identifying specific mutations, such as KIT D816V, helps categorize patients into distinct subgroups with varying disease severity and treatment responses. Molecular profiling informs treatment decisions, guiding the selection of targeted therapies that address the underlying molecular drivers of the disease.
Biomarker assessment plays a crucial role in personalized treatment by providing insights into disease activity, prognosis, and treatment response. Biomarkers such as serum tryptase levels, histamine metabolites, and cytokine profiles help monitor disease progression and therapeutic efficacy. Regular monitoring of biomarkers allows healthcare providers to adjust treatment strategies accordingly, optimizing patient outcomes and minimizing the risk of disease complications. The emergence of targeted therapies represents a paradigm shift in systemic mastocytosis treatment, offering precise interventions that selectively inhibit aberrant signaling pathways implicated in disease pathogenesis. Tyrosine kinase inhibitors, such as midostaurin and avapritinib, target the KIT receptor and have shown efficacy in patients with advanced forms of the disease. Other targeted agents, including masitinib and ripretinib, are under investigation, highlighting the expanding therapeutic options available to patients.
Immunomodulatory approaches are being explored as adjunctive therapies in systemic mastocytosis, aiming to modulate the dysregulated immune response associated with mast cell activation. Immunomodulators such as interferon-alpha and monoclonal antibodies targeting immune checkpoints hold promise in modulating the inflammatory milieu and restoring immune homeostasis. These therapies may complement existing treatments, particularly in patients with refractory disease or those intolerant to conventional therapies. Personalized treatment extends beyond pharmacological interventions to encompass holistic patient-centric care. Multidisciplinary care teams collaborate to address the diverse needs of patients, including symptom management, psychosocial support, and lifestyle modifications. Shared decision-making empowers patients to actively participate in their treatment journey, fostering a collaborative therapeutic alliance and improving treatment adherence.
Restraints:
- Rarity of the disease
- Diagnostic complexities
- Limited treatment options
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High therapy costs -The Global Systemic Mastocytosis Treatment Market faces challenges related to high therapy costs, which can pose significant barriers to access for patients seeking optimal care. The complex nature of systemic mastocytosis often necessitates a multidisciplinary treatment approach involving various medications, therapies, and supportive care measures, leading to substantial financial burdens for patients and healthcare systems alike. One of the primary factors contributing to high therapy costs is the limited availability of targeted treatments specifically designed for systemic mastocytosis. Many of the medications used in the treatment regimen, such as tyrosine kinase inhibitors and mast-cell stabilizers, may be expensive due to factors such as research and development costs, manufacturing expenses, and patent protections. As a result, patients may face challenges in accessing these essential therapies, particularly in regions with limited healthcare resources or inadequate insurance coverage.
The chronic and often lifelong nature of systemic mastocytosis requires ongoing management and monitoring, leading to cumulative healthcare expenditures over time. This includes regular medical consultations, laboratory tests, imaging studies, and other diagnostic procedures necessary for disease monitoring and treatment adjustments. Additionally, the need for emergency medications, such as epinephrine, further adds to the overall therapy costs, especially for patients at risk of severe allergic reactions or anaphylaxis. The high therapy costs associated with systemic mastocytosis treatment can have detrimental effects on patients' financial well-being, potentially leading to treatment non-adherence, delayed care-seeking behavior, and compromised health outcomes. Moreover, these costs can strain healthcare systems, exacerbating existing disparities in access to quality care.
The issue of high therapy costs in the systemic mastocytosis treatment market requires collaborative efforts from various stakeholders, including pharmaceutical companies, policymakers, healthcare providers, patient advocacy groups, and insurers. Strategies such as price negotiation, reimbursement reform, patient assistance programs, and research initiatives aimed at developing more affordable treatment options are essential for ensuring equitable access to effective therapies for all patients affected by systemic mastocytosis. By addressing the financial barriers to care, stakeholders can improve treatment adherence, enhance patient outcomes, and alleviate the burden of systemic mastocytosis on individuals and healthcare systems worldwide.
Opportunities:
- Genetic Testing
- Healthcare Infrastructure
- Patient Advocacy
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Non-profit Organizations -Non-profit organizations play a crucial role in the global systemic mastocytosis treatment market by providing support, advocacy, education, and funding for research initiatives. These organizations are dedicated to improving the lives of individuals affected by systemic mastocytosis and their families. Here are some notable non-profit organizations focused on systemic mastocytosis: This organization is one of the leading advocates for individuals with mast cell diseases, including systemic mastocytosis. The Mastocytosis Society provides support and resources for patients, caregivers, and healthcare professionals. They also fund research projects aimed at advancing the understanding and treatment of mast cell disorders.
The Mast Cell Disease Society is committed to raising awareness about mast cell diseases, including systemic mastocytosis, and supporting research efforts to find better treatments and ultimately a cure. They offer educational materials, support groups, and advocacy resources for individuals living with mast cell diseases. While not specific to systemic mastocytosis, the RDCRN supports research on rare diseases, including mast cell disorders. They facilitate collaboration among researchers, clinicians, and patient advocacy groups to accelerate the development of new therapies and improve patient outcomes.
This organization focuses on providing education, advocacy, and support for individuals with allergies, asthma, and related conditions, including mast cell disorders. They offer resources to help patients and caregivers better understand their condition and navigate the healthcare system. Global Genes is a non-profit organization dedicated to advocating for rare disease patients worldwide. They provide support and resources for patients, caregivers, and advocacy groups, including those focused on systemic mastocytosis. Global Genes also hosts events and initiatives to raise awareness and promote collaboration in the rare disease community.
Systemic Mastocytosis Treatment Market Competitive Landscape Analysis
Systemic Mastocytosis Treatment Market presents a competitive landscape shaped by leading pharmaceutical firms, biotechnology innovators, and specialty healthcare providers. The market reflects increasing collaboration, merger, and partnerships driving enhanced treatment modalities. With more than 50% of participants investing in technological advancements, this segment shows sustained growth potential across targeted therapeutic areas.
Market Structure and Concentration
The market displays a moderately concentrated structure, with established players holding over 60% of the total share while emerging firms rapidly scale capabilities. Strategic alliances and innovation pipelines have increased competitive intensity, enabling faster expansion into specialty care segments. The sector’s evolving strategies indicate a shift toward patient-centric therapies and advanced diagnostics.
Brand and Channel Strategies
Major players are focusing on brand differentiation through diversified treatment platforms and global licensing agreements. Over 45% of companies emphasize multi-channel outreach integrating partnerships with healthcare providers and digital platforms. This coordinated approach supports growth and enhances market visibility while improving access to specialized therapies for systemic mastocytosis treatment.
Innovation Drivers and Technological Advancements
With nearly 70% of market participants prioritizing research and innovation, new-generation therapies and advanced diagnostic tools are reshaping the competitive environment. Strategic collaboration with research institutions accelerates technological advancements, including biomarker-based solutions. Such integrated efforts underpin long-term growth and signal a strong focus on targeted, efficient treatment pathways.
Regional Momentum and Expansion
North America and Europe collectively account for about 65% of market share, with emerging regions experiencing faster expansion. Companies are adopting localized strategies, joint ventures, and partnerships to meet regional demand. This balanced approach accelerates growth while reinforcing a global supply footprint and addressing unmet clinical needs in systemic mastocytosis treatment.
Future Outlook
The future of the market shows more than 55% of stakeholders planning sustained investment in digital health platforms, advanced therapeutics, and expanded distribution channels. Continuous innovation, strategic collaboration, and targeted expansion are expected to strengthen competitive positions. This forward-looking strategy highlights robust potential for long-term growth in systemic mastocytosis treatment solutions.
Key players in Systemic Mastocytosis Treatment Market include.
- Blueprint Medicines
- Novartis
- AB Science
- Deciphera Pharmaceuticals
- AbbVie
- Bristol-Myers Squibb
- Arog Pharmaceuticals
- Medscape
- Mylan
- Sanofi
- Patara Pharma
- Seagen
- GT Biopharma
- MacroGenics
- Cogent Biosciences
In this report, the profile of each market player provides following information:
- Market Share Analysis
- Company Overview and Product Portfolio
- Key Developments
- Financial Overview
- Strategies
- Company SWOT Analysis
- Introduction
- Research Objectives and Assumptions
- Research Methodology
- Abbreviations
- Market Definition & Study Scope
- Executive Summary
- Market Snapshot, By Type
- Market Snapshot, By Route Of Administration
- Market Snapshot, By End User
- Market Snapshot, By Region
- Systemic Mastocytosis Treatment Market Dynamics
- Drivers, Restraints and Opportunities
- Drivers
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Therapeutic interventions
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Tyrosine kinase inhibitors
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Monoclonal antibodies
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Personalized treatment
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- Restraints
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Rarity of the disease
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Diagnostic complexities
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Limited treatment options
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High therapy costs
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- Opportunities
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Genetic Testing
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Healthcare Infrastructure
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Patient Advocacy
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Non-profit Organizations
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- Drivers
- PEST Analysis
- Political Analysis
- Economic Analysis
- Social Analysis
- Technological Analysis
- Porter's Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- Drivers, Restraints and Opportunities
- Market Segmentation
- Systemic Mastocytosis Treatment Market, By Type, 2021 - 2031 (USD Million)
- AK-002
- BLU-285
- Brentuximab Vedotin
- Crenolanib Besylate
- Others
- Systemic Mastocytosis Treatment Market, By Route Of Administration, 2021 - 2031 (USD Million)
- Oral
- Injectables
- Topical
- Systemic Mastocytosis Treatment Market, By End User, 2021 - 2031 (USD Million)
- Hospitals
- Specialty Clinics
- Research & Academic Institutes
- Others
- Systemic Mastocytosis Treatment Market, By Geography, 2021 - 2031 (USD Million)
- North America
- United States
- Canada
- Europe
- Germany
- United Kingdom
- France
- Italy
- Spain
- Nordic
- Benelux
- Rest of Europe
- Asia Pacific
- Japan
- China
- India
- Australia & New Zealand
- South Korea
- ASEAN (Association of South East Asian Countries)
- Rest of Asia Pacific
- Middle East & Africa
- GCC
- Israel
- South Africa
- Rest of Middle East & Africa
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- North America
- Systemic Mastocytosis Treatment Market, By Type, 2021 - 2031 (USD Million)
- Competitive Landscape
- Company Profiles
- Blueprint Medicines
- Novartis
- AB Science
- Deciphera Pharmaceuticals
- AbbVie
- Bristol-Myers Squibb
- Arog Pharmaceuticals
- Medscape
- Mylan
- Sanofi
- Patara Pharma
- Seagen
- GT Biopharma
- MacroGenics
- Cogent Biosciences
- Company Profiles
- Analyst Views
- Future Outlook of the Market