Chronic Myelomonocytic Leukemia (CMML) Market

Key Highlights

• Chronic myelomonocytic leukemia (CMML) is a rare, clonal hematopoietic stem cell disorder characterized by the presence of sustained (>3 months) peripheral blood (PB) monocytosis (≥1 × 109/L; monocytes ≥10% of white blood cell count) along with dysplastic features in the bone marrow (BM).
• CMML can develop into acute myeloid leukemia (AML) if the number of blast cells in the blood rises above 20%. This is called transformation, which happens in 15–30% of CMML patients.
• Overall, CMML patients have a median overall survival of 20-40 months with highly heterogeneous subgroups contained within the overarching diagnosis.
• The market is anticipated to witness a substantial positive shift owing to better uptake of existing drugs, the expected market launch therapies, and raised awareness.
• The United States accounts for the largest market size of Chronic myelomonocytic leukemia, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
• In June 2024, Immune-Onc Therapeutics announced positive interim Phase Ib expansion cohort data for IO-202 in chronic myelomonocytic leukemia (CMML) patients at the 2024 European Hematology Association Annual Meeting in Madrid, Spain.
• In February 2024, Immune-Onc Therapeutics announced that the FDA granted an Orphan Drug Designation for IO-202 for treating chronic myelomonocytic leukemia (CMML). IO-202 had previously received Fast Track Designation for relapsed or refractory CMML in 2023, and Fast Track and Orphan Drug Designations for acute myeloid leukemia (AML) in 2022 and 2020, respectively.
• The approved therapies for CMML include VIDAZA, DACOGEN, INQOVI, and others.
• The chronic myelomonocytic leukemia pipeline possesses potential drugs like SL-401 and IO-202.

DelveInsight's “Chronic Myelomonocytic Leukemia – Market Insights, Epidemiology and Market Forecast – 2034” report delivers an in-depth understanding of Chronic Myelomonocytic Leukemia, historical and forecasted epidemiology as well as the Chronic Myelomonocytic Leukemia market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Chronic myelomonocytic leukemia market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM chronic myelomonocytic leukemia market size from 2020 to 2034. The report also covers current chronic myelomonocytic leukemia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.

Geography Covered

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

Chronic Myelomonocytic Leukemia (CMML) Understanding and Treatment Algorithm

Chronic Myelomonocytic Leukemia Overview, Country-Specific Treatment Guidelines and Diagnosis

Chronic myelomonocytic leukemia (CMML) is a rare hematopoietic stem cell disorder characterized by sustained peripheral blood monocytosis (≥1 × 10^9/L, ≥10% of white blood cell count) and dysplastic features in the bone marrow. Classified by the WHO as a myelodysplastic syndrome/myeloproliferative neoplasm, CMML is categorized into three types based on blast cell percentages in blood and bone marrow: Type 0 (<2% blasts), Type 1 (2-4% blasts or 5-9% in marrow), and Type 2 (5-19% blasts or 10-19% in marrow). Risk factors include advanced age, male sex, prior chemotherapy or radiotherapy, and genetic mutations like TET2, RAS, and ASXL1. Symptoms may include enlarged spleen, anemia, infections, skin abnormalities, and pleural effusion. 

Diagnosis involves multiple tests including CBC, blood smear, bone marrow biopsy, and cytogenetic testing for DNA abnormalities.

Further details related to country-based variations in diagnosis are provided in the report.

Chronic Myelomonocytic Leukemia Treatment

Treatment for Chronic Myelomonocytic Leukemia (CMML) typically involves a combination of supportive care, chemotherapy, and other therapies. For patients with high white blood cell counts, hydroxyurea can help lower monocyte counts and reduce the need for transfusions. Chemotherapy, including hypomethylating agents like azacitidine and decitabine, is often used to control symptoms and slow disease progression. In some cases, stem cell transplantation may be considered, particularly for younger patients with higher-risk disease. Additionally, supportive care measures such as transfusions, blood cell growth factors, and antibiotics are used to manage symptoms and prevent complications. The choice of treatment depends on factors such as the patient's age, overall health, and the severity of the disease, to improve quality of life and manage the disease effectively.

Chronic Myelomonocytic Leukemia (CMML) Epidemiology

The chronic myelomonocytic leukemia epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. Chronic myelomonocytic leukemia epidemiology is segmented with detailed insights into incident cases, type-specific cases, age-specific cases, gender-specific, mutation-specific cases, and treated cases of chronic myelomonocytic leukemia.

• Genetic mutations are common in CMML patients, with TET2 observed in ~60%, followed by ASXL1.
• DelveInsight's estimation indicates that the median age at diagnosis for CMML typically falls between 65 and 75 years.
• High-risk CMML affects a higher proportion of males than females.

Chronic Myelomonocytic Leukemia (CMML) Drug Chapter 

The drug chapter segment of the Chronic Myelomonocytic Leukemia report encloses a detailed analysis of Chronic Myelomonocytic Leukemia marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the Chronic Myelomonocytic Leukemia pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations. 

Marketed Drugs

INQOVI: Otsuka Pharmaceutical

INQOVI is a combination of decitabine and cedazuridine, indicated for the treatment of adult patients with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML).

Decitabine is a nucleoside metabolic inhibitor that, upon phosphorylation and incorporation into DNA, inhibits DNA methyltransferase. This action leads to DNA hypomethylation, which can trigger cellular differentiation or apoptosis in cancer cells. Cedazuridine, a cytidine deaminase inhibitor, enhances decitabine's systemic exposure by preventing its degradation by cytidine deaminase, thereby improving its effectiveness in treatment.

Note: Detailed current therapies assessment will be provided in the full report of Chronic Myelomonocytic Leukemia

Emerging Drugs

TAGRAXOFUSP (SL-401): Stemline Therapeutics

TAGRAXOFUSP is a novel targeted therapy directed to the IL-3 receptor α (CD123), a target present in a wide range of malignancies. It comprises human IL-3 recombinantly fused to a truncated diphtheria toxin (DT) payload engineered such that IL-3 replaces the native DT receptor-binding domain. The IL-3 domain of TAGRAXOFUSP directs the cytotoxic DT payload to cells expressing CD123. Upon internalization, TAGRAXOFUSP irreversibly inhibits protein synthesis and induces apoptosis of the target cell. 

TAGRAXOFUSP is now being investigated in Phase II (NCT02268253) for the treatment of chronic myelomonocytic leukemia.

IO-202: Immune-Onc Therapeutics

IO-202 is a first-in-class antagonist antibody targeting LILRB4, a myeloid checkpoint inhibitor, in Phase 1 clinical development for the treatment of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), and solid tumors. It blocks the interaction of LILRB4 with multiple ligands, including ApoE and fibronectin, and has broad potential as an immunotherapy in both blood cancers and solid tumors. IO-202 has shown promising results in preclinical studies, converting a "don't kill me" to a "kill me" signal by activating T cell killing and reducing leukemia tissue infiltration. It is currently being evaluated in clinical trials as a monotherapy and in combination with other agents, including azacitidine and venetoclax.

In June 2024, Immune-Onc Therapeutics announced that it would present additional positive interim Phase 1b expansion cohort data for IO-202 in patients with chronic myelomonocytic leukemia (CMML) at the 2024 European Hematology Association (EHA) Annual Meeting, held virtually and in Madrid, Spain from June 13 to 16.

Note: Detailed emerging therapies assessment will be provided in the final report.

Chronic Myelomonocytic Leukemia (CMML) Market Outlook

Key players, such as Novartis, Immune-Onc Therapeutics, Stemline Therapeutics, and others are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of chronic myelomonocytic leukemia.

• Emerging therapies currently in development offer promising new treatment strategies with novel mechanisms of action for patients with chronic myelomonocytic leukemia (CMML).
• Surgery is rarely used for CMML treatment. Growth factors like EPO (erythropoietin) or PROCRIT are used to enhance red blood cell production, and OPRELVEKIN to stimulate platelet production as needed.
• The market size growth is driven by improved treatment options and survival rates, along with rising CMML incidence in the elderly, reflecting global population aging.

Chronic Myelomonocytic Leukemia (CMML) Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024–2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake. 

Further detailed analysis of emerging therapies drug uptake in the report

Chronic Myelomonocytic Leukemia (CMML) Activities

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

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Chronic Myelomonocytic Leukemia emerging therapies. 

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.

DelveInsight’s analysts connected with 40+ KOLs to gather insights; however, interviews were conducted with 25+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging treatment patterns of Chronic Myelomonocytic Leukemia. 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 and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. 

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided. 

Market Access and Reimbursement

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used 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, descriptive overview of Chronic Myelomonocytic Leukemia, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country-specific treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the Chronic Myelomonocytic Leukemia 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 and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Chronic Myelomonocytic Leukemia market.

Chronic Myelomonocytic Leukemia Report Insights

• Patient Population
• Therapeutic Approaches
• Chronic Myelomonocytic Leukemia Pipeline Analysis
• Chronic Myelomonocytic Leukemia Market Size and Trends
• Existing and future Market Opportunity 

Chronic Myelomonocytic Leukemia Report Key Strengths

• Eleven Years Forecast
• 7MM Coverage 
• Chronic Myelomonocytic Leukemia Epidemiology Segmentation
• Inclusion of Country specific treatment guidelines
• KOL’s feedback on approved and emerging therapies 
• Key Cross Competition 
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Chronic Myelomonocytic Leukemia Report Assessment

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

FAQs

• What is the growth rate of the 7MM Chronic Myelomonocytic Leukemia treatment market?
• What was the Chronic Myelomonocytic Leukemia 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/key catalysts for this growth?
• Is there any unexplored patient setting that can open the window for growth in the future?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends? Although multiple expert guidelines recommend testing for targetable mutations before therapy initiation, why do barriers to testing remain high?
• What are the current and emerging options for the treatment of Chronic Myelomonocytic Leukemia? 
• How many companies are developing therapies for the treatment of Chronic Myelomonocytic Leukemia?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies? 
• Patient/physician acceptability in terms of preferred treatment 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 in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Chronic Myelomonocytic Leukemia Market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• 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 United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• 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.