The chronic myelomonocytic leukemia market is estimated to be valued at USD 950.2 Mn in 2024 and is expected to reach USD 1462.5 Mn by 2031, growing at a compound annual growth rate (CAGR) of 6.4% from 2024 to 2031. According to recent studies, prevalence of chronic myelomonocytic leukemia has increased by 1.5% annually over the past 5 years. Furthermore, ongoing R&D and clinical trials for novel combination therapies and targeted drugs for treatment of CML indicate promising growth opportunities for players in this market.

Market Driver - A Rise in the Number of CMML Cases Globally is Propelling Market Growth

As the global population grows older, more and more individuals are entering risk categories associated with chronic myelomonocytic leukemia (CMML). Advanced age is the strongest predisposing element, with the median age of CMML diagnosis being around 70 years. Furthermore, improvements in diagnostic abilities have enabled more accurate identification and classification of CMML cases over time. These factors combined have led to a steady rise in the number of people being diagnosed with CMML year after year.

A sizeable portion of CMML patients also tend to develop acute myeloid leukemia (AML) as the disease progresses. This translates to a continual increase in the pool of people requiring treatment. The growing prevalence of associated conditions such as myelodysplastic syndromes (MDS) has additionally expanded the potential market as CMML sometimes arises from MDS or can transform into it.

Geographic expansion of healthcare access in developing regions is another element amplifying the patient population trends. Underdeveloped areas that previously had limited capacity to diagnose rarer disorders are now better equipped to detect CMML. The advanced industrialized markets will maintain their high diagnosis rates while emerging countries contribute increasingly to global incidence.

Market Driver - Development of Novel Therapies and Drugs Enhancing Patient Outcomes

Considerable research activities in recent times have generated exciting novel treatment options for CMML patients. Though still an orphan disease, heightened awareness about CMML sparked focused drug development initiatives. New molecular entities directly targeting identified pathologies are entering the market enriching therapeutic choices.

A prime instance is the United States FDA approval of the first CD33 target drug gilteritinib in 2018. CD33 is highly expressed on CMML cells and provides a viable target for differentiation and apoptosis induction. Clinical trial results exhibited gilteritinib's meaningful efficacy especially for advanced or refractory disease. It represented a critical breakthrough improving outcomes beyond conventional care.

Another significant milestone was the approval of decitabine for CMML in Europe in 2021 based on results demonstrating prolonged overall survival compared to best supportive care. Decitabine is a DNA methyltransferase inhibitor interfering with DNA methylation to stimulate cell suicide. Its new CMML indication expanded options beyond a sole previous approval for higher-risk MDS.

Such innovations are directly enhancing patient outcomes through longer survival times and fewer symptoms – a central driver positively mobilizing CMML market forces. Novel therapeutics present expanding options for effectively managing disease manifestations at various stages. This stimulates provider and patient confidence in available treatments.

Market Challenge - Expensive Therapies Limit Accessibility for Patients, Especially in Low-Income Regions

One of the major challenges facing the chronic myelomonocytic leukemia market is the high cost of available therapies. CML therapies such as chemotherapy and bone marrow transplants require lengthy and expensive hospital stays.

Additionally, newer targeted therapies such as midostaurin and quizartinib that have improved survival outcomes come with six-figure annual price tags. Such expensive treatments put them out of reach for many patients, especially those in low and middle-income countries without strong public healthcare systems or insurance coverage.

The affordability issue is a major barrier to access that slows clinical adoption and leaves patients with limited or no options for effective care. To expand access, new pricing and healthcare delivery models may be needed that incorporate income-based cost control strategies without negatively impacting the incentives for continued R&D investment in better CML treatments.

Market Opportunity - Growing Investment in R&D for Innovative and Targeted Therapies

There is a growing opportunity in the chronic myelomonocytic leukemia market driven by increasing investment in research and development of novel, targeted therapy approaches. With a better understanding of CML pathophysiology, many biotechnology and pharmaceutical companies are exploring more precisely targeted agents such as small molecule inhibitors, antibody therapies, immunotherapies and gene therapies.

Several high-potential candidates are currently in clinical trials, with potential to improve response rates, long-term outcomes and safety profiles over existing standards of care. With large patient populations and high unmet need, CML represents an attractive therapeutic area for R&D investment.

Further successful clinical validation of innovative pipeline therapies could lead to multiple new product approvals and blockbuster sales potential in the coming years.

CMML usually follows a step-wise treatment approach based on disease severity and symptoms. For low-risk early stage CMML with mild symptoms, prescribers often recommend close monitoring only.

As the disease progresses to an intermediate stage with worsening blood cell counts and enlarged spleen/liver, first-line treatments are initiated. Hydroxyurea (Hydrea) is commonly prescribed to control fast-growing white blood cells and reduce related symptoms. Prescribers may also opt for hypomethylating agents like azacitidine (Vidaza) or decitabine (Dacogen) which are well-tolerated oral/IV therapies shown to extend survival.

For high-risk late stage CMML with severe organ involvement, second-line therapies become crucial. Many prescribers prefer clinical trial enrollment to evaluate new targeted therapies like the FLT3 inhibitor gilteritinib (Xospata) which has shown promising remissions. For transplant-eligible patients, stem cell transplantation remains the only potential cure but is reserved only for younger and healthier cases due to risks.

Prescriber preferences are also impacted by patient comorbidities, supporting care, and insurance approvals for costly drugs. Oral therapies like hydroxyurea are preferred for easier adherence in older comorbid CMML cases versus IV drugs requiring clinic visits. Understanding both clinical factors and real-world treatment considerations is important for effectively addressing prescriber needs.

CMML is broadly classified into low-risk and high-risk disease based on white blood cell count and percentage of blasts in the blood and bone marrow. For low-risk early stage CMML, treatment focuses on managing symptoms through hydroxyurea or immunosuppressive therapy with glucocorticoids like prednisone.

For higher risk disease or when the disease progresses, the preferred first-line treatment is a combination of the DNA methyltransferase inhibitor azacitidine (Vidaza) and the histone deacetylase inhibitor decitabine (Dacogen). This combination helps reprogram faulty DNA and induces cell cycle arrest and cell death in the MDS and leukemia cells. It has shown better response rates and survival benefits over the single agents alone for CMML.

For those who do not respond to hypomethylating agents or have relapsed after initial response, the primary treatment is a stem cell transplant if the patient is fit enough. The stem cells help replace the defective bone marrow with healthy cells. Clinical trials are also investigating drugs like the FLT3 inhibitor gilteritinib (Xospata) and the BCL2 inhibitor venetoclax (Venclexta) for relapsed/refractory CMML.

The choice of treatment depends on disease risk, patient fitness, prior response, and availability of stem cell donor.

Strategy 1: Acquisitions & collaborations to gain access to novel drug candidates

Companies have focused on acquiring promising drug development programs through mergers and acquisitions. For example, in 2021, Otsuka Pharmaceutical acquired Astex Pharmaceuticals, gaining access to guadecitabine, an experimental drug for CMML and other myeloid malignancies. Such deals strengthen pipelines and diversify portfolios.

Strategy 2: Expanding indications of existing drugs

Given the rarity and heterogeneity of CMML, players aim to broaden the labeling of approved drugs. In 2018, a pivotal Phase 3 trial showed Vidaza (azacitidine) improved overall survival in CMML compared to best available therapy, leading the FDA to approve its use.

Strategy 3: Fast track designation and accelerated approval pathways

Companies pursue expedited regulatory designations when possible. In 2014, Dartmouth's Thompson Center received an orphan drug designation for quercetin for CMML. This allowed 7 years of market exclusivity upon approval, incentivizing investment.

Strategy 4: Partnering for late-stage development and commercialization

Given the small patient population and high costs, partners may be sought. In 2017, MEI Pharma out-licensed zandelisib to Kyowa Kirin for a $55M upfront payment and up to $630M in milestones, with Kyowa Kirin leading Phase 3 development and commercialization in certain territories.

Insights, By Treatment: Chemotherapy Dominates CMML Treatment Due to Broad Applicability

In terms of treatment, chemotherapy is estimated to account for 40.7% share of the chronic myelomonocytic leukemia market in 2024, owning to its broad applicability across patient populations. Chemotherapy, consisting primarily of cytarabine and hydroxyurea, offers clinicians a treatment option for a wide range of CMML cases from early stage to advanced. Unlike targeted therapies or stem cell transplants which are limited to certain patients based on risk factors, chemotherapy can be utilized as a first-line or subsequent treatment regardless of disease characteristics.

This flexibility allows chemotherapy to effectively treat the bulk of CMML cases at some point during their care. Cytarabine in particular provides a standardized regimen that oncologists are highly familiar with for initiating treatment in low-risk patients not yet needing a more aggressive approach. For higher-risk or relapsed cases that fail other options, hydroxyurea offers a palliative treatment that can control symptoms and prolong survival time. The stability and adjustability of chemotherapy dosing also makes it amenable for older or frailer CMML patients who may not tolerate other options well.

Due to its broad applicability across the diverse CMML patient population, chemotherapy persists as the anchor of treatment strategies. It gives physicians a reliable standard of care to begin management for most newly diagnosed cases before advancing to subsequent lines of targeted therapies or stem cell transplantation if needed.

Insights, By Distribution Channel: Hospital Pharmacies Lead CMML Drug Distribution

In terms of distribution channel, hospital pharmacies are likely to hold 60.3% share of the CMML market in 2024, due to their central role in dispensing and managing treatment regimens. As CMML often requires complex multi-drug chemotherapy or targeted therapy combination regimens administered via intravenous infusion or injection, hospitals serve as the primary care setting.

Related services like teaching patients or caregivers how to properly administer take-home injections or handle drug availability are also provided. The need for robust clinical pharmacy support further increases for stem cell transplant patients who may experience complications. Close monitoring during inpatient admission is required to rapidly adjust or intervene in treatment plans based on lab results and side effect profiles. Due to requirements for specialized handling, preparation, and medication reconciliation across multiple providers, hospital pharmacies are the hub for distributing CMML medications.

Outpatient needs are also often directed through hospital pharmacies with retail locations onsite. This streamlines continuity of care as patients transitions between inpatient and outpatient settings. The comprehensive services provided by hospital pharmacy departments have established them as the central point of dispensing for complicated CMML regimens from diagnosis onward. Their expertise in cellular therapy products and supportive care make them vital partners in combating this high-risk leukemia subtype.

• Approximately 1,500 new cases of CMML are diagnosed annually in the United States, highlighting the need for effective treatment options and increased awareness of the disease.
• CMML is characterized by sustained peripheral blood monocytosis and dysplastic bone marrow features. CMML is classified into three types based on blast cell percentages.

The major players operating in the Chronic Myelomonocytic Leukemia Market include Immune-Onc Therapeutics, Stemline Therapeutics, Otsuka Pharmaceutical, Novartis, Merck Sharp & Dohme, Novartis AG, Celgene Corporation (now part of Bristol Myers Squibb), AbbVie Inc., Jazz Pharmaceuticals, and Takeda Pharmaceutical Company Limited.

• In June 2024, Immune-Onc Therapeutics announced positive Phase Ib expansion cohort data for their drug IO-202 in Chronic Myelomonocytic Leukemia (CMML) patients at the European Hematology Association (EHA) Annual Meeting. The data presented highlighted that IO-202, in combination with azacitidine, showed promising results, including early and sustained complete remissions among CMML patients. This outcome suggests that IO-202 could become a valuable addition to frontline therapy for CMML patients, improving upon the limited treatment options currently available.
• In February 2024, Immune-Onc Therapeutics received FDA Orphan Drug Designation for IO-202 for treating Chronic Myelomonocytic Leukemia (CMML). IO-202 is a first-in-class humanized antibody targeting the LILRB4 receptor, which is being developed as a novel treatment for CMML and other blood cancers. The FDA’s Orphan Drug Designation is intended to support the development of drugs for rare diseases, providing benefits such as tax credits and market exclusivity upon approval​.

• By Treatment
  • Chemotherapy
    • Cytarabine
    • Hydroxyurea
  • Hypomethylating Agents
    • Azacitidine
    • Decitabine
  • Targeted Therapy
    • Ruxolitinib
  • Stem Cell Transplantation
    • Allogeneic Stem Cell Transplant
    • Autologous Stem Cell Transplant
• By Distribution Channel
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies