Essential Thrombocythemia (ET) - Market Insight, Epidemiology, and Market Forecast - 2034

Essential Thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50.

• The primary cause of ET is the overproduction of hematopoietic cells due to the mutations of the JAK2, CALR, or MPL genes. These genes are known as ’driver mutations due to their role in developing a myeloproliferative neoplasm. Though 90% of adults have JAK2, CALR, or MPL mutations, it is not unusual to find children with a molecular triple wildtype status.
• Many ET patients are asymptomatic. Consequently, the disease is often diagnosed as part of a routine check-up after a blood test reveals a high platelet count. When symptoms are present, they may include fatigue or may be related to small or large vessel disturbance or bleeding.
• The median estimate of survival among ET patients is 20 years. However, depending on the age of presentation, this varies and as a result, median survival of patients younger than 60 years of age approaches 33 years.
• The most common cause of morbidity and mortality is thrombosis, which occurs among 20% of ET patients, compared to bleeding complications in 10% of this population.
• As per the current market scenario, treatment for essential thrombocythemia focuses on reducing the risk of blood clots and managing symptoms with a range of therapeutic options tailored to individual risk levels.
• Aspirin is commonly recommended in low, intermediate, as well as high-risk patients, as it helps prevent clot formation. First-line treatment often includes a combination of aspirin along with cytoreductive therapies such as hydroxyurea, anagrelide, and interferon-alpha, which aim to reduce platelet counts and prevent complications.
• Cytoreductive therapy is utilized to lower the risk of hemorrhage in patients with platelet counts exceeding 1 million/µL. In certain cases, additional medications such as busulfan, clopidogrel, and anticoagulants may be prescribed, depending on the patient’s condition and response to other treatments.
• Currently, there is only one drug approved specifically for the treatment of ET, and it is authorized exclusively in Europe. In contrast, no therapies have received FDA approval for the treatment of ET in the United States.
• In August 2024, pharmaand GmbH (pharma&) announced that the EC had granted marketing authorization for a Type II variation for PEGASYS (peginterferon alfa-2a) as a monotherapy treatment for adults with ET.
• Beyond PEGASYS, the treatment landscape for ET is evolving with several promising assets in the pipeline. These include BESREMi (ropeginterferon alfa-2b), Bomedemstat (MK-3543/IMG-7289), Pelabresib (CPI-0610), Dencatistat (STP 938), INCA033989, and VAC85135.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase III and Phase II) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the ET market, providing an in-depth examination of its historical and projected market size (2020-2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM ET market.

Essential Thrombocythemia Market

Various key players are leading the treatment landscape of essential thrombocythemia, such as pharma&, AOP Orphan Pharmaceuticals AG, Merck Sharp and Dohme, Novartis, Incyte Corporation, and Others. The details of the country-wise and therapy-wise market size have been provided below.

• In the total market size of Essential Thrombocythemia in the 7MM, the United States accounted for the highest market share, i.e. ~60% in 2024, followed by Japan.
• Among EU4 and the UK, France and the United Kingdom accounted for the highest market size in 2024.
• The United States accounted for approximately USD 250 million in 2024.
• Among the emerging therapies, BESREMI and Bomedemstat appear to be the drugs that can potentially transform the ET market.
• By 2034, among the emerging therapies, the highest revenue is expected to be generated by BESREMI.

Essential Thrombocythemia (ET) Drug Chapters

The section dedicated to drugs in the Essential Thrombocythemia report provides an in-depth evaluation of late-stage pipeline drugs (Phase III and Phase II) related to Essential Thrombocythemia. The drug chapters section provides valuable information on various aspects related to clinical trials of Essential Thrombocythemia, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting Essential Thrombocythemia.

Marketed Therapies

PEGASYS (peginterferon alfa-2a): pharma&

PEGASYS is a Type I interferon; it is made when interferon alfa-2a undergoes the process of pegylation in which one or more chains of PEG are attached to another molecule. PEGASYS was previously approved by the European Commission (EC) for the treatment of Chronic Hepatitis B (CHB) in adults and children aged 3 years and older or Chronic Hepatitis C (CHC) in adults and children aged 5 years and older in combination with other medicinal products in adults or ribavirin in children.

In August 2024, pharma& announced that the EC had granted marketing authorization for a Type II variation for PEGASYS (peginterferon alfa-2a) as a monotherapy treatment for adults with essential thrombocythemia.

Emerging Therapies

BESREMi (ropeginterferon alfa-2b/P1101): AOP Orphan Pharmaceuticals AG/PharmaEssentia

Ropeginterferon alfa-2b is a novel, site-specific, monopegylated, stable IFN-a analog. The unique single isoform differentiates ropeginterferon alfa-2b from earlier generation polypegylated IFN, which utilized random pegylation methods and, therefore, contains many isoforms, with each Polyethylene Glycol (PEG) conjugate having its own activity and stability properties.

PharmaEssentia conducts clinical trials to study the efficacy and safety of ropeginterferon alfa-2b, while AOP Orphan often collaborates in the European market for regulatory approvals and distribution. Ropeginterferon alfa-2b is currently an FDA-approved treatment for patients with polycythemia vera. The company plans to pursue regulatory discussion with the FDA to seek a label expansion to include ET. They anticipate regulatory submission by the end of 2025.

Bomedemstat (MK-3543/IMG-7289): Merck Sharp and Dohme

Bomedemstat (MK-3543) is an investigational small molecule, irreversible LSD1 inhibitor being developed by Merck. LSD1 regulates the proliferation of hematopoietic stem cells, playing an essential role in cell differentiation and maturation. Bomedemstat is being evaluated in a wide range of MPNs, including ET, MF, and PV.

Bomedemstat has Orphan Drug Designation (ODD), and Fast Track Designations (FTDs) for the treatment of ET and myelofibrosis (MF), ODD for the treatment of Acute Myeloid Leukemia (AML), and Priority Medicines scheme designation by the European Medicines Agency for the treatment of MF. Merck presented updated data from the Phase IIb Shorespan-003 trial, including first-time genomic data, at the American Society of Hematology (ASH) Annual Meeting in December 2023.

Essential Thrombocythemia (ET) Market Outlook

Essential thrombocythemia is a rare blood disorder where the bone marrow produces an excessive number of platelets. This overproduction can increase the risk of clot formation, potentially leading to serious complications like stroke, heart attack, or pulmonary embolism. It is characterized by elevated platelet counts and an increased clotting tendency.

As per the current market scenario, treatment for essential thrombocythemia focuses on reducing the risk of blood clots and managing symptoms with a range of therapeutic options tailored to individual risk levels. Aspirin is commonly recommended in low, intermediate, as well as high-risk patients, as it helps prevent clot formation. First-line treatment often includes a combination of aspirin along with cytoreductive therapies such as hydroxyurea, anagrelide, and interferon-alpha, which aim to reduce platelet counts and prevent complications. Further, cytoreductive therapy is utilized to lower the risk of hemorrhage in patients with platelet counts exceeding 1 million/µL. In certain cases, additional medications such as busulfan, clopidogrel, and anticoagulants may be prescribed, depending on the patient’s condition and response to other treatments. Regular monitoring is crucial to adjust therapy and minimize risks.

The development of new therapies for essential thrombocythemia is currently steady, with few new drugs on the horizon. novel agents are being explored, such as BESREMi (Ropeginterferon Alfa-2b), which offers long-acting, sustained platelet-lowering effects with a promising safety profile. Additionally, drugs like Bomedemstat (MK-3543/IMG-7289), targeting specific molecular pathways in the disease, are advancing in clinical trials, potentially offering a more targeted approach to treatment.

In a nutshell, many potential therapies are being investigated to manage Essential Thrombocythemia. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034), it is safe to assume that the future of this market is bright. Eventually, these drugs will create a significant difference in the landscape of ET in the coming years. The treatment space is expected to experience a significant positive shift in the coming years owing to the improvement in healthcare spending worldwide.

Essential Thrombocythemia (ET) Disease Understanding and Treatment

Essential Thrombocythemia (ET) Overview

Essential Thrombocythemia (ET) is a chronic Myeloproliferative Neoplasm (MPN) characterized by an increased number of platelets in the blood. Most commonly diagnosed in women over the age of 50, ET is associated with a proliferation of platelet precursors in the bone marrow, and complications frequently include blood clotting and/or bleeding. Less common consequences in the later stages of ET include a transformation to myelofibrosis (marrow scarring) or acute leukemia.

The exact cause of ET and other MPNs remains unknown. Although ET, like many MPNs, is not considered a genetically inherited condition, some patients may have a familial predisposition. Researchers have identified mutations in many individuals with ET that affect proteins involved in signaling pathways, which play a crucial role in regulating cell growth and development. Many ET patients are asymptomatic. Consequently, the disease is often diagnosed as part of a routine check-up after a blood test reveals a high platelet count. When symptoms are present, they may include fatigue or may be related to small or large vessel disturbance or bleeding.

Essential Thrombocythemia (ET) Diagnosis

Diagnosis of ET should be based on a composite assessment of clinical, morphological, and laboratory features. In this regard, it should be noted that the overwhelming majority of thrombocytosis cases in routine clinical practice are non-clonal and associated with a spectrum of unrelated conditions such as infections, inflammation, post-surgical state, splenectomy, and iron deficiency. On the contrary, while the detection of JAK2V617F, CALR, or MPL mutations confirms the presence of an underlying MPN, their absence does not rule out the possibility of ET since up to 20% of patients might be triple-negative (i.e., negative for all three mutations). It is also important to note that other JAK2/CALR/MPL-mutated MPN (or MDS/MPN) can mimic ET in their presentation; these include pre-fibrotic PMF and MDS/MPN with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). However, in the presence of JAK2/CALR/MPL mutation, the main distinction is with prefibrotic PMF. Bone marrow examination is necessary to make an accurate morphologic diagnosis of ET and distinguish it from other myeloid neoplasms, especially from prefibrotic PMF.

Essential Thrombocythemia (ET) Treatment

Median survival in young patients with ET and PV exceeds 35 years and is not that much worse for older patients. Therefore, it is very important to avoid non-evidence-based therapeutic adventures that might shorten life expectancy and increase the rate of fibrotic or leukemic transformations, as has been previously reported with chlorambucil, radiophosphorus, pipobroman, and anagrelide. To date, drug therapy has not been shown to improve survival or prevent leukemic/fibrotic transformation in either ET or polycythemia vera; therefore, treatment is instead directed at preventing thrombotic complications. In this regard, the decision to institute drug therapy should take into consideration the individual risk of thrombosis, the availability of controlled evidence of value, and potential harm to the patient, both short-term and long-term. In the latter regard, drug-induced alteration of host immunity and impact on clonal evolution are particularly highlighted in the context of opportunistic infections, induction of second malignancies, and leukemic or fibrotic transformation.

Essential Thrombocythemia (ET) Epidemiology

The Essential Thrombocythemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by - Total diagnosed prevalent cases, symptom-specific cases, gender-specific cases, mutation-specific cases, risk-specific cases, age-specific cases and total treated cases in the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2020 to 2034.

• The total diagnosed cases of Essential Thrombocythemia in the US were around 167,450 cases in 2024.
• In the United States, the highest number of age-specific cases were recorded for > 60 yrs of age, i.e., ~108,800 cases in 2024. Followed by the age group of 40-60 yrs and < 40yrs.
• Germany had the highest diagnosed prevalent cases of ET among the EU4 and the UK, accounting for more than 20% of cases in 2024.
• JAK2 was responsible for the majority of ET cases, accounting for 75% of them.
• In 2024, the total treated cases of Essential Thrombocythemia in the US were ~85,000 for the first-line treated patients.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of Essential Thrombocythemia, including the evolving treatment landscape, patients’ reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Analysts connected with more than 15 KOLs across the 7MM. We contacted institutions such as the University of Munich, the University of Tokyo, MD Anderson Cancer Center, Johns Hopkins Kimmel Cancer Center, etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Essential Thrombocythemia market, which will assist our clients in analyzing the overall epidemiology and market scenario.

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 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.

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

In efficacy, the trial’s primary and secondary outcome measures are evaluated; for instance, in trials for Essential Thrombocythemia, one of the most important primary endpoints was achieving hemolysis control, LDH normalization, etc. Based on these, the overall efficacy is evaluated.

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 route of administration, order of entry and designation, 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

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

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.

Essential Thrombocythemia (ET) Report Insights

• Patient Population
• Therapeutic Approaches
• Essential Thrombocythemia Market Size and Trends
• Existing Market Opportunity

Essential Thrombocythemia (ET) Report Key Strengths

• Ten-year Forecast
• The 7MM Coverage
• Essential Thrombocythemia Epidemiology Segmentation
• Key Cross Competition

Essential Thrombocythemia (ET) Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in Essential Thrombocythemia management recommendations?
• Would research and development advances pave the way for future tests and therapies for Essential Thrombocythemia?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of Essential Thrombocythemia?
• What kind of uptake will the new therapies witness in the coming years in Essential Thrombocythemia patients?