Essential Thrombocythemia Market to Witness Robust Growth During the Forecast Period (2025-2034) Amid Rising R&D Activity | DelveInsight

Beyond PEGASYS, the essential thrombocythemia treatment landscape 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.

LAS VEGAS, June 3, 2025 /PRNewswire/ -- Essential thrombocythemia is a chronic form of myeloproliferative neoplasm (MPN) marked by an abnormally high platelet count in the bloodstream. It is most frequently diagnosed in women over 50 and involves the excessive production of platelet precursors in the bone marrow. Common complications include blood clots and bleeding, while less frequent but more serious outcomes in advanced stages can involve progression to myelofibrosis or acute leukemia.

As of 2024, approximately 167K individuals in the US were diagnosed with essential thrombocythemia, with about 75% of these cases linked to mutations in the JAK2 gene. During the same year, an estimated 85K patients in the US received first-line treatment for essential thrombocythemia.

The primary goal of essential thrombocythemia management is to reduce the risk of vascular complications, such as thrombotic and hemorrhagic events, which are major contributors to illness and death. Treatment approaches are guided by a risk stratification system that classifies patients as either low or high risk for thrombosis. Typically, low-risk individuals are younger and have no history of clotting events, while high-risk patients are generally over 60 years old and have experienced thrombosis in the past.

Learn more about the essential thrombocythemia cancer @ New Treatment for Essential Thrombocythemia

For low-risk patients, aspirin is recommended, provided there are no major contraindications. However, caution is advised when platelet counts exceed 1,000 × 10⁹/L, as this raises the risk of acquired von Willebrand disease. In such cases, if laboratory findings suggest von Willebrand abnormalities, aspirin should be avoided. Aspirin is also the preferred treatment in patients experiencing microvascular complications like erythromelalgia. 

In contrast, high-risk patients are typically managed with a combination of antiplatelet and cytoreductive therapies. Hydroxyurea is generally considered the first-line agent for cytoreduction. However, anagrelide may be used as an alternative when hydroxyurea is poorly tolerated or ineffective.

At present, only one drug is officially approved for the treatment of essential thrombocythemia, and its use is restricted to Europe. There are currently no FDA-approved therapies for essential thrombocythemia in the United States. PEGASYS, a Type I interferon, is made by pegylating interferon alfa-2a, which involves attaching polyethylene glycol (PEG) chains to the molecule. 

Find out more on FDA-approved essential thrombocythemia drugs @ Essential Thrombocythemia Treatment Options

PEGASYS was previously approved by the European Commission (EC) for treating Chronic Hepatitis B in adults and children over 3, and Chronic Hepatitis C in adults and children over 5, in combination with other medications. In August 2024, pharmaand GmbH announced that the EC had granted a Type II variation approval for PEGASYS, allowing its use as a monotherapy for adults with essential thrombocythemia.

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.

Discover which therapies are expected to grab major essential thrombocythemia market share @ Essential Thrombocythemia Market Report

Bomedemstat (MK-3543) is an experimental small-molecule drug developed by Merck that irreversibly inhibits LSD1, a key regulator of hematopoietic stem cell proliferation, differentiation, and maturation. The drug is under clinical evaluation for various myeloproliferative neoplasms (MPNs), including essential thrombocythemia, myelofibrosis, and polycythemia vera.

In 2018, Bomedemstat was granted Orphan Drug Designation (ODD) for essential thrombocythemia treatment. In January 2020, Imago BioSciences received Fast Track Designation (FTD) from the US FDA for bomedemstat for the same indication.

By November 2022, Merck announced its acquisition of Imago BioSciences, a clinical-stage biotechnology company focused on bomedemstat for myelofibrosis treatment, for $36 per share in cash, totaling approximately $1.35 billion. This strategic move aimed to strengthen Merck's hematology portfolio and establish its presence in the growing myelofibrosis market.

PharmaEssentia is advancing ropeginterferon alfa-2b (P1101) for essential thrombocythemia, offering a biweekly injection regimen aimed at improving tolerability and patient adherence, thereby enhancing therapeutic outcomes. A global Phase III clinical trial for essential thrombocythemia is currently in progress. Participants who respond positively during the initial study period will continue into an extension phase for long-term evaluation.

Pelabresib, another investigational therapy, is a selective small molecule targeting BET (bromodomain and extra-terminal domain) proteins, which are involved in gene regulation associated with hematologic malignancies. The ongoing Phase II MANIFEST trial (NCT02158858) is assessing its efficacy in patients with myelofibrosis and high-risk ET through an open-label study.

In February 2024, MorphoSys entered into a Business Combination Agreement with Novartis AG, reflecting Novartis's plan to launch a voluntary public takeover offer for all outstanding MorphoSys bearer shares without nominal value.

Discover more about drugs for essential thrombocythemia in development @ Essential Thrombocythemia Clinical Trials

The anticipated launch of these emerging therapies for essential thrombocythemia are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the essential thrombocythemia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

DelveInsight estimates that the market size for essential thrombocythemia is expected to grow from USD 417 million in the 7MM in 2024 at a significant CAGR by 2034. The market size of essential thrombocythemia is expected to grow due to several factors, including an increase in the prevalent pool, the expected entry of emerging therapies with novel targets, and attractive pricing in the 7MM.

DelveInsight's latest published market report, titled as Essential Thrombocythemia Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the essential thrombocythemia country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The essential thrombocythemia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Diagnosed Prevalent Cases
• Symptom-specific Cases
• Gender-specific Cases
• Mutation-specific Cases
• Risk-specific Cases
• Age-specific Cases
• Total Treated Cases

The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM essential thrombocythemia market. Highlights include:

• 10-year Forecast
• 7MM Analysis
• Epidemiology-based Market Forecasting
• Historical and Forecasted Market Analysis upto 2034
• Emerging Drug Market Uptake
• Peak Sales Analysis
• Key Cross Competition Analysis
• Industry Expert's Opinion
• Access and Reimbursement

Download this essential thrombocythemia market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the essential thrombocythemia market. Also, stay abreast of the mitigating factors to improve your market position in the essential thrombocythemia therapeutic space.

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SOURCE DelveInsight Business Research, LLP