The Polycythemia Vera Market Landscape Heats Up Amid Rising Competition Among Pharma Companies | DelveInsight

The polycythemia vera market over the next few years is expected to substantially change and experience growth, as it will be dominated by two already approved products, Incyte/Novartis' JAKAFI and PharmaEssentia/AOP Orphan Pharmaceuticals' BESREMi. In contrast, we also anticipate the launch and potential uptake of a third product, Protagonist Therapeutics' Rusfertide, in the US market in the coming years.

, /PRNewswire/ -- Polycythemia Vera is a rare disorder marked by a continuous rise in the number of red blood cells in the blood, with an accompanying increase in white blood cells and platelets. It is the most prevalent myeloproliferative neoplasm (MPN), being the most common among these conditions and the only one associated with an increase in red blood cell production.

The total number of prevalent cases of polycythemia vera in the United States was around 182K cases in 2024. According to DelveInsight estimates, there were around 73K cases of asymptomatic polycythemia vera in the United States in 2024. 

Polycythemia vera currently has no cure, but treatments are available to manage the disease and its associated complications. A combination of procedures, medications, and other approaches may be required to control the condition. Two drugs have been approved for treating polycythemia vera: JAKAFI (ruxolitinib) and BESREMi (ropeginterferon alfa-2B/AOP2014/P1101), both of which are authorized for use in the US, Europe, and Japan.

Learn more about the polycythemia vera treatment landscape @ Drugs for Polycythemia Vera Treatment

JAKAFI is an oral inhibitor targeting the JAK1 and JAK2 tyrosine kinases. In the United States, it is marketed under the name JAKAFI by Incyte, while outside the U.S., Novartis holds the license to develop and commercialize it as JAKAVI. In 2014, the U.S. FDA approved the drug for patients with polycythemia vera (PV) who did not respond adequately to or could not tolerate hydroxyurea. A similar approval followed in the EU and Japan in 2015 for the same patient group. In September 2023, NICE issued its Final Draft Guidance (FDG) recommending ruxolitinib for eligible adults in England and Wales with PV who are resistant to or intolerant of hydroxycarbamide/hydroxyurea (HC/HU) chemotherapy.

Blockbuster JAKAFI's revenue-driving indications are Myelofibrosis, Polycythemia Vera, and GvHD. JAKAFI's year-on-year cost increase of more than 5% since 2020 (ICER reports) is one factor driving its market in the US.

BESREMi is a mono-pegylated proline interferon authorized as a first-line monotherapy for adults with PV who do not have symptomatic splenomegaly. It is the only drug approved for first-line use. In 2022, it was added as a recommended treatment in the latest NCCN guidelines. The drug has received approval in both the US and EU and is currently under regulatory review in Japan. In May 2023, the company announced an update to the NCCN Guidelines, designating BESREMi as a preferred treatment option for adults with high- and low-risk PV, regardless of their treatment history.

BESREMi was approved in Q4 2021 in the US; therefore, meaningful sales began in 2022. Before the launch in the US, the drug was approved in the EU in 2019 and has been available in Germany since 2020, however, G-BA did not give a positive nod for additional benefits during assessment. Later on in April 2022, the drug was approved for reimbursement by Italy's National Health Service (SSN) and was approved in Japan in 2023, and now it has been approved in approximately 40 countries globally.

Since BESREMi has a broad label covering both first- and later-line treatments, PharmaEssentia has greater flexibility in positioning it across various therapeutic settings. As BESREMi is recommended earlier than JAKAFI in the NCCN guidelines, it does not directly compete with JAKAFI. However, it may delay patient progression to JAKAFI, potentially impacting its overall revenue. 

The biggest challenge to JAKAFI's dominance in the Polycythemia Vera market could be its patent expiration in mid-2028. To counter this, Incyte launched the LIMBER (Leadership in MPNs and GVHD BEyond Ruxolitinib) initiative, aimed at developing new monotherapy and combination approaches to broaden treatment options for patients with MPNs and GVHD.

In addition to the two approved products, in August 2024, pharmaand GmbH (pharma&) was also granted marketing authorization of a Type II variation for PEGASYS (peginterferon alfa-2a) for adults with polycythemia vera or essential thrombocythemia. Interferon alpha has already been used as an off-label treatment for a long time.

To know more about polycythemia vera treatment options, visit @ New Treatment for Polycythemia Vera

Polycythemia vera treatment landscape is entering a new era with changing dynamics. Several potential therapies are currently under investigation for managing polycythemia vera. If approved, they could bring significant advancements to the treatment landscape in the coming years. The polycythemia vera treatment sector is poised for notable changes due to rising global healthcare expenditures. 

Leading pharmaceutical companies, including Protagonist Therapeutics (rusfertide), Merck (bomedemstat), Italfarmaco (DUVYZAT), Ionis Pharmaceuticals (sapablursen), Silence Therapeutics (Divesiran), and Perseus Proteomics (PPMX-T003), among others, are actively progressing their key drug candidates through various stages of clinical development. 

Discover which therapies are expected to grab major polycythemia vera market share @ Polycythemia Vera Market Report

Rusfertide is an injectable drug designed to mimic the function of the natural hormone hepcidin but with enhanced potency, solubility, and stability. The FDA has granted it orphan drug and fast-track designations for the treatment of polycythemia vera. The drug is currently being evaluated in the pivotal Phase III VERIFY trial for polycythemia vera. 

As per Protagonist Therapeutics' corporate presentation from January 2025, topline results from the Phase III trial (NCT05210790, VERIFY) are expected in Q1 2025 (March). The company also plans to submit a New Drug Application (NDA) for rusfertide in Q4 2025 for polycythemia vera treatment. 

In December 2024, the final results from the Phase II REVIVE trial were presented at the American Society of Hematology (ASH) meeting. The data showed that rusfertide when combined with therapeutic phlebotomy—with or without cytoreductive therapy—effectively controlled erythrocytosis, maintained durable hematocrit control, and reduced the need for therapeutic phlebotomy in polycythemia vera patients. 

Protagonist Therapeutics expects to finalize the study report and submit it to the FDA in Q1 2025. Additionally, the company intends to present the findings at the European Hematology Association (EHA) meeting in Q2 2025 and at the ASH meeting in Q4 2025.

Given its very different mechanism of action compared to JAKAFI and BESREMi, Takeda/Protagonist's rusfertide could become a viable alternative as an earlier line of therapy. We expect the early launch of Rusfertide in 2026-2027, as the therapy has already received both Fast-track and Breakthrough Designations, and has successfully reduced the frequency of phlebotomies.

Givinostat is an orally available hydroxamate-based histone deacetylase (HDAC) inhibitor with potential anti-inflammatory, anti-angiogenic, and anti-cancer properties. The most recent data from its Phase II study, published in 2021, confirmed givinostat's long-term safety and effectiveness, with over 80% of patients achieving clinical and hematologic responses. Following these encouraging results, a pivotal Phase III trial was initiated in 2024, raising hopes for a new therapeutic option for polycythemia vera patients

Bomedemstat is an orally available small molecule being developed by Merck (acquired Imago BioSciences) that targets lysine-specific demethylase 1 (LSD1 or KDM1A), an enzyme crucial for cancer stem and progenitor cells, particularly in abnormal bone marrow cells. Bomedemstat is currently being assessed in a Phase III clinical trial (NCT06351631) for PV. If bomedemstat proves successful in essential thrombocythemia, an expansion into polycythemia vera could be a logical next step. However, given that polycythemia vera remains a secondary focus for now, with no data available, the drug is likely several years away from approval.

Discover more about drugs for polycythemia vera in development @ Polycythemia Vera Clinical Trials

The anticipated launch of these emerging therapies for polycythemia vera 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 polycythemia vera market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.

DelveInsight estimates that the market size for polycythemia vera in the 7MM is expected to grow from USD 1.9 billion in 2024 with a significant CAGR by 2034. This growth is mainly driven by the introduction of new therapies, along with the enhanced integration of early patient screening, secondary care treatments, clinical research on optimal implementation strategies, and increased awareness of polycythemia vera.

DelveInsight's latest published market report titled as Polycythemia Vera 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 polycythemia vera country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The polycythemia vera market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalent Population of Polycythemia Vera
• Prevalent Population of Polycythemia Vera Based on Symptoms
• Gender-specific Prevalence of Polycythemia Vera
• Prevalence of Polycythemia Vera by Gene Mutation
• Prevalence of Polycythemia Vera Based on Risk
• Age-specific Prevalence of Polycythemia Vera

The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM polycythemia vera 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 polycythemia vera 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 polycythemia vera market. Also, stay abreast of the mitigating factors to improve your market position in the polycythemia vera therapeutic space.

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SOURCE DelveInsight Business Research, LLP