Latest news with #PolycythemiaVera
Globe and Mail
01-08-2025
- Business
- Globe and Mail
Polycythemia Vera Market Expands as FDA Fast Tracks New Treatments and Clinical Trials Show Promising Results
The 7MM Polycythemia Vera market size reached approximately USD 1,900 million in 2024 and is projected to grow significantly through 2034. Key players such as Incyte/Novartis, PharmaEssentia/AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck, Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience and Disc Medicine, GluBio Therapeutics, and others are spearheading innovations. DelveInsight's report, titled ' Polycythemia Vera Market Insight, Epidemiology and Market Forecast -2034 ', provides a detailed assessment of historical and projected market trends from 2020 to 2034. The analysis encompasses epidemiology segmentation, treatment paradigms, emerging therapies, and competitive dynamics, offering stakeholders actionable insights into market opportunities and challenges. The report highlights how the anticipated introduction of targeted therapies and improved diagnostic tools will reshape the PV treatment landscape over the next decade. In 2024, the United States dominated the Polycythemia market, accounting for over 75% of the total market size, which amounts to USD 1.9 Billion across the 7MM. Among EU4 countries, Germany accounts for the maximum market size, while the United Kingdom occupies the bottom of the ladder in 2024. This trend is expected to continue reflecting disparities in treatment accessibility, Polycythemia Vera prevalence, and diagnostic rates across regions. Download the report to understand which factors are driving the Polycythemia Vera therapeutic market @ Polycythemia Vera Market Trends. The report also provides historical as well as forecasted PV epidemiology segmented by 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, and Age-specific Prevalence of Polycythemia Vera in the 7MM. The report estimates that the 7MM had approximately 320,000 Polycythemia Vera cases in 2024, with numbers expected to rise steadily during the forecast period. Gender-specific data indicates that approximately 56% of the patient share in 7MM is attributed to males, whereas 44% are females. Additionally, across all races and ethnicities, the PV incidence is about 2.8 per 100,000 men and roughly 1.3 per 100,000 women. Discover evolving trends in Polycythemia Vera patient pool forecasts @ Polycythemia Vera Epidemiology Analysis. Recent Developments: In the latest financial statement, Incyte reported strong growth for Jakafi with net revenues of $773 million in Q4 2024 and $2.79 billion for the full year. In March 2025, Protagonist Therapeutics and Takeda announced positive topline results from the Phase 3 VERIFY study, where rusfertide met its primary and all secondary endpoints in treating polycythemia vera. These recent developments indicate positive momentum in the Polycythemia Vera treatment market. Rusfertide achieved a major milestone with the presentation of Phase 3 VERIFY trial results at the ASCO Annual Meeting in June 2025. The study met its primary endpoint and all key secondary endpoints, showing that rusfertide plus current standard of care more than doubled clinical response rates across high- and low-risk PV groups compared to placebo. In May 2025, the FDA granted Fast Track designation to givinostat, an orally administered histone deacetylase (HDAC) inhibitor for treating patients with polycythemia vera, highlighting givinostat's potential to make a meaningful difference. Learn more about the evolving trends in the Polycythemia Vera treatment market @ Polycythemia Vera Recent Developments. Polycythemia Vera management currently relies on a combination of treatments, including phlebotomy, cytoreductive agents such as hydroxyurea, and JAK inhibitors like JAKAFI (RUXOLITINIB) from Incyte/Novartis. Other notable medications used in the management of PV include BESREMi (PharmaEssentia/AOP Orphan Pharmaceuticals) and PEGASYS (pharmaand GmbH). Given its very different MoA compared to JAKAFI and BESREMi, rusfertide could become a viable alternative as an earlier line of therapy. In 2024, JAKAFI generated the highest revenue among these Polycythemia Vera therapies. While these therapies help manage symptoms and reduce thrombotic risks, they are associated with limitations including drug resistance, long-term toxicity, and inadequate control of disease progression. DelveInsight's report emphasizes that a significant number of patients experience suboptimal responses to existing treatments, highlighting a pressing need for novel mechanisms of action. The report highlights that the Polycythemia Vera pipeline features several investigational therapies across phases of development. Notable candidates include DIVESIRAN (Silence Therapeutics), PPMX-T003 (Perseus Proteomics), NAVITOCLAX (AbbVie), VAC85135 (Johnson & Johnson), 9MW3011/DISC-3405 (Mabwell), GLB-001 (GluBio Therapeutic) and others. Despite progress, challenges such as delayed diagnosis, treatment-related anemia, and lack of consensus on optimal monitoring strategies persist. DelveInsight's analysis identifies biomarker development and patient-centric drug delivery systems as key areas for innovation. Table of Contents 1. Key Insights 2. Report Introduction 3. Executive Summary 4. Polycythemia Vera Market Overview at a Glance 5. Epidemiology and Market Forecast Methodology 6. Key Events 7. Polycythemia Vera Background and Overview 8. Polycythemia Vera Treatment 9. Polycythemia Vera Epidemiology and Patient Population in the 7MM 10. Polycythemia Vera Patient Journey 11. Polycythemia Vera Marketed Drug 12. Polycythemia Vera Emerging Drugs 13. Polycythemia Vera: 7MM Analysis 14. Unmet needs 15. SWOT Analysis 16. KOL Views 17. Market Access and Reimbursement 18. Appendix 19. DelveInsight Capabilities 20. Disclaimer 21. About DelveInsight Related Reports Polycythemia Vera Pipeline Insight Polycythemia Vera Pipeline Insight provides comprehensive insights about the Polycythemia Vera pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the Polycythemia Vera companies, including Silence Therapeutics, Ionis Pharmaceuticals, Perseus Proteomics, Kartos Therapeutics, and Disc Medicine among others. About DelveInsight DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.
Associated Press
21-03-2025
- Business
- Associated Press
Pint Pharma Announces ANVISA's Approval of BESREMi® (ropeginterferon alfa-2b) for the Treatment of Polycythemia Vera
SíO PAULO--(BUSINESS WIRE)--Mar 20, 2025-- Pint Pharma and PharmaEssentia announced today that ANVISA (Brazilian Health Regulatory Agency) has approved BESREMi® (ropeginterferon alfa-2b) for the treatment of adult patients with Polycythemia Vera (PV). This press release features multimedia. View the full release here: Polycythemia Vera (PV) is a rare, chronic, debilitating, and potentially fatal myeloproliferative neoplasm, originating from a disease-initiating stem cell in the bone marrow. This results in a persistent increase in red blood cells, white blood cells, and platelets. PV can lead to cardiovascular complications such as thrombosis and embolism, and in a significant number of patients it can progress to secondary myelofibrosis or leukemia. The global incidence of PV is estimated at approximately 2.8 cases per 100,000 people per year. In Brazil, between 2016 and 2020, DataSUS recorded 1,843 cases of Polycythemia Vera, representing an incidence of 0.16 per 100,000 inhabitants. Despite being a rare disease, these numbers suggest the possibility of underdiagnosis and underreporting, highlighting a challenge in identifying affected patients. According to Fernanda Bertasi, General Manager of Pint Pharma in Brazil, 'ANVISA's approval of BESREMi® represents a transformative milestone for patients with Polycythemia Vera in Brazil. This new therapeutic option brings real hope, offering innovation and progress in disease treatment. This is an essential step in providing better health and well-being for these patients.' Valnei Canutti, Hematologist and Chief Scientific Officer at Pint Pharma, emphasized that ' BESREMi® is an innovative, monopegylated interferon with extended action due to its unique pharmacokinetic properties. Its approval was based on robust evidence from the PEGINVERA clinical study, which demonstrated superior and more sustained hematologic and clinical responses compared to standard therapy.' For David Ricardo Muñoz Guzmán, CEO of Pint Pharma, 'This therapeutic advancement reaffirms Pint Pharma's commitment to being a community-centered company. Our mission goes beyond innovation and high technology, focusing on solutions that truly impact patients' lives by expanding access to treatments and improving healthcare quality.' Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia, added, 'The approval of BESREMi® by ANVISA is a testament to the power of strong cross-border partnerships in advancing healthcare and reinforces our mission to transform care for PV patients worldwide. We deeply appreciate the collaboration with the Pint Pharma team, whose expertise and dedication have been instrumental in bringing BESREMi® to patients in Brazil.' BESREMi® is exclusively registered, marketed, and distributed by Pint Pharma in Brazil. About BESREMi® (ropeginterferon alfa-2b) in Polycythemia Vera (PV) BESREMi® is an innovative, monopegylated, long-acting interferon that has marketing authorization in over 40 countries, with approval from the European Medicines Agency (EMA) in 2019, the U.S. Food and Drug Administration (FDA) in 2021, and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023.. With its exclusive pegylation technology, BESREMi® offers extended activity in the body and is designed for administration once every two weeks (or once every four weeks if hematologic stability is maintained for at least 1.5 years), allowing for flexible dosing tailored to individual patient needs. About PharmaEssentia PharmaEssentia Corporation (TWSE: 6446) headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. About Pint Pharma Pint Pharma is a biopharmaceutical company that specialises in the commercialisation of innovative therapies with the potential to transform outcomes in rare disease and other patient communities across the Latin American region. By collaborating with world-class biotech and pharma companies, Pint Pharma is committed to improving the lives of Latin American patients in areas of significant unmet medical need. The company is headquartered in Europe and counts with over 250 employees across its territories in Latin America including Brazil, Mexico, Argentina, Colombia, Chile, Peru and Ecuador. Pint Pharma licensed from PharmaEssentia the rights to commercialize BESREMi® in Brazil. Chief of Scientific Affairs SOURCE: Pint Pharma Copyright Business Wire 2025. PUB: 03/20/2025 07:52 PM/DISC: 03/20/2025 07:52 PM
Yahoo
27-02-2025
- Health
- Yahoo
The Polycythemia Vera Market Landscape Heats Up Amid Rising Competition Among Pharma Companies
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. LAS VEGAS, Feb. 26, 2025 /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. Related Reports Polycythemia Vera Pipeline Polycythemia Vera Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key polycythemia vera companies, including Silence Therapeutics, Ionis Pharmaceuticals, Perseus Proteomics, Kartos Therapeutics, Disc Medicine, among others. Polycythemia Vera Epidemiology Polycythemia Vera Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted polycythemia vera epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Post-Polycythemia Vera Myelofibrosis Market Post-Polycythemia Vera Myelofibrosis Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key post-polycythemia vera myelofibrosis companies, including Kartos Therapeutics, Inc., Parexel, Constellation Pharmaceuticals, Incyte Corporation, NS Pharma, Inc., Celgene, Lynk Pharmaceuticals Co., Ltd, Imago BioSciences, Inc., Karyopharm Therapeutics Inc, AbbVie Inc, Acceleron Pharma Inc, Actuate Therapeutics Inc, Bristol Myers Squibb Co, CTI BioPharma Corp, F. Hoffmann-La Roche Ltd, Geron Corp, Hangzhou East China Pharmaceutical Group Co., Ltd, Italfarmaco SpA, JW Pharmaceutical Corp, MEI Pharma Inc, Merck & Co., Inc., Millennium Pharmaceuticals Inc, Novartis AG, Pharmaxis Ltd, Rhizen Pharmaceuticals SA, Samus Therapeutics Inc, Sierra Oncology Inc, Sino Biopharmaceutical Ltd, Sumitomo Dainippon Pharma Oncology, Inc., Suzhou Zelgen Biopharmaceutical Co., Ltd, Telios Pharma Inc, Lynk Pharmaceutical Hangzhou Co., Ltd, Constellation Pharmaceuticals Inc, among others. Post-Polycythemia Vera Myelofibrosis Pipeline Post-Polycythemia Vera Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key post-polycythemia vera myelofibrosis companies, including Kartos Therapeutics, Inc., Parexel, Constellation Pharmaceuticals, Incyte Corporation, NS Pharma, Celgene, Lynk Pharmaceuticals Co., Ltd, Imago BioSciences, Inc., Karyopharm Therapeutics Inc, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP Sign in to access your portfolio
