Global Myelofibrosis Therapeutics Market - 2024-2031

Global Myelofibrosis Therapeutics Market - 2024-2031

The global myelofibrosis therapeutics market reached US$YY million in 2023 and is expected to reach US$YY million by 2031, growing at a CAGR ofYY% during the forecast period 2024-2031.

Myelofibrosis is a rare form of blood cancer in which the scar tissue builds up in the bone marrow, as a result, the production of new blood cells becomes deficit. Myelofibrosis is related to myeloproliferative disorders, a plethora of conditions affecting bone marrow and causing abnormal blood cell production. There are two types of myelofibrosis, primary and secondary. Primary myelofibrosis occurs on its own, whereas secondary myelofibrosis occurs due to other types of bone marrow conditions such as polycythemia vera or thrombocythaemia.



The exact cause of primary myelofibrosis is unknown, but certainly caused by mutations in the hematopoietic stem cells which divide abnormally and form megakaryocytes in the bone marrow. These megakaryocytes build up in the bone marrow and trigger the cytokines which causes inflammation and buildup of fibrous tissue.



The treatment of myelofibrosis is focused on symptomatic relief mostly as the majority of the patients slowly progress to severity and do not show symptoms in the beginning. The common treatment modalities are blood transfusions, targeted drug therapy, chemotherapy, surgery, bone marrow transplantation, palliative care, etc. Several targeted therapies have been approved in recent times which are revolutionizing the myelofibrosis therapeutics.

Market Dynamics: Drivers

Rising product development and pipeline drugs

Myelofibrosis is a chronic condition whose etiology is still not clearly known. The therapy options currently available are majorly focused on symptomatic relief. In 2011, Jakafi (ruxolitinib) became the first drug approved for the treatment of myelofibrosis. Later only a couple of drugs made it to the commercial-level approval. Due to the scarcity of targeted therapies, the demand is higher and concurrently the research and development activities have also risen. Several manufacturers have been developing innovative therapies, some of them are launched and some are in clinical trials, which if found successful may enter the market and create a positive impact on market growth.

For instance, on September 15, 2023, the US Food and Drug Administration (FDA) approved momelotinib (Ojjaara) developed by GSK plc. for the therapeutics of intermediate or high-risk myelofibrosis in adult patients with anemia. Ojjaara acts by inhibiting three major defective signaling pathways in myelofibrosis, namely JAK1, JAK2, and activin A type 1 receptor. Ojjaara is the first-of-its-kind therapy that aims at alleviating key manifestations such as anemia, spleen enlargement, and constitutional symptoms. In January 2024, the European Commission has approved Ojjaara for marketing in the European Union.

On February 28, 2022, US Food and Drug Administration (FDA) has approved VONJO (pacritinib) developed by CTI BioPharma Corp. for the treatment of both primary and secondary myelofibrosis. FDA has provided accelerated approval for Vonjo based on the positive results from the phase 3 clinical trials. CTI aims to complete the whole trial by mid of 2025 to describe the clinical efficacy of Vonjo to the FDA.

On July 17, 2023, the US Food and Drug Administration (FDA) has provided fast track designation for Selinexor which is being developed by Karyopharm Therapeutics Inc. for the treatment of primary and secondary myelofibrosis. Karyopharm has initiated phase 3 clinical trials for Selinexor and is anticipating to finish by 2028 and produce the primary endpoint data by 2025.

As per DataM analysis, several drugs are being developed for the treatment of myelofibrosis which are in several phases of clinical trials and are anticipated to enter into the market in the forecasted period. Since the demand for specific therapy for myelofibrosis is high, the chances of fast track approvals by the regulatory agencies is high and the products may enter into the market before the anticipated time line. Below is the list of some pipeline drugs which have the potential to enter the market in near future.

S.No Drug Candidate Manufactuter Clinical Trial Phase Estimated Trial Completion Date Estimated Market Entry

1 Pelabresib MorphoSys AG Phase 3 2027 2028

2 Pacritinib CTI BIOPHARMA CORP. Phase 3 2025 Approved in the U.S.

3 Selinexor Karyopharm Therapeutics Inc. Phase 3 2028 2029



Note: Only the drugs under phase 3 were considered in leu with our forecast period. Several drugs are currently in the early phases of clinical trials, which are anticipated to enter the market in the next decade. Also, the market entry year is estimated based on the approval timeline for already marketed drugs.

Moreover, the rising prevalence of myelofibrosis and rising funding for research and development activities are expected to drive market growth in the forecast period.

Restraints

Factors such as limited awareness of the disease to patients and the high cost of treatment are expected to restrain the market growth.

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Segment Analysis

The global myelofibrosis therapeutics market is segmented based on type, therapy type, gender, end user, and region.

The targeted therapies in the treatment type segment accounted for approximately 41.30% of the global myelofibrosis therapeutics market share

Although the etiology of myelofibrosis is not known, a strong emphasis was made on the Janus kinase (JAK) enzyme, whose mutation is the major cause of myelofibrosis. Approximately 50% to 60% of myelofibrosis patients have the JAK1/JAK2 mutation which leads to abnormal blood cell production. Targeting these mutant components which ought to cause myelofibrosis is the main idea behind developing targeted therapy. As of now, regulatory bodies such as the FDA or EU Commission have only approved targeted therapy drugs against myelofibrosis which have gained a lot of importance.

Other therapies such as blood transfusion, androgen therapy, hydroxyurea, and immunomodulators focus on alleviating the symptoms but not the cure. The only cure available for myelofibrosis is stem cell transplantation, but only a limited number of the population with myelofibrosis are eligible who are at high risk and also healthy. For instance, an article published in Elsevier on November 16, 2023 states that hematopoietic stem cell transplantation procedures are rising, but the morbidities associated with it limit the target population. Only the elderly population presents with severe symptoms and severe bone marrow fibrosis and managing them through transplantation procedures poses a great challenge.

Since the majority of the patients with myelofibrosis have mild to moderate severity managing them with targeted therapies has become a gold standard approach. For instance, on February 19, 2024, an article published in the Journal of American Cancer Society stated that the current standard of care treatment for myelofibrosis is the targeted therapy drugs that inhibit JAK/STAT signaling pathways. Ruxolitinib, and fedratinib which are JAK inhibitors have revolutionized the myelofibrosis treatment.

Moreover, in 2023, momelotinib which is another targeted therapy was approved by the FDA and EU to use in myelofibrosis patients with anemia. Likewise, several are in clinical trials anticipated to be launched in this decade. This growing focus on targeted therapies is a major factor contributing to the greater market share for targeted therapies.

The sales of these approved targeted therapies have been rising since their launch and have attained lucrative growth till now. For instance, As per dataM analysis, the revolutionary drug Jakafi (Ruxolitinib) sales were growing at a compound annual growth rate of 11.4% and generated a whopping $2.5 billion in revenue in 2023.



Fig 1: Sales growth projection of Jakafi (Ruxolitinib)

As per DataM analysis, the market value of all the approved targeted therapies for myelofibrosis is approximately $2.8 billion.



Fig:2 Sales value (in USD million) of approved targeted therapies for myelofibrosis in 2023

Geographical Analysis

North America is expected to dominate the market with a 45.30% share in the Global myelofibrosis therapeutics Market

North America is expected to dominate the market due to its well-equipped healthcare facilities and cancer therapeutics centers. The manufacturers of targeted therapies generate the majority of the revenue from the region which is a strong contributing factor for the region’s highest market share. Moreover, the region is well known for providing better access to healthcare services by helping patients with financial coverage. Several government and non-profit organizations in the U.S. provide support for patients who cannot afford the treatment.

For instance, The Leukemia & Lymphoma Society (LLS) in the U.S. provides financial assistance to blood cancer patients including those with myelofibrosis as a part of a co-pay assistance program. The funding is provided to LLS by pharmaceutical and biotechnology companies in the U.S. The program provides financial assistance of up to $4000 per patient with myelofibrosis.

In addition, due to the region's highest expenditure on healthcare, the healthcare facilities provide a plethora of treatment services to myelofibrosis patients. Stem cell therapy which is considered to be the ultimate cure for myelofibrosis is provided by various hospitals and medical institutes across the region.

For instance, The University of Texas MD Anderson Cancer Center has the largest stem cell transplantation program in the U.S. which provides approximately 850 transplants annually in both adults and pediatrics. Likewise, several cancer institutes across the region provide top-tier treatment for myelofibrosis patients.

Moreover, the regulatory scenario in the region is robust with FDA being the governing body that stringently regulates the products and also provides support for innovative therapies. FDA also conducts expedited review programs and provides accelerated approval for absolutely necessary and breakthrough therapies.

For instance, in February 2022, the US Food and Drug Administration provided accelerated approval for Vonjo (pacritinib) to treat intermediate to high-risk myelofibrosis patients. After the approval, the drug was launched in the U.S. market and has attained approximately $60 million in sales in 2023. These are the strong contributing factors for the region’s highest market share in the myelofibrosis therapeutics market.

COVID-19 Impact Analysis

The COVID-19 pandemic has significantly impacted the global market for myelofibrosis therapeutics. Due to imposed restrictions on movement, lack of healthcare resources, and disruption in the supply chain the research activities related to oncology have experienced a halt, especially the clinical trials. Many clinical trials were paused during the pandemic causing delays in the drug development and approval process. Moreover, the healthcare settings where clinical trials take place were allocated to COVID-19-infected patients and set up a triage area for providing necessary care.

For instance, on November 25, 2021, an article published in the National Center for Biotechnology Information stated that during the peak pandemic period in March and April 2020, cancer patient referrals to healthcare facility have fallen by 60% and the new oncology clinical trials have reduced by 60% in march 2021.

Market Segmentation

By Type
• Primary Myelofibrosis
• Secondary Myelofibrosis

By Therapy Type
• Targeted Therapy
Ruxolitinib
Fedratinib
Pacritinib
Momelotinib
• Surgery
Stem Cell Transplantation
Splenectomy
• Immunomodulators
• Androgens
• Cytoreductive Drugs
• Others

By Gender
• Male
• Female

By End Users
• Hospitals & Clinics
• Specialty Centers
• Academia and Research Institutes
• Others

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
U.K.
France
Spain
Italy
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
South Korea
Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major players in the myelofibrosis therapeutics market include Daiichi Sankyo, Inc., F. Hoffmann-La Roche Ltd, Novartis AG, Pfizer Inc., AstraZeneca., Eisai Inc., Puma Biotechnology, Inc., Bristol-Myers Squibb Company., Bayer, and Eli Lilly and Company among others.

Why Purchase the Report?
• To visualize the global myelofibrosis therapeutics market segmentation based on type, therapy type, gender, end-user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development
• Excel data sheet with numerous data points of myelofibrosis therapeutics market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global myelofibrosis therapeutics market report would provide approximately 70 tables, 64 figures, and 187 Pages.

Target Audience 2024
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies