Pulmonary Arterial Hypertension Market Insight, Epidemiology And Market Forecast - 2034

Pulmonary Arterial Hypertension Market Insight, Epidemiology And Market Forecast - 2034

Key Highlights

In 2023, the market size of myelofibrosis was highest in the US among the 7MM, accounting for approximately USD 1,400 million, which is further expected to increase by 2034.

In the 7MM, JAKAFI/JAKAVI (ruxolitinib) accounted for the largest market size in 2023, with approximately USD 1,500 million.

JAK inhibitors have emerged as the centerpiece of pharmacologic therapy for patients with myelofibrosis, providing unprecedented benefits in terms of spleen shrinkage, symptom improvement, and quality of life that can enhance longevity in patients with advanced disease.

Coming therapies are focusing on different mechanisms other than JAK inhibitors, such as imetelstat (telomerase inhibitor), navitoclax (BCL-XL/BCL-2 inhibitor), navtemadlin (MDM2 protein inhibitor), pelabresib (BET inhibitor), and others which may cover the patient need and provide an alternative treatment for the patients.

AbbVie is expecting Phase III data readout from the TRANSFORM-2 trial of navitoclax in relapsed/refractory myelofibrosis in 2024.

As per Disc Medicine, the updated Phase Ib/II data from DISC-0974 in anemia in myelofibrosis patients is expected to be presented in the first half of 2024.

In January 2024, dosing in Phase I IMproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis (frontline myelofibrosis) by Geron Corporation was escalated to the third of four doses following a decision by the study’s independent safety evaluation team (SET).

DelveInsight’s ‘Myelofibrosis - Market Insight, Epidemiology, and Market Forecast—2034’ report delivers an in-depth understanding of the myelofibrosis, historical and forecasted epidemiology as well as the myelofibrosis market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The myelofibrosis market report provides current treatment practices, emerging drugs, myelofibrosis market share of the individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034 segmented by seven major markets. The report also covers the current myelofibrosis treatment practice/algorithm, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered

The United States

EU4 (Germany, France, Italy, and Spain) and the United Kingdom

Japan

Study Period: 2020–2034

Myelofibrosis: Disease Understanding and Treatment Algorithm

Myelofibrosis Overview

Myelofibrosis is a rare type of blood cancer characterized by the buildup of scar tissue, called “fibrosis,” in the bone marrow. The bone marrow cannot make enough healthy blood cells due to increased scar tissue. It is one of the related groups of blood cancers known as “myeloproliferative neoplasms (MPNs)” in which blood cells produced by bone marrow cells develop and function abnormally. When myelofibrosis develops on its own (and not as the result of another bone marrow disease), it is called primary myelofibrosis. In other cases, another type of MPN, such as polycythemia vera (PV) or essential thrombocythemia (ET), can transform into myelofibrosis. In these cases, it is known as secondary myelofibrosis, which may also be referred to as post-PV myelofibrosis or post-ET myelofibrosis.

Myelofibrosis usually develops slowly, and it often does not cause early symptoms and may be found during a routine blood test. When fibrosis develops in the bone marrow, the bone marrow is unable to produce enough normal blood cells. The lack of blood cells causes many signs and symptoms of myelofibrosis. Several specific gene mutations have been identified in people with myelofibrosis. The most common is the Janus kinase 2 (JAK2) gene mutation, and other less common mutations include CALR and MPL. Some people with myelofibrosis do not have any identifiable gene mutations.

Prominent clinical features in myelofibrosis include anemia, hepatosplenomegaly, and constitutional symptoms including fatigue, night sweats, low-grade fever, and progressive cachexia with loss of muscle mass, bone pain, splenic infarct, pruritus, non hepatosplenic EMH, thrombosis, and bleeding.

Myelofibrosis Diagnosis

Myelofibrosis can be diagnosed by using a series of tests such as blood tests, bone marrow tests, molecular testing, and mutation-enhanced morphologic diagnosis. To confirm the diagnosis, the doctor tests the bone marrow. Bone marrow testing involves two steps usually performed at the same time in a doctor’s office or a hospital: a bone marrow aspiration removes a liquid marrow sample, and a bone marrow biopsy removes a small amount of bone filled with marrow. Molecular tests are used for diagnosis and treatment planning to look for abnormal changes in the genes, chromosomes, proteins, or other molecules within the patient’s cancer cells.

Myelofibrosis Treatment

The only potential cure for myelofibrosis is allogeneic stem cell transplantation, but this procedure is risky for older patients and those with other health problems. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. For most people with myelofibrosis, treatment remains aimed at controlling disease symptoms and complications, enhancing the quality of life, and extending survival. Drugs approved for the treatment of myelofibrosis include JAKAFI/JAKAVI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA/OMJJARA (momelotinib). For patients with low-risk symptoms, treatment options include ruxolitinib, Interferon alfa, and hydroxyurea. Patients with Intermediate and high-risk treatment may include pacritinib, ruxolitinib, fedratinib, and allogeneic stem cell transplantation.

Further details in the report...

Myelofibrosis Epidemiology

The myelofibrosis epidemiology division provides insights into the historical and current myelofibrosis patient pool and forecasted trends for seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken. The disease epidemiology covered in the report provides historical as well as forecasted myelofibrosis epidemiology segmented by total prevalent cases, type-specific cases, based on risk stratification, age-specific cases, based on molecular alterations, treatment eligible pool of myelofibrosis, and transplant eligible/ineligible pool of myelofibrosis in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom and Japan from 2020 to 2034.

The total prevalent cases of myelofibrosis in the 7MM were nearly 56,000 in 2023 and is projected to increase during the study period (2020–2034).

Among the EU4 and the UK, Germany accounted for the highest number of myelofibrosis diagnosed prevalent cases, followed by the Spain, whereas the UK accounted for the lowest number of cases in 2023.

Based on risk, myelofibrosis cases are stratified as low risk, intermediate-1 risk, intermediate-2, and high risk. The high-risk accounted for the highest number of patients in 2023 in the US.

Myelofibrosis can be further categorized into primary myelofibrosis and secondary myelofibrosis. In 2023, primary myelofibrosis accounted for 75% of all cases in the US.

In the US, based on age, myelofibrosis cases are stratified in the age group =49 years, 40-69 years, and =70 years. =70 years of age group accounted for the highest number of patients i.e. nearly 12,000 in 2023 in the US.

In the US, the cases of JAK2 mutations account for approximately 60% in 2023.

Myelofibrosis Drug Chapters

The drug chapter segment of the myelofibrosis report encloses a detailed analysis of Myelofibrosis late-stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Myelofibrosis clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Myelofibrosis Marketed Drugs

JAKAFI/JAKAVI (ruxolitinib): Incyte/Novartis

Ruxolitinib belongs to the class of medications called kinase inhibitors. It works to treat myelofibrosis by blocking the signals that cause cancer cell proliferation, thereby inhibiting the spread of cancer cells. It works to treat GVHD by blocking the signals of the cells that cause GVHD. JAKAFI works by targeting JAKs, which control the production of blood cells. In doing so, JAKAFI helps reduce overactive JAK signaling to help keep the production of blood cells under control. It was approved by the United States Food and Drug Administration (US FDA) for the treatment of adults with intermediate or high-risk myelofibrosis in November 2011, and in August 2012, it was approved by the European Medicines Agency (EMA). In July 2014, it was approved in Japan.

INREBIC (fedratinib): Bristol Myers Squibb

INREBIC is an oral kinase inhibitor with activity against wild-type and mutationally activated JAK2 and FMS-like tyrosine kinase 3 (FLT3). INREBIC is a JAK2-selective inhibitor with higher potency for JAK2 over family members JAK1, JAK3, and TYK2. Abnormal activation of JAK2 is associated with myeloproliferative neoplasms, including myelofibrosis and polycythemia vera. In cell models expressing mutationally active JAK2 or FLT3, INREBIC reduced phosphorylation of signal transducer and activator of transcription (STAT3/5) proteins, inhibited cell proliferation, and induced apoptotic cell death. On August 16, 2019, the US FDA approved INREBIC for adults with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis. In February 2021, the European Commission granted full marketing authorization for INREBIC.

Note: Detailed marketed therapies will be provided in the report.

Myelofibrosis Emerging Drugs

XPOVIO (selinexor): Karyopharm Therapeutics

Selinexor is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This re-initiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells while largely sparing normal cells. In May 2022, the US FDA granted selinexor Orphan Drug Designation (ODD) for the treatment of myelofibrosis, and in October 2022, the EC granted Orphan Medicinal Product Designation for selinexor for the treatment of myelofibrosis. In July 2023, Karyopharm Therapeutics received Fast Track Designation (FTD) from the US FDA for selinexor for the treatment of patients with myelofibrosis, including primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis. In June 2023, Karyopharm Therapeutics announced the initiation of a pivotal Phase III clinical trial (XPORT-MF-034) (NCT04562389) to assess the efficacy and safety of once-weekly selinexor 60 mg in combination with ruxolitinib in JAKi-naïve patients with myelofibrosis.

Imetelstat: Geron Corporation

Imetelstat is an investigational telomerase inhibitor that binds to telomerase and inhibits its activity, selectively killing the malignant stem and progenitor cells in the bone marrow that are the source of disease in blood cancers, such as myelodysplastic syndromes (MDS) and myelofibrosis. By inhibiting the proliferation of these malignant stem and progenitor cells, which enables the recovery of non-malignant bone marrow and blood cell production, this telomerase inhibitor has exhibited disease-modifying activity and clinical benefits for patients in Phase III clinical trials of myelofibrosis. In December 2023, Geron achieved 50% enrollment in the Phase III IMpactMF clinical trial investigating imetelstat versus best available therapy (BAT) in patients with intermediate-2 or high-risk myelofibrosis who are relapsed/refractory myelofibrosis to JAK inhibitor treatment. In January 2024, dosing in Phase I IMproveMF study evaluating imetelstat as a combination therapy with ruxolitinib in patients with intermediate-2 or high-risk myelofibrosis (frontline myelofibrosis) was escalated to the third of four doses following a decision by the study’s independent safety evaluation team (SET).

REBLOZYL (luspatercept/ACE-536): Bristol Myers Squibb

Luspatercept, an erythroid maturation agent, is a recombinant fusion protein consisting of a modified form of the extracellular domain (ECD) of the human activin receptor Type IIB (ActRIIB) linked to the human immunoglobin G1 (IgG1) Fc domain. ActRIIB receptor and its ligands are members of the transforming growth factor-ß (TGF-ß) superfamily. Members of the TGF-ß superfamily ligands bind to activin receptors and are involved in modulating the differentiation of late-stage erythrocyte precursors (normoblasts) in the bone marrow. In January 2020, the US FDA granted ODD to luspatercept for the treatment of myelofibrosis. In February 2020, the EMA granted ODD to luspatercept for the treatment of primary myelofibrosis.

Drug Class Insights

There are currently only four US FDA-approved drugs for the treatment of myelofibrosis. They are OJJAARA/OMJJARA (momelotinib), VONJO (pacritinib), INREBIC (fedratinib), and JAKAFI/JAKAVI (ruxolitinib). The only targeted treatments for MPNs at this time are JAK inhibitors, which were created in response to the identification of the JAK2 V617F mutation as the most frequent recurrent mutation in MPNs. JAK inhibitors have emerged as the centerpiece of pharmacologic therapy for patients with myelofibrosis, providing unprecedented benefits in terms of spleen shrinkage, symptom improvement, and quality of life that can enhance longevity in patients with advanced disease. However, JAK inhibitor therapy is linked with certain complications. Ruxolitinib-related anemia and thrombocytopenia that are dose-dependent in some patients may result in cessation. Anemia and thrombocytopenia can be reduced with dosing techniques, although this could lead to less-than-ideal clinical results. Coming therapies are focusing on different mechanisms other than JAK inhibitors, such as imetelstat (telomerase inhibitor), navitoclax (BCL-XL/BCL-2 inhibitor), navtemadlin (MDM2 protein inhibitor), pelabresib (BET inhibitor), and others which may cover the patient need and provide an alternative treatment for the patients.

Myelofibrosis Market Outlook

Myelofibrosis has limited treatment options, and only a few patients received allogeneic hematopoietic cell transplantation, the only curative therapy. Myelofibrosis has a wide range of clinical manifestations, and the best treatment frequently involves managing many symptoms, such as anemia, splenomegaly, constitutional symptoms, bone pain, and bleeding. JAKAFI (ruxolitinib) was the sole drug approved to treat intermediate- or high-risk myelofibrosis for a long time until the approval of a second JAK inhibitor, INREBIC (fedratinib), in August 2019. In February 2022, VONJO (pacritinib) was approved for the treatment of adults with intermediate or high-risk primary or secondary myelofibrosis with a platelet count below 50 × 109/L in which no other JAK inhibitor was approved. Then in September 2023, the US FDA approved OJJAARA (momelotinib). To date, it is the only approved agent for both newly diagnosed and previously treated patients with myelofibrosis and anemia that addresses the key manifestations of the disease, namely anemia, constitutional symptoms, and splenomegaly. Combination strategies are being investigated to further decrease MF-related symptoms while overcoming dose-limiting cytopenias, especially in patients who cannot tolerate or whose disease is refractory to JAKi monotherapy. In two international Phase III trials in myelofibrosis, drugs given in combination with the JAK inhibitor ruxolitinib in JAK inhibitor–naive patients significantly improved outcomes vs. ruxolitinib alone.

As per DelveInsight’s estimates, the drugs that can mark a significant change in the forecast period include Navtemadlin (KRT-232), Selinexor, Imetelstat, Navitoclax, Luspatercept, Pelabresib, and others.

The total market size of myelofibrosis in the 7MM was approximately USD 1,800 million in 2023 and is projected to increase during the forecast period (2024–2034).

Among EU4 and the UK, Germany accounted for the maximum market size in 2023, while the UK occupied the bottom of the ladder.

Among the therapies, JAKAFI/JAKAVI (ruxolitinib) is expected to generate the highest revenue in the 7MM by 2034.

Myelofibrosis Drugs Uptake

This section focuses on the rate of uptake of the potential drugs expected to be launched in the market during the study period. The analysis covers myelofibrosis market uptake by drugs; patient uptake by therapies; and sales of each drug.

Further details in the report...

Pipeline Development Activities

The report covers detailed information on collaborations, acquisition and merger, licensing, and patent details for Myelofibrosis emerging therapies.

KOL-Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the Myelofibrosis domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or Myelofibrosis market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT 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.

In efficacy, the trial’s primary and secondary outcome measures are 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.

Market Access and Reimbursement

Reimbursement is a crucial factor affecting the drug’s market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models. IncyteCARES (connecting to access, reimbursement, education, and support) provides a single point of contact through a registered nurse to assist eligible patients and healthcare providers in obtaining access to its oncology drugs JAKAFI (ruxolitinib) tablets or PEMAZYRE (pemigatinib) tablets, and to connect them with continuing support and resources. If the patient has been prescribed JAKAFI, IncyteCARES may be able to assist the patient in meeting the copay/coinsurance.

Further details in the report...

Scope of the Report

The report covers the descriptive overview of myelofibrosis, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.

Comprehensive insight has been provided into the myelofibrosis epidemiology and treatment in the 7MM.

Additionally, an all-inclusive account of both the current and emerging therapies for Myelofibrosis is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.

A detailed review of the myelofibrosis market; historical and forecasted is included in the report, covering drug outreach in the 7MM.

The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Myelofibrosis market.

Myelofibrosis Report Insights

Patient Population

Therapeutic Approaches

Myelofibrosis Pipeline Analysis

Myelofibrosis Market Size and Trends

Market Opportunities

Impact of Upcoming Therapies

Myelofibrosis Report Key Strengths

11 Years Forecast

7MM Coverage

Myelofibrosis Epidemiology Segmentation

Key Cross Competition

Highly Analyzed Market

Drugs Uptake

Myelofibrosis Report Assessment

SWOT Analysis

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Conjoint Analysis

Market Attractiveness

FAQs

What was the myelofibrosis total market size, the market size by therapies, and market share (%) distribution in 2023, and what would it look like in 2034? What are the contributing factors for this growth?

Which class is going to be the largest contributor in 2034?

What are the pricing variations among different geographies for approved and off-label therapies?

How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

What are the disease risks, burdens, and unmet needs of myelofibrosis? What will be the growth opportunities across the 7MM concerning the patient population of myelofibrosis?

What is the historical and forecasted myelofibrosis patient pool in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?

What are the current options for the treatment of myelofibrosis? What are the current guidelines for treating myelofibrosis in the US and Europe?

How many emerging therapies are in the mid-stage and late stage of development for the treatment of myelofibrosis?

What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?

What key designations have been granted for the emerging therapies for myelofibrosis?

Patient acceptability in terms of preferred treatment options as per real-world scenarios?

What are the country-specific accessibility issues of expensive, current therapies? Focusing on the reimbursement policies.

Reasons to Buy

The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the myelofibrosis market.

Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.

Understand the existing market opportunities in varying geographies and the growth potential over the coming years.

Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.

Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.

Detailed analysis and ranking of class-wise potential current and emerging therapies.

Highlights of Access and Reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.

To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.

Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.