Whim Syndrome - Market Insight, Epidemiology and Market Forecast - 2032

This ‘WHIM Syndrome - Market Insights, Epidemiology, and Market Forecast - 2032' report deliver an in-depth understanding of the WHIM Syndrome, historical and forecasted epidemiology as well as the market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The WHIM Syndrome market report provides current treatment practices, emerging drugs, and their market share of the individual therapies, current and forecasted WHIM Syndrome symptoms market size from 2019 to 2032 segmented by seven major markets. The report also covers current WHIM Syndrome symptoms treatment practice/algorithm and unmet medical needs to curate the best opportunities. It assesses the underlying potential of the market.

Geography Covered

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2019-2032

WHIM Syndrome Disease Understanding and Treatment Algorithm

WHIM Syndrome Overview

WHIM syndrome is a primary immunodeficiency disorder, one of a group of disorders characterized by irregularities in the cell development and/or cell maturation process of the immune system. The term WHIM is an acronym for the main signs of the syndrome: warts, hypogammaglobulinemia, infections, and myelokathexis. It is an autosomal-dominant combined immunodeficiency disease caused by mutations in the receptor CXCR4, resulting in increased bone marrow retention and severe reduction in circulating neutrophils.

WHIM Syndrome Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnosis available in the WHIM Syndrome market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

This WHIM Syndrome market report thoroughly understands WHIM Syndrome symptoms by including disease definition, symptoms, causes, physiology, and diagnosis. It also provides WHIM Syndrome treatment algorithms and treatment guidelines for WHIM Syndrome symptoms in the US, Europe, and Japan.

The treatment for WHIM syndrome can be mainly categorized into two categories: curative treatment and symptomatic treatment. Hematopoietic stem cell transplantation (allogenic) is the only available curative treatment for WHIM syndrome, whereas the symptomatic treatment involves the exogenous administration of immunoglobulins (IV/subcutaneous), G-CSF, and CXCR4 antagonists.

Few major unmet needs in the market include no approved therapies, challenges in current symptomatic treatment options, challenges in hematopoietic stem cell transplantation (HSCT), lack of universal diagnostic criteria, and complicated diagnosis of the condition.

WHIM Syndrome Epidemiology

The WHIM Syndrome symptoms epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends and assumptions.

Key Findings

The total diagnosed prevalent cases of WHIM Syndrome patients in the 7MM are increasing during the study period, i.e., 2019-2032.

The disease epidemiology covered in the report provides historical as well as forecasted WHIM Syndrome symptoms epidemiology segmented as the total diagnosed prevalent cases of WHIM Syndrome, gender-specific cases of WHIM Syndrome, and age-specific cases of WHIM Syndrome. The report includes the Incident cases scenario of WHIM Syndrome in 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise WHIM Syndrome Epidemiology

The epidemiology segment also provides the WHIM Syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The total number of diagnosed prevalent cases of WHIM Syndrome associated in 7MM countries was approximately 180 in 2021.

WHIM Syndrome Drug Chapters

The WHIM Syndrome report's drug chapter segment encloses the detailed analysis of WHIM Syndrome early-stage (Phase I, II, and III) pipeline drugs. It also helps understand the WHIM Syndrome 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.

WHIM Syndrome Emerging Drugs

Mavorixafor: X4 Pharmaceutical
Mavorixafor (X4P-001) is a potential first-in-class, once-daily, oral small molecule, and selective antagonist of the CXCR4 receptor that increases mobilization and trafficking of white blood cells from the bone marrow. The drug is currently in Phase III clinical trials for the treatment of WHIM syndrome in patients aged 12 years or older.

In October 2020, US FDA granted Fast Track Designation to mavorixafor, the lead asset of X4 pharmaceuticals for treating WHIM syndrome in adult patients.

Plerixafor (Mozobil, AMD3100): National Institute of Allergy and Infectious Diseases (NIAID)
Plerixafor is a US FDA-approved product indicated to mobilize CD34+ hematopoietic stem cells to the peripheral blood for collection (in combination with granulocyte-colony stimulating factor [G-CSF]) and subsequent autologous transplantation in patients with non-Hodgkin's lymphoma and multiple myeloma. WHIMS is a rare primary immunodeficiency that is known to be caused by mutations that enhance CXCR4 signaling. Plerixafor can be used safely to partially block CXCR4 and treat neutropenia resulting from myelokathexis at doses considerably lower than that being used for CD34+ cell mobilization. National Institute of Allergy and Infectious Diseases is developing plerixafor for the treatment of WHIM syndrome, and the drug has completed Phase III clinical trials.

Other Products detailed in the report…

WHIM Syndrome Market Outlook

The report's WHIM Syndrome market outlook helps to better understand the historic, current, and forecasted WHIM Syndrome market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

This segment gives a thorough detail of WHIM Syndrome market trend of each marketed drug and early-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to view the market at first sight.

According to the publisher, the WHIM Syndrome market in 7MM is expected to grow steadily during the study period 2019-2032.

The current management options for WHIM syndrome can be mainly categorized into two categories: curative treatment and symptomatic treatment. Hematopoietic stem cell transplantation (allogenic) is the only available curative treatment for WHIM syndrome, whereas the symptomatic treatment involves administration of granulocyte-colony stimulating factor (G-CSF), exogenous administration of immunoglobulins (IV/subcutaneous), G-CSF, and CXCR4 antagonists.

The major market players in WHIM Syndrome therapeutic market are Amgen, Sandoz (Novartis), Pfizer, Takeda, and X4 Pharmaceuticals, among others.

Key Findings

This section includes a glimpse of the WHIM Syndrome market in 7MM. WHIM Syndrome market size in the seven major markets was ~USD 8 million in 2021.

The United States Market Outlook

This section provides the total WHIM Syndrome market size and market size by therapies in the United States.

The United States accounts for the largest market size of WHIM Syndrome compared to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

EU-5 Countries: Market Outlook

The total WHIM Syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total WHIM Syndrome market size and market size by therapies in Japan are also mentioned.

WHIM Syndrome Drugs Uptake

This section focuses on the uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2019-2032. The analysis covers the WHIM Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug.

This helps understand the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and the comparison of the drugs based on market share and size, which will again be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

WHIM Syndrome Pipeline Development Activities

The report provides insights into the therapeutic candidate in Phase I, II, and III stages. It also analyses WHIM Syndrome key players involved in developing targeted therapeutics.

Major market players include X4 Pharmaceuticals, Amgen, Sandoz (Novartis), Pfizer, and Takeda.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for WHIM Syndrome emerging therapies.

In December 2020, the US FDA granted Rare Pediatric Disease Designation to mavorixafor for the treatment of WHIM syndrome.

Reimbursement Scenario in WHIM Syndrome

Proactively approaching reimbursement can induce a positive impact both during the early stages of product development and well after product launch. The report considers reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with better market access can be a critical business and price strategy.

Treatment of WHIM syndrome includes granulocyte colony-stimulating factor (G-CSF) and immunoglobulin replacement; however, long-term efficacy remains undefined. Moreover, G-CSF does not correct monocytopenia, lymphopenia, hypogammaglobulinemia, and disabling bone pain that may occur due to its use.

G-CSF has limited Medicare coverage, and filgrastim is covered only for FDA-approved labeled indications for cancer patients and severe chronic neutropenic patients when it is not self/caregiver administered.

The substantial costs associated with G-CSFs are a limiting factor in administering them for prophylaxis. Filgrastim biosimilars have been authorized in the EU since 2008, and a specific approval pathway for biosimilars ensures similarity to the originator is demonstrated in terms of quality characteristics, biological activity, safety, and efficacy. Higher costs of biological medicines have become a major concern for national healthcare systems operating in limited-resource environments.

In an economic evaluation of immunoglobin replacement in patients with primary antibody deficiencies, the estimated direct medical costs ranged from EUR 19,484 for home-based IVIG up to EUR 25,583 for hospital-based IVIG, with home-based SCIG in between EUR 24,952 per year.

KOL Views

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

Competitive Intelligence Analysis

The publisher performs a Competitive and Market Intelligence analysis of the WHIM Syndrome Market by using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of WHIM Syndrome, explaining its causes, signs and symptoms, physiology, and currently available therapies.
• Comprehensive insight has been provided into the WHIM Syndrome epidemiology and treatment in the 7MM.
• Additionally, an all-inclusive account of both the current and emerging therapies for WHIM Syndrome is provided, along with the assessment of new therapies, which will impact the current treatment landscape.
• A detailed review of the WHIM Syndrome 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 WHIM Syndrome market.

Report Highlights

• In the coming years, the WHIM Syndrome market is set to change due to the rising awareness of the disease, development of diagnostic techniques, and the favorable environment from regulators, for innovative first-in-class curative drugs, as there were no approved therapies until recently and are likely to expand the market's size and enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence WHIM Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for WHIM Syndrome. The launch of emerging therapies will significantly impact the WHIM Syndrome market.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase I, II, and III), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, and launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

WHIM Syndrome Report Insights

• Patient Population
• Therapeutic Approaches
• WHIM Syndrome Pipeline Analysis
• WHIM Syndrome Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

WHIM Syndrome Report Key Strengths

• 11-Year Forecast
• 7MM Coverage
• WHIM Syndrome Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

WHIM Syndrome Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness

Key Questions

Market Insights:

• What was the WHIM Syndrome Market share (%) distribution in 2019, and how would it look in 2032?
• What would be the WHIM Syndrome total market size and market size by therapies across the 7MM during the forecast period (2019-2032)?
• What are the key findings of the market across 7MM, and which country will have the largest WHIM Syndrome market Size during the forecast period (2019-2032)?
• At what CAGR, the WHIM Syndrome market is expected to grow in 7MM during the forecast period (2019-2032)?
• What would be the WHIM Syndrome market outlook across the 7MM during the forecast period (2019-2032)?
• What would be the WHIM Syndrome market growth till 2032, and what will be the resultant market Size in the year 2032?

Epidemiology Insights:

• What are the disease risk, burden, and unmet needs of WHIM Syndrome?
• What is the historical WHIM Syndrome patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of WHIM Syndrome in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What will be the growth opportunities in the 7MM concerning the patient population about WHIM Syndrome?
• Out of all 7MM countries, which country would have the largest incident population of WHIM Syndrome during the forecast period (2019-2032)?
• At what CAGR is the population expected to grow in 7MM during the forecast period (2019-2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of WHIM Syndrome along with the approved therapy?
• What are the current treatment guidelines for treating WHIM Syndrome in the USA, Europe, and Japan?
• What are the WHIM Syndrome marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
• How many companies are developing therapies for the treatment of WHIM Syndrome?
• How many therapies are developed by each company for the treatment of WHIM Syndrome?
• How many emerging therapies are in the mid-stage and late stages of development to treat WHIM Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to WHIM Syndrome therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for WHIM Syndrome and their status?
• What key designations have been granted for the emerging therapies for WHIM Syndrome?
• What are the global historical and forecasted markets of WHIM Syndrome?

Reasons to Buy

• The report will help develop business strategies by understanding trends shaping and driving the WHIM Syndrome market.
• To understand the future market competition in the WHIM Syndrome market and an Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for WHIM Syndrome in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identifying strong upcoming players in the market will help devise strategies that will help get ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the WHIM Syndrome market.
• To understand the future market competition in the WHIM Syndrome market.