Whim Syndrome Market

• In 2022, the market size of WHIM Syndrome was highest in the US among the 7MM, accounting for approximately USD 4.6 million which is further expected to increase by 2032.
• Some of the products that are likely to hit the market during our study period 2019–2032 include Mavorixafor.

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DelveInsight's “WHIM Syndrome - Market Insights, Epidemiology and Market Forecast– 2032” report delivers an in-depth understanding of the WHIM Syndrome, historical and forecasted epidemiology as well as the WHIM Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

WHIM Syndrome market report provides current treatment practices, emerging drug, market size and market share of the individual therapies, current and forecasted 7MM from 2019 to 2032. The report also covers current WHIM Syndrome treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

WHIM Syndrome Treatment Market

The WHIM Syndrome Treatment Market has witnessed significant growth in recent years as advancements in medical research and biotechnology continue to pave the way for innovative therapies. WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) Syndrome is a rare immunodeficiency disorder, and its treatment landscape has expanded with the development of targeted therapies and personalized approaches.

WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome is an ultra-rare and difficult-to-diagnose primary immunodeficiency disease. In almost all patients of WHIM syndrome, the disease is caused by “gain-of-function” mutations in the single gene that encodes for the CXCR4 receptor.

WHIM Syndrome Diagnosis

The diagnosis of WHIM Syndrome is done by Complete Blood Count (CBC), bone marrow biopsy, and molecular genetic testing.

Further details related to country-based variations are provided in the report

WHIM Syndrome Treatment

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

A few emerging therapies, like Mavorixafor (X4 Pharmaceutical), Plerixafor (National Institute of Allergy and Infectious Diseases), etc., are being used to meet the urgent need for safe and efficacious approved treatments.

In a nutshell, it is estimated that the WHIM Syndrome treatment space will experience significant changes during the forecast period of 2023–2032, owing to the launch of novel therapies in clinical development for the management of WHIM Syndrome. However, the challenges of pricing and reimbursement, along with associated side effects, are two major factors likely to influence the market success of the upcoming therapies.

WHIM Syndrome Epidemiology

As the market is derived using a patient-based model, the WHIM Syndrome epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total diagnosed prevalent cases of WHIM Syndrome, Gender specific diagnosed prevalent cases of WHIM Syndrome, and age-specific diagnosed prevalent cases of WHIM Syndrome in the 7MM covering, the United States, EU4 countries (Germany, France, Italy, and Spain), United Kingdom, and Japan from 2019 to 2032.

• There were 174 diagnosed prevalent cases of WHIM Syndrome estimated to have occurred in the 7MM in 2022 of which 117 of the accounted cases were estimated to be from the US alone and these cases are anticipated to increase during forecasted period
• The diagnosed prevalent cases were further divided into gender specific cases. The gender-specific diagnosed prevalent cases of WHIM Syndrome is further categorized into male and female with 69 and 105 cases respectively in the 7MM in 2022 which will further increase in 2032.
• The age specific cases of WHIM Syndrome was divided into less than 18 years and 18 years and above with 91 and 83 cases in the 7MM in 2022.

WHIM Syndrome Drug Chapters

The drug chapter segment of the WHIM Syndrome report encloses a detailed analysis of WHIM Syndrome late-stage and mid-stage (Phase-III and Phase-II) pipeline drug. It also helps to 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 (X4P-001): X4 Pharmaceuticals

Mavorixafor (X4P-001) is a potential first-in-class, once-daily oral small molecule and selective antagonist of the CXCR4 receptor that increases the mobilization and trafficking of white blood cells from the bone marrow. The drug is currently in Phase III clinical trials for treating WHIM syndrome in patients aged 12 years or older. Mavorixafor is also being investigated to treat severe congenital neutropenia (SCN), Waldenström’s macroglobulinemia (WM), breast cancer, malignant melanoma, and clear cell renal cell carcinoma (ccRCC).

Detailed emerging therapies list will be provided in the final report.

WHIM Syndrome Market Outlook

Treatment for WHIM patients is not standardized but aims to mitigate hematologic defects and associated clinical symptoms. Current treatment strategies and clinical trials focus on Neutropenia, hypogammaglobulinemia, and bacterial infections. No pharmacologic agents have a demonstrated ability to prevent or treat warts in WHIM patients. Successful treatment of warts in WHIM patients is typically restricted to destructive therapies. The HPV vaccine is limited to a subset of the most highly cancer-associated strains.

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

The major market players in WHIM syndrome therapeutic market is X4 Pharmaceuticals.

Mavorixafor (X4P-001) is a potential first-in-class, once-daily oral small molecule and selective antagonist of the CXCR4 receptor that increases the mobilization and trafficking of white blood cells from the bone marrow. The US FDA granted FTD, BTD, ODD, and Rare Pediatric Disease Designation to mavorixafor for treating WHIM syndrome. The drug is currently in Phase III clinical trials for treating WHIM syndrome in patients aged 12 years or older.

• The total market size of WHM Syndrome in the 7MM is approximately USD 6.1 million in 2022 and is projected to increase during the forecast period (2023-2032)
• The market size in the 7MM will increase at a CAGR of about 16.3% due to increasing awareness of the disease and launch of the emerging drug
• Among EU countries, France, Italy, and UK accounts for the maximum market size of USD 0.4, USD 0.3 and USD 0.3 million in 2022 while Spain occupies the bottom of the ladder in the same year with USD 0.1 million.
• Japan accounts for market size of USD 0.29 million in 2022, but these dynamics are expected to change in the forecasted years.

WHIM Syndrome Drug Uptake

This section focuses on the rate of uptake of the potential WHIM Syndrome pipeline drugs expected to get launched in the market during the study period 2019-2032. For example, we estimate the drug (Mavorixafor) will be launched in the year 2024 in the US, respectively. Currently, Mavorixafor is in the Phase III clinical trial stage.

Further detailed analysis of emerging therapies drug uptake are in the report…

WHIM Syndrome Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I stage. It also analyzes key players involved in developing targeted therapeutics.

KOL- Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on WHIM Syndrome evolving treatment landscape, patient reliance on conventional therapies, patient’s therapy switching acceptability, drug uptake along with challenges related to accessibility, include the David Geffen School of Medicine, Los Angeles, the US; University Hospital Aachen, Germany; Department of Pediatrics, Institute of Molecular Medicine, Brescia, Italy, Immunologist, Sant Joan de Déu Research, Spain; Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan, and Others.

DelveInsight’s analysts connected with 50+ KOLs to gather insights, however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as the Department of Pediatrics, Department of Immunology, etc. were contacted. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or WHIM Syndrome 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.

WHIM Syndrome Market Access and Reimbursement

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

WHIM Syndrome Market Report Scope

• The report covers a segment of key events, an executive summary, descriptive overview of WHIM Syndrome, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression along with treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies along with the elaborative profiles of late-stage and prominent therapies will have an impact on the current treatment landscape.
• A detailed review of the WHIM Syndrome market; historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The patient-based WHIM Syndrome market forecasting report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM WHIM Syndrome market.

WHIM Syndrome Market Report Insights

• Patient-based WHIM Syndrome Market Forecasting
• WHIM Syndrome Therapeutic Approaches
• WHIM Syndrome Pipeline Analysis
• WHIM Syndrome Market Size and Trends
• Existing and future Market Opportunity

WHIM Syndrome Market Report Key Strengths

• 10 Years WHIM Syndrome Market Forecast
• 7MM Coverage
• WHIM Syndrome Epidemiology Segmentation
• Key Cross Competition
• Drug Uptake and Key Market Forecast Assumptions

WHIM Syndrome Market Report Assessment

• Current WHIM Syndrome Treatment Practices
• WHIM Syndrome Unmet Needs
• WHIM Syndrome Pipeline Drugs Profiles
• WHIM Syndrome Market Attractiveness
• Qualitative Analysis (SWOT Analysis)

Key Questions

Market Insights:

• What was the WHIM Syndrome total market size, the market size by therapies, and market share (%) distribution in 2019, and how it would all look in 2032? What are the contributing factors for this growth?
• Will the coverage of drug depend on their efficacy in WHIM Syndrome?
• What will be the impact on the market with the launch of Mavorixafor?
• How much market will be captured by Mavorixafor after its launch?
• What are the pricing variations among different geographies for G-CSF treatment, IG replacement, prophylactic antibiotics?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What are the disease risk, burdens, and unmet needs of WHIM Syndrome? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to WHIM Syndrome?
• What is the historical and forecasted WHIM Syndrome patient pool in the United States, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan?
• How many male and female patients are of WHIM Syndrome?
• How many patients are of WHIM Syndrome of above 18 years and below 18 years?
• What factors are affecting the increase in the diagnosis of WHIM Syndrome?

Current Treatment Scenario, Marketed Drug, and Emerging Therapies:

• What are the current options for the standard of care of WHIM Syndrome? What are the current treatment guidelines for the treatment of WHIM Syndrome in the US and Europe?
• How many companies are developing therapies for the treatment of WHIM Syndrome?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of WHIM Syndrome?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the patents of emerging therapies for WHIM Syndrome?
• What will be the impact on the market after the expected patent expiry of the emerging drug?
• What is the cost burden of marketed therapies on patients?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of recommended therapies? Focus on reimbursement policies.
• What are the 7MM historical and forecasted market of WHIM Syndrome?

Reasons to buy

• The patient-based WHIM Syndrome market forecasting report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the WHIM Syndrome Market
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• To understand the existing market opportunity 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.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and potential of current and emerging therapies to provide visibility around leading emerging drug.
• Highlights of Access and Reimbursement policies of G-CSF, Prophylactic antibiotics, IG replacement and patient assistance programs
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in future.
• Detailed insights on the unmet need of the existing market so that the upcoming players can strengthen their development and launch strategy.