WHIM Syndrome - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights

• WHIM syndrome is a rare genetic disorder caused by CXCR4 mutations, leading to immunodeficiency, warts, hypogammaglobulinemia, and myelokathexis. In 2023, the diagnosed prevalent population in the 7MM was estimated to be around 179 individuals.
• WHIM is a significant health issue in the United States, and its prevalence has been increasing over the past few decades. According to the estimation, among the 7MM, the US has most number of cases of WHIM accounting for 68% of cases alone, followed by France in 2023.
• Assessments as per The analysts show that the overall diagnosed prevalent cases of WHIM syndrome in both genders in EU4 and the UK are anticipated to increase in the coming years in comparison to 19 males and 29 females, in 2023.
• The market size of WHIM syndrome is influenced by various factors, such as the prevalence of the condition, the availability and adoption of emerging therapies, regulatory approvals, treatment costs, and the overall awareness of the disease. Additionally, advancements in diagnostics and personalized medicine may also impact market dynamics.
• In 2023, the market size of WHIM was highest in the US among the 7MM, accounting for approximately USD 4.8 million which is further expected to increase by 2034.
• The market size of WHIM in Japan was nearly USD 0.3 million in 2023, accounting for only 5% of the total market of the 7MM. The total market size in the 7MM is expected to rise by 2034 at a compound annual growth rate (CAGR) of 16.4%.
• The pipeline for WHIM syndrome is notably limited, indicating a lack of ongoing research and development for new therapies. This scarcity highlights the need for increased investment and innovation in addressing this rare condition. The total market size of WHIM is anticipated to upsurge during the forecast period due to the expected entry of emerging therapies.
• In April 2024, the US FDA granted approval of X4 Pharmaceuticals’ XOLREMDI (mavorixafor) capsules for WHIM syndrome, making it the first officially approved treatment for this condition. This breakthrough therapy, approved following a Priority Review of Phase III trial data, addresses CXCR4 pathway dysfunction, the root cause of WHIM syndrome. X4 also received a Rare Pediatric Disease Priority Review Voucher. This approval is a significant advancement, offering a new treatment option for patients aged 12 and older, aimed at increasing circulating mature neutrophils and lymphocytes.

The “WHIM Syndrome Market Insights, Epidemiology, and Market Forecast - 2034” report delivers an in-depth understanding of the WHIM, historical and forecasted epidemiology as well as the WHIM market trends in the United States, EU4 and the UK (Germany, France, Italy, Spain), the United Kingdom, and Japan.

The WHIM market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM WHIM market size from 2020 to 2034. The Report also covers current WHIM treatment practice, market drivers, market barriers, SWOT analysis, reimbursement and market access, 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

WHIM Syndrome Treatment Market

WHIM Overview

WHIM syndrome is a rare genetic disorder caused by mutations in the CXCR4 gene, leading to a triad of symptoms: immunodeficiency, warts, and hypogammaglobulinemia, often accompanied by myelokathexis, a condition where neutrophils are trapped in the bone marrow. This syndrome significantly impairs immune function and increases susceptibility to infections. The term WHIM is an acronym for the main signs of the syndrome: warts, hypogammaglobulinemia, infections, and myelokathexis.

WHIM syndrome manifests through several distinct symptoms. Individuals with this condition experience immunodeficiency, which leads to increased vulnerability to infections due to compromised immune function. Another prominent symptom is the development of persistent and recurrent warts on the skin and mucous membranes. Hypogammaglobulinemia, characterized by low levels of immunoglobulins (antibodies) in the blood, further exacerbates immune system dysfunction. Additionally, myelokathexis, a condition where neutrophils accumulate in the bone marrow but are reduced in the bloodstream, is a key feature of WHIM syndrome. These symptoms arise from mutations in the CXCR4 gene, impacting the body's ability to effectively respond to infections and maintain overall health.

WHIM Diagnosis

Diagnosing WHIM syndrome involves clinical evaluation of symptoms such as recurrent infections, warts, hypogammaglobulinemia, and myelokathexis, followed by genetic testing to confirm CXCR4 gene mutations. While genetic testing is crucial for a definitive diagnosis, there are unmet needs in this process. Many patients face delays due to lack of awareness and underdiagnosis, as symptoms may overlap with other conditions.

Additionally, the availability of specialized testing and expertise can be limited, further complicating timely diagnosis. Addressing these gaps through improved awareness, broader access to genetic testing, and enhanced diagnostic tools is essential for early intervention and effective management of WHIM syndrome.

WHIM Treatment

Treatment for WHIM syndrome focuses on managing symptoms and addressing the underlying genetic defect. Current approaches include continuous positive airway pressure (CPAP) therapy for associated respiratory issues, and medications to manage infections and immune deficiencies. CXCR4 antagonists, such as XOLREMDI (mavorixafor), aim to target the root cause by inhibiting the CXCR4 receptor, potentially improving immune function and reducing symptoms. Additionally gene therapy is being explored as a long-term solution to correct the genetic mutation.

However, significant unmet needs remain. Adherence to current therapies, such as CPAP and infection management, can be challenging for patients. Additionally, many available treatments only partially address the syndrome's complex symptoms, and there is a lack of personalized therapeutic options. More research into effective, tailored treatments and better access to emerging therapies are critical for improving outcomes and quality of life for individuals with WHIM syndrome.

WHIM Epidemiology

As the market is derived using the patient-based model, the WHIM epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by 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), the United Kingdom, and Japan from 2020 to 2034.

• The total diagnosed prevalent cases of WHIM in the US comprised approximately 121 in 2023 and are projected to increase during the forecast period.
• As per the estimation, the US alone accounts the highest number of total diagnosed prevalent cases, of WHIM in the 7MM, followed by EU4 and the UK and Japan, contributing to 27% and 6% of all WHIM cases respectively.
• Forecast assessments suggest a growing prevalence of diagnosed WHIM syndrome cases, projecting an increase to approximately 48 males and 73 females by 2023 in the US. This trend reflects an anticipated rise in both genders over the forecast period.
• EU4 and the UK accounted for around 48 diagnosed prevalent cases of WHIM in 2023. Of these cases, France accounted for the highest number of cases.

WHIM Drug Chapters

The drug chapter segment of the WHIM report encloses a detailed analysis of WHIM approved and off-label drugs along with late-stage (Phase III) pipeline drugs. It also helps to understand the WHIM 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 Marketed Drugs

XOLREMDI (mavorixafor): X4 Pharmaceuticals

XOLREMDI (mavorixafor) is the first FDA approved capsules indicated in patients with WHIM syndrome promising first-in-class, oral small molecule, functioning as a selective CXCR4 receptor antagonist. This drug enhances the mobilization and trafficking of white blood cells from the bone marrow. Beyond WHIM syndrome, Mavorixafor is being explored for severe congenital neutropenia (SCN), Waldenström’s macroglobulinemia, breast cancer, malignant melanoma, and clear cell renal cell carcinoma (ccRCC).

In terms of regulatory milestones, XOLREMDI has garnered significant attention. The US FDA granted Fast Track Designation (FTD) in October 2020 and Breakthrough Therapy Designation (BTD) in November 2019 for treating WHIM syndrome in adults. Additionally, it received Orphan Drug Designation (ODD) from the FDA in October 2018 and Rare Pediatric Disease Designation in December 2020. The European Commission also granted ODD for WHIM syndrome in July 2019.

XOLREMDI, according to the forecast model would capture a peak share of 60% in 2024 and is anticipated to take 6 years to peak with a medium-fast uptake.

Emerging WHIM Syndrome Drugs

Plerixafor: National Institute of Allergy and Infectious Diseases (NIAID)

Plerixafor (MOZOBIL, AMD3100), approved by the US FDA, mobilizes CD34+ hematopoietic stem cells for autologous transplantation in non-Hodgkin’s lymphoma and multiple myeloma patients. It also addresses WHIM syndrome by partially blocking CXCR4, used at lower doses than for stem cell mobilization. The National Institute of Allergy and Infectious Diseases is advancing plerixafor for WHIM syndrome, which has completed Phase III trials. Its mechanism involves specific, reversible inhibition of the CXCR4 receptor, disrupting the binding of stromal cell-derived factor-1 (SDF-1) and affecting cellular adhesion.

However, since the conclusion of Phase II/III trials in 2021, there have been no updates or further advancements reported for this drug. This stagnation suggests a potential pause or lack of progress in its development pipeline or regulatory status.

Note: Detailed emerging therapies assessment will be provided in the final report of WHIM Syndrome...

WHIM Market Outlook

With first approved therapy for WHIM Syndrome in 2024, the market outlook for WHIM is expected to expand.

Treatment for WHIM syndrome includes both pharmacological and non-pharmacological approaches. Pharmacological treatments primarily involve Plerixafor (MOZOBIL), a CXCR4 receptor antagonist that helps manage neutropenia by partially blocking CXCR4 signaling. This is often used alongside Granulocyte-Colony Stimulating Factor (G-CSF) to enhance white blood cell mobilization. Additionally, antibiotics and antifungals are prescribed to manage infections stemming from the immune deficiencies characteristic of WHIM syndrome.

On the non-pharmacological side, supportive care is crucial, involving regular monitoring to manage and prevent infections. Lifestyle adjustments such as practicing good hygiene and minimizing infection risks are also recommended. Furthermore, psychosocial support is important to help patients cope with the emotional and psychological challenges associated with living with a chronic condition. These combined strategies aim to improve overall health outcomes and quality of life for individuals with WHIM syndrome.

• The launch of emerging therapies, such as Plerixafor (Mozobil), and others are expected to impact the market positively. The approval of these therapies could significantly impact market dynamics, although their success rates remain uncertain.
• The market size of WHIM in the 7MM was nearly USD 6.3 million in 2023, which is further anticipated to increase during the forecast period.
• The EU4 and the UK accounted for the approximately USD 1.3 million market size of WHIM approximately in 2023.
• Among the EU countries, France had the highest market size with nearly USD 0.4 million in 2023, while Spain had the lowest market size for WHIM with USD 0.1 million in 2023.
• With the expected launch of upcoming therapies, such as Plerixafor (Mozobil), among others, the total market size of WHIM is expected to show change in the upcoming years.

WHIM Pipeline Development Activities

The report provides insights into WHIM syndrome clinical trials within Phase III, Phase II, and Phase I stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for WHIM emerging therapies.

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 the secondary research. Industry Experts were contacted for insights on WHIM evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including KOL from Associate Research Physician, National Institute of Allergy and Infectious Disease, the US; University Hospital Aachen, Germany; Department of Pediatrics, Institute of Molecular Medicine, Brescia, Italy; Department of Pediatrics, Jikei University School of Medicine, Tokyo, Japan; Immunologist, Sant Joan de Déu Research, Spain, and others.

The analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or WHIM 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 and Conjoint 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.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

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. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

The high cost of therapies for the treatment is a major factor restraining the growth of the global drug market. Because of the high cost, the economic burden is increasing, leading the patient to escape from proper treatment.

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.

Scope of the WHIM Syndrome Market Report

• The report covers a segment of key events, an executive summary, descriptive overview of WHIM syndrome, explaining its causes, signs and symptoms, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the WHIM market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind the approach is included in the report covering the 7MM drug outreach.
• The 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 shape and drive the 7MM WHIM market.

WHIM Report Insights

• WHIM Syndrome Patient Population
• Therapeutic Approaches
• WHIM Pipeline Analysis
• WHIM Market Size and Trends
• Existing and Future Market Opportunity

WHIM Report Key Strengths

• 11 years Forecast
• The 7MM Coverage
• WHIM Epidemiology Segmentation
• Key Cross Competition
• Conjoint Analysis
• WHIM Syndrome Drugs Uptake
• Key WHIM Syndrome Market Forecast Assumptions

WHIM Report Assessment

• Current WHIM Syndrome Treatment Practices
• WHIM Syndrome Unmet Needs
• WHIM Syndrome Pipeline Product Profiles
• WHIM Syndrome Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)
• WHIM Syndrome Market Drivers
• WHIM Syndrome Market Barriers

Key Questions Answered In The WHIM Syndrome Market Report

WHIM Syndrome Market Insights

• What was the WHIM market share (%) distribution in 2020 and how it would look like in 2034?
• What would be the WHIM total market size as well as market size by therapies across the 7MM during the forecast period (2024-2034)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest WHIM market size during the forecast period (2024-2034)?
• At what CAGR, the WHIM market is expected to grow at the 7MM level during the forecast period (2024-2034)?
• What would be the WHIM market outlook across the 7MM during the forecast period (2024-2034)?
• What would be the WHIM market growth till 2034 and what will be the resultant market size in the year 2034?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

WHIM Syndrome Epidemiology Insights

• What is the disease risk, burden, and unmet needs of WHIM Syndrome?
• What is the historical WHIM patient population in the United States, EU5 (Germany, France, Italy, Spain, and the UK), and Japan?
• What would be the forecasted patient population of WHIM at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to WHIM Syndrome?
• Out of the above-mentioned countries, which country would have the highest prevalent population of WHIM syndrome during the forecast period (2024-2034)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2024-2034)?

Current WHIM Syndrome Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of WHIM along with the approved therapy?
• What are the current treatment guidelines for the treatment of WHIM in the US, Europe, And Japan?
• What are the WHIM 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 emerging therapies are in the mid-stage and late stages of development for the treatment of WHIM Syndrome?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the WHIM therapies?
• What are the recent therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for WHIM and their status?
• What are the key designations that have been granted for the emerging therapies for WHIM Syndrome?
• What are the 7MM historical and forecasted market of WHIM Syndrome?

Reasons to Buy WHIM Syndrome Market Report

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the WHIM 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.
• 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 under the conjoint analysis section to provide visibility around leading emerging drugs.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, 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.

Frequently Asked Questions

1. What is the forecast period covered in the report?

The WHIM Epidemiology and Market Insight report for the 7MM covers the forecast period from 2024 to 2034, providing a projection of market dynamics and trends during this timeframe.

2. Who are the key players in the WHIM market?

The WHIM market currently lacks robust development. However, the forecast period may see significant activity as major players are anticipated to enter the market and focus on developing treatments for WHIM syndrome.

3. How is the market size estimated in the forecast report?

The market size is estimated through data analysis, statistical modeling, and expert opinions. It may consider factors such as incident cases, treatment costs, revenue generated, and market trends.

4. What is the key driver of the WHIM market?

The increase in diagnosed prevalent cases of WHIM and the launch of emerging therapies are attributed to be the key drivers for increasing the WHIM market.

5. What is the expected impact of emerging therapies or advancements in WHIM treatment on the market?

Introducing new therapies, advancements in diagnostic techniques, and innovations in treatment approaches can significantly impact the WHIM treatment market. Market forecast reports may provide analysis and predictions regarding the potential impact of these developments.

6. Does the report provide insights into the competitive landscape of the market?

The market forecast report may include information on the competitive landscape, profiling key market players, their product offerings, partnerships, and strategies, and helping stakeholders understand the competitive dynamics of the WHIM market.

This product will be updated with the latest data at the time of order. Consequently, dispatch time for this product will be 7-10 business days.