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시장보고서
상품코드
1705096
WHIM 증후군 시장 - 시장 인사이트, 역학, 시장 예측(2034년)WHIM Syndrome - Market Insight, Epidemiology, and Market Forecast - 2034 |
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이 보고서는 미국, EU 4개국 및 영국(독일, 프랑스, 이탈리아, 스페인), 일본의 WHIM, 역사적 및 예측 역학, WHIM 시장 동향에 대한 상세한 이해를 제공합니다.
또한, 현재 치료법, 신약, 개별 치료법의 시장 점유율, 2020년에서 2034년까지 주요 7개국 WHIM 시장 규모 현황 및 예측을 제공합니다. 또한, 현재 WHIM 치료 관행, 시장 촉진요인, 시장 장벽, SWOT 분석, 상환 및 시장 접근성, 미충족 의료 수요 등을 다루며, 최상의 기회를 선별하고 시장 잠재력을 평가합니다.
조사 기간 : 2020-2034년
WHIM 증후군 치료 시장
WHIM 증후군은 CXCR4 유전자의 돌연변이로 인해 발생하는 희귀한 유전병으로 면역 결핍, 사마귀, 저감마글로불린혈증의 세 가지 증상을 유발하며, 종종 골수이영양증(호중구가 골수에 포획되는 상태)을 동반합니다. WHIM은 이 증후군의 주요 증상인 사마귀, 저감마글로불린혈증, 감염, 골수이형성증의 머리글자를 딴 것으로, 이 증후군은 면역기능을 현저하게 저하시켜 감염에 쉽게 걸리게 합니다.
WHIM 증후군은 몇 가지 특징적인 증상을 보이는데, WHIM 증후군 환자는 면역력이 약해져 면역기능이 저하되어 감염에 취약해집니다. 또 다른 두드러진 증상은 피부와 점막에 지속적이고 반복적인 사마귀가 생기는 것입니다. 저감마글로불린혈증은 혈액 내 면역글로불린(항체) 수치가 감소하여 면역계 기능 장애를 더욱 악화시키는 것이 특징입니다. 또한, 골수에 호중구가 축적되지만 혈류에서는 감소하는 골수이형성증은 WHIM 증후군의 중요한 특징입니다. 이러한 증상은 CXCR4 유전자의 돌연변이로 인해 발생하며, 감염에 효과적으로 대응하고 전반적인 건강을 유지하는 신체의 능력에 영향을 미칩니다.
WHIM 증후군의 진단은 재발성 감염, 사마귀, 저감마글로불린혈증, 골수이형성증 등의 증상에 대한 임상적 평가와 CXCR4 유전자의 돌연변이를 확인하기 위한 유전자 검사가 필요합니다. 유전자 검사는 확진 진단을 위해 매우 중요하지만, 이 과정에는 미충족 수요가 있습니다. 증상이 다른 질환과 중복될 수 있기 때문에 많은 환자들이 인식 부족과 과소 진단으로 인해 진단이 늦어지는 경우가 많습니다.
WHIM 증후군의 조기 개입과 효과적인 관리를 위해서는 인식 제고, 유전자 검사에 대한 폭넓은 접근성, 진단 도구의 강화를 통해 이러한 격차를 해소하는 것이 필수적입니다.
WHIM 증후군의 치료는 증상 관리와 근본적인 유전자 이상에 대한 대처에 초점을 맞추고 있습니다. 현재 치료법으로는 호흡기질환에 대한 지속적 양압호흡요법(CPAP), 감염 및 면역부전에 대한 약물 치료 등이 있으며, XOLREMDI(mavorixafor)와 같은 CXCR4 길항제는 CXCR4 수용체를 억제하여 근본적인 원인을 겨냥하여 면역기능을 개선하고 증상을 완화할 수 있습니다. 면역기능을 개선하고 증상을 완화시킬 수 있습니다. 또한, 유전자 변이를 교정하는 장기적인 해결책으로 유전자 치료가 검토되고 있습니다.
그러나 CPAP 및 감염 관리와 같은 현재 치료법을 준수하는 것은 환자들에게 어려운 일이며, 현재 사용 가능한 많은 치료법은 이 증후군의 복잡한 증상에 부분적으로만 대응하고 있으며, 개별화된 치료 옵션이 부족합니다. WHIM 증후군 환자의 예후와 삶의 질을 개선하기 위해서는 효과적인 맞춤 치료에 대한 더 많은 연구와 새로운 치료법에 대한 접근성 개선이 필수적입니다.
본 보고서의 WHIM 역학 장에서는 2020년부터 2034년까지 미국, EU 4개국(독일, 프랑스, 이탈리아, 스페인), 영국, 일본 등 주요 7개국을 대상으로 WHIM 증후군 진단 유병자 수, 성별에 따른 WHIM 증후군 진단 유병자 수, 연령별 WHIM 증후군 진단 유병자 수, 연령에 따른 WHIM 증후군의 진단 유병자 수를 세분화하여 과거 역학 및 예측을 보여줍니다.
XOLREMDI(mavorixafor) : X4 Pharmaceuticals
XOLREMDI(mavorixafor)는 선택적 CXCR4 수용체 길항제로 작용하는 최초의 경구용 저분자 약물로, WHIM 증후군 환자를 위한 최초의 FDA 승인 캡슐입니다. 마볼릭사폴은 골수에서 백혈구의 동원과 수송을 촉진하며, WHIM 증후군 외에도 중증 선천성 호중구감소증(SCN), 와르덴슈트렘 거대글로불린혈증, 유방암, 악성 흑색종, 세뇨관 신세포암(ccRCC)에 대한 적응증으로 검토되고 있습니다.
XOLREMDI는 규제 당국의 마일스톤으로 큰 주목을 받고 있습니다. 미국 FDA는 성인 WHIM 증후군 치료제로 2020년 10월 Fast Track Designation(FTD), 2019년 11월 Breakthrough Therapy Designation(BTD)을 승인했습니다. 또한, 2018년 10월에는 FDA로부터 희귀질환 치료제 지정(ODD)을, 2020년 12월에는 희귀 소아질환 지정(ODD)을 받았습니다. 유럽연합 집행위원회도 2019년 7월 WHIM 증후군에 대해 ODD를 부여했습니다.
XOLREMDI는 2024년 60%의 점유율을 달성할 것으로 예상되며, 정점에 도달하기까지 6년이 소요될 것으로 예상되며, 중속으로 흡수될 것으로 보입니다.
미국 FDA의 승인을 받은 프레릭사포르(MOZOBIL, AMD3100)는 비호지킨림프종 및 다발성 골수종 환자의 자가 이식을 위해 CD34 조혈모세포를 동원하는 치료제입니다. 또한, CXCR4를 부분적으로 억제하여 WHIM 증후군에 대응하며, 줄기세포 동원보다 낮은 용량으로 사용됩니다. 미국 국립알레르기감염병연구소는 WHIM 증후군에 대한 plerixafor의 개발을 진행하고 있으며, 임상 3상 시험을 완료하였습니다. 그 기전은 CXCR4 수용체를 특이적이고 가역적으로 억제하여 간질세포유래인자-1(SDF-1)의 결합을 억제하고 세포 접착에 영향을 미친다는 것입니다.
그러나 2021년 임상 II/III상 시험이 종료된 이후, 이 약에 대한 최신 정보나 추가 진전이 보고되지 않고 있습니다. 이러한 정체는 개발 파이프라인과 규제 상황에서 개발이 중단되거나 진전이 없을 수 있음을 시사합니다.
2024년 WHIM 증후군에 대한 첫 번째 치료제가 승인되면서 WHIM의 시장 전망은 확대될 것으로 예상됩니다.
WHIM 증후군의 치료에는 약리학적 접근과 비약리학적 접근이 모두 포함됩니다. 약리학적 치료에는 주로 CXCR4 신호전달을 부분적으로 차단하여 호중구감소증 관리에 도움을 주는 CXCR4 수용체 길항제인 프레릭사포르(MOZOBIL)가 포함됩니다. 이는 백혈구 동원을 촉진하기 위해 과립구 집락 자극 인자(G-CSF)와 병용하는 경우가 많습니다. 또한, WHIM 증후군의 특징적인 면역 결핍으로 인한 감염을 관리하기 위해 항생제와 항진균제를 처방하기도 합니다.
약물 치료 이외의 측면에서는 감염증의 관리와 예방을 위한 정기적인 모니터링을 포함한 지지요법이 중요합니다. 위생 관리를 철저히 하고 감염 위험을 최소화하는 등의 생활습관 조절도 권장됩니다. 또한, 환자가 만성질환을 앓고 있는 데 따른 정서적, 심리적 어려움에 대처할 수 있도록 심리사회적 지원도 중요합니다. 이러한 전략의 조합은 WHIM 증후군 환자의 전반적인 건강 결과와 삶의 질을 개선하는 것을 목표로 하고 있습니다.
본 보고서는 주요 7개국 WHIM 증후군 시장에 대해 조사했으며, 시장 개요, 역학, 환자 동향, 새로운 치료법, 2034년까지의 시장 규모 예측, 미충족 의료 수요 등을 조사하여 전해드립니다.
DelveInsight's "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.
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.
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 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), Waldenstrom'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 DelveInsight's 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.
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.
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 Deu Research, Spain, and others.
Delveinsight's 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
Key Questions Answered In The WHIM Syndrome Market Report
WHIM Syndrome Market Insights
WHIM Syndrome Epidemiology Insights
Current WHIM Syndrome Treatment Scenario, Marketed Drugs, and Emerging Therapies
Reasons to Buy WHIM Syndrome Market 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.
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.
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.
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.
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.
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.