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1705096

WHIM 증후군 시장 - 시장 인사이트, 역학, 시장 예측(2034년)

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

발행일: | 리서치사: DelveInsight | 페이지 정보: 영문 200 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




■ 보고서에 따라 최신 정보로 업데이트하여 보내드립니다. 배송일정은 문의해 주시기 바랍니다.

주요 하이라이트

  • WHIM 증후군은 CXCR4 돌연변이에 의해 유발되는 희귀 유전질환으로 면역결핍, 사마귀, 저감마글로불린혈증, 골수이형성증을 유발하는 희귀 유전질환으로, 2023년 주요 7개국에서 진단된 유병자 수는 약 179명으로 추산됩니다.
  • WHIM은 미국에서 심각한 건강 문제로 지난 수십 년 동안 그 유병률이 계속 증가하고 있으며, 12E Insight의 추정에 따르면, 주요 7개국 중 미국에서만 WHIM 환자 수가 68%로 가장 많으며, 2023년에는 프랑스가 그 뒤를 이었습니다.
  • 12elveInsight의 애널리스트별 평가에 따르면, EU 4개국과 영국에서 남녀 모두 WHIM 증후군으로 진단받은 유병자 수는 2023년 남성 19명, 여성 29명으로 향후 몇 년 동안 증가할 것으로 예측됩니다.
  • WHIM 증후군의 시장 규모는 질병의 유병률, 새로운 치료법의 가용성 및 채택, 규제 당국의 승인, 치료 비용, 질병에 대한 전반적인 인식 등 다양한 요인의 영향을받습니다. 또한, 진단 및 맞춤형 의료의 발전도 시장 역학에 영향을 미칠 수 있습니다.
  • 2023년 WHIM 시장 규모는 미국이 주요 7개국 중 가장 큰 시장으로 약 480만 달러로 2034년까지 더욱 확대될 것으로 예상됩니다.
  • 일본의 WHIM 시장 규모는 2023년 약 30만 달러로 주요 7개국 전체 시장의 5%에 불과합니다. 주요 7개국 전체 시장 규모는 2034년까지 CAGR 16.4%로 상승할 것으로 예상됩니다.
  • WHIM 증후군의 파이프라인은 매우 제한적이며, 이는 새로운 치료법의 연구 개발이 활발히 이루어지지 않고 있음을 보여줍니다. 이러한 희소성은 이 희귀 질환을 해결하기 위한 투자와 혁신의 필요성을 강조하고 있으며, WHIM의 총 시장 규모는 새로운 치료법의 진입이 예상됨에 따라 예측 기간 동안 급증할 것으로 예상됩니다.
  • 2024년 4월, 미국 FDA는 X4 Pharmaceuticals의 XOLREMDI(mavorixafor) 캡슐을 WHIM 증후군 치료제로 승인하여 이 질환에 대한 최초의 공식 승인 치료제로 만들었습니다. 이 획기적인 치료제는 WHIM 증후군의 근본 원인인 CXCR4 경로의 기능 장애를 치료하는 약물로, 임상 3상 데이터에 대한 우선 심사 승인을 받았습니다. 또한, X4는 희귀 소아질환 우선 심사 품목으로 지정되었습니다. 이번 승인은 12세 이상의 환자들에게 순환 성숙 호중구와 림프구를 증가시키는 새로운 치료 옵션을 제공하는 중요한 진전입니다.

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.

대상 지역

  • 미국
  • EU 4개국(독일, 프랑스, 이탈리아, 스페인) 및 영국
  • 일본

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.

  • 미국에서 진단된 WHIM 유병자 수는 2023년 약 121명으로 예측 기간 동안 증가할 것으로 예상됩니다.
  • WHIM 진단을 받은 유병자 수는 미국이 주요 7개국 중 가장 많으며, EU 4개국, 영국, 일본이 각각 전체 WHIM의 27%, 6%를 차지합니다.
  • 예측 평가에 따르면, WHIM 증후군으로 진단받은 유병률은 증가하고 있으며, 미국에서는 2023년까지 남성 약 48명, 여성 약 73명으로 증가할 것으로 예상됩니다. 이러한 추세는 예측 기간 동안 예상되는 남녀 모두의 증가를 반영하고 있습니다.
  • EU 4개국과 영국에서 2023년 WHIM 진단을 받은 환자 수는 약 48명입니다. 이 중 프랑스에서 가장 많은 사례가 발생했습니다.

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.

  • 프레릭사포(모조비르)와 같은 새로운 치료제의 출시는 시장에 긍정적인 영향을 미칠 것으로 예상됩니다. 이들 치료제가 승인되면 시장 역학에 큰 영향을 미칠 수 있지만, 그 성공률은 아직 불투명합니다.
  • 주요 7개국의 WHIM 시장 규모는 2023년 약 630만 달러에 달했으며, 예측 기간 동안 더욱 확대될 것으로 예상됩니다.
  • EU 4개국과 영국은 2023년 WHIM 시장 규모 약 130만 달러를 차지했습니다.
  • EU 국가 중 프랑스가 2023년 약 40만 달러로 시장 규모가 가장 크고, 스페인은 2023년 10만 달러로 WHIM 시장 규모가 가장 작습니다.
  • 프레릭사포(모조비르) 등 향후 치료제 출시가 예상됨에 따라 WHIM 시장 규모는 향후 몇 년 동안 변화를 보일 것으로 예상됩니다.

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.

목차

제1장 중요한 인사이트

제2장 보고서 소개

제3장 WHIM 증후군 시장 개요

  • WHIM 증후군 시장 점유율(%) 분포(2020년)
  • WHIM 증후군 시장 점유율(%) 분포(2034년)

제4장 역학과 시장 예측 조사 방법

제5장 주요 사건

제6장 WHIM 증후군 주요 요약

제7장 질환 배경과 개요 : WHIM 증후군

  • 소개
  • 임상적 특징
  • 원인
  • 면역 병태
  • 조직병리학 목표 소견
  • 진단
  • 진단 알고리즘
  • 치료

제8장 환자 동향

제9장 WHIM 증후군 역학과 환자 인구

  • 주요 조사 결과
  • 가정과 근거 : 주요 7개국
    • 미국
    • EU 4개국과 영국
    • 일본
  • 주요 7개국의 WHIM 증후군 유병률 높은 진단 환자
  • 미국
    • 미국의 WHIM 증후군 유병률
    • 미국의 WHIM 증후군 유병률, 성별
    • 미국의 WHIM 증후군 유병률, 연령별
  • EU 4개국국과 영국
    • 독일
    • 프랑스
    • 이탈리아
    • 스페인
    • 영국
  • 일본
    • 일본의 WHIM 증후군 유병률
    • 일본의 WHIM 증후군 유병률, 성별
    • 일본의 WHIM 증후군 유병률, 연령별

제10장 WHIM 증후군 시판 치료

  • XOLREMDI(mavorixafor) : X4 Pharmaceuticals
    • 제품 프로파일
    • 기타 개발 활동
    • 임상 개발
    • 임상시험 정보
    • 안전성과 유효성

제11장 WHIM 증후군의 새로운 치료법

제12장 WHIM 증후군 : 주요 7개국 시장 분석

  • 주요 조사 결과
  • 시장 전망
  • 주요 시장 예측 가정
  • 주요 7개국의 WHIM 증후군 시장 규모
  • 주요 7개국의 WHIM 증후군 시장 규모(치료법별)
  • 미국의 WHIM 증후군 시장 규모
  • EU 4개국과 영국의 WHIM 증후군 시장 규모
  • 일본의 WHIM 증후군 시장 규모

제13장 주요 오피니언 리더의 견해

제14장 SWOT 분석

제15장 미충족 수요

제16장 시장 접근과 상환

  • 미국
    • 메디케어·메디케이드 서비스 센터(CMS)
  • EU 4개국과 영국
    • 독일
    • 프랑스
    • 이탈리아
    • 스페인
    • 영국
  • 일본
    • 후생 노동성

제18장 부록

제19장 DelveInsight의 서비스 내용

제20장 면책사항

ksm 25.04.22

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 DelveInsight estimates, 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 DelveInsight's 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.

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.

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 DelveInsight's estimates, 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), 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.

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 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

  • 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.

Table of Contents

1 Key Insights

2 Report Introduction

3 WHIM Syndrome Market Overview at a Glance

  • 3.1 Market Share (%) Distribution of WHIM Syndrome in 2020
  • 3.2 Market Share (%) Distribution of WHIM Syndrome in 2034

4 Epidemiology and Market Forecast Methodology

5 Key Events

6 Executive Summary of WHIM Syndrome

7 Disease Background and Overview: WHIM syndrome

  • 7.1 Introduction
  • 7.2 Clinical Features
  • 7.3 Causes
  • 7.4 Immunopathogenesis
  • 7.5 Histopathological Findings
  • 7.6 Diagnosis
  • 7.6 1 Diagnostic Algorithm
  • 7.7 Treatment

8 Patient Journey

9 Epidemiology and Patient Population of WHIM Syndrome

  • 9.1 Key Findings
  • 9.2 Assumptions and Rationale: The 7MM
    • 9.2.1 The United States
    • 9.2.2 EU4 and the UK
    • 9.2.3 Japan
  • 9.3 Diagnosed Prevalent Cases of WHIM Syndrome in the 7MM
  • 9.4 The United States
    • 9.4.1 Diagnosed Prevalent Cases of WHIM Syndrome in the US
    • 9.4.2 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in the US
    • 9.4.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the US
  • 9.5 EU4 Countries and the UK
    • 9.5.1 Germany
      • 9.5.1.1 Diagnosed Prevalent Cases of WHIM Syndrome in Germany
      • 9.5.1.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Germany
      • 9.5.1.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Germany
    • 9.5.2 France
      • 9.5.2.1 Diagnosed Prevalent Cases of WHIM Syndrome in France
      • 9.5.2.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in France
      • 9.5.2.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in France
    • 9.5.3 Italy
      • 9.5.3.1 Diagnosed Prevalent Cases of WHIM Syndrome in Italy
      • 9.5.3.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Italy
      • 9.5.3.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Italy
    • 9.5.4 Spain
      • 9.5.4.1 Diagnosed Prevalent Cases of WHIM Syndrome in Spain
      • 9.5.4.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in Spain
      • 9.5.4.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Spain
    • 9.5.5 The United Kingdom
      • 9.5.5.1 Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom
      • 9.5.5.2 Gender-specific Diagnosed Prevalent Cases of WHIM syndrome in the United Kingdom
      • 9.5.5.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in the United Kingdom
  • 9.6 Japan
    • 9.6.1 Diagnosed Prevalent Cases of WHIM Syndrome in Japan
    • 9.6.2 Gender-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan
    • 9.6.3 Age-specific Diagnosed Prevalent Cases of WHIM Syndrome in Japan

10 WHIM Syndrome Marketed Therapies

  • 10.1 XOLREMDI (mavorixafor): X4 Pharmaceuticals
    • 10.1.1 Product Profile
    • 10.1.2 Other Developmental Activities
    • 10.1.3 Clinical Development
    • 10.1.4 Clinical Trials Information
    • 10.1.5 Safety and Efficacy
    • 10.1.6 Product Profile

11 Emerging WHIM Syndrome Therapies

  • 11.1 Key Cross Competition
  • 11.2 Plerixafor: National Institute of Allergy and Infectious Diseases (NIAID)
    • 11.2.1 Drug Description
    • 11.2.2 Regulatory Milestone
    • 11.2.3 Other Development Activities
    • 11.2.4 Clinical Trials Information
    • 11.2.5 Safety and Efficacy
    • 11.2.6 Analysts' views

12 WHIM Syndrome: Seven Major Market Analysis

  • 12.1 Key Findings
  • 12.2 Market Outlook
  • 12.3 Key Market Forecast Assumptions
  • 12.4 Market Size of WHIM Syndrome in the 7MM
  • 12.5 Market Size of WHIM Syndrome by Therapies in the 7MM
  • 12.6 Market Size of WHIM Syndrome in the United States
    • 12.6.1 Total Market Size of WHIM Syndrome
    • 12.6.2 Market Size of WHIM Syndrome by Therapies
  • 12.7 Market Size of WHIM Syndrome in EU4 and the UK
    • 12.7.1 Germany
      • 12.7.1.1 Total Market Size of WHIM Syndrome
      • 12.7.1.2 Market Size of WHIM Syndrome by Therapies
    • 12.7.2 France
      • 12.7.2.1 Total Market Size of WHIM Syndrome
      • 12.7.2.2 Market Size of WHIM Syndrome by Therapies
    • 12.7.3 Italy
      • 12.7.3.1 Total Market Size of WHIM Syndrome
      • 12.7.3.2 Market Size of WHIM Syndrome by Therapies
    • 12.7.4 Spain
      • 12.7.4.1 Total Market Size of WHIM Syndrome
      • 12.7.4.2 Market Size of WHIM Syndrome by Therapies
    • 12.7.5 The United Kingdom
      • 12.7.5.1 Total Market Size of WHIM Syndrome
      • 12.7.5.2 Market Size of WHIM Syndrome by Therapies
  • 12.8 Market Size of WHIM Syndrome in Japan
    • 12.8.1 Total Market Size of WHIM Syndrome
    • 12.8.2 Market Size of WHIM Syndrome by Therapies

13 Key Opinion Leaders' Views

14 SWOT Analysis

15 Unmet Needs

16 Market Access and Reimbursement

  • 17.1 The United States
    • 17.1.1 Centers for Medicare & Medicaid Services (CMS)
  • 17.2 In EU4 and the UK
    • 17.2.1 Germany
    • 17.2.2 France
    • 17.2.3 Italy
    • 17.2.4 Spain
    • 17.2.5 The United Kingdom
  • 17.3 Japan
    • 17.3.1 MHLW

18 Appendix

  • 18.1 Bibliography
  • 18.2 Acronyms and Abbreviations
  • 18.3 Report Methodology

19 DelveInsight Capabilities

20 Disclaimer

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