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이식편대숙주질환(GVHD) : 시장 인사이트, 역학 및 예측(2036년)

Graft Versus Host Disease - Market Insight, Epidemiology, and Market Forecast - 2036

발행일: | 리서치사: 구분자 DelveInsight | 페이지 정보: 영문 312 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




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이식편대숙주질환(GVHD)에 대한 인사이트 및 동향

  • 이식편대숙주질환(GVHD)은 동종 조혈모세포 이식(HSCT) 후에 발생하는 심각한 면역 합병증으로, 기증자의 면역 세포가 수혜자의 조직을 공격하는 질환입니다. 일반적으로 피부, 간, 소화관에 영향을 미치며, 높은 발병률을 초래할 뿐만 아니라 환자의 전반적인 예후와 삶의 질에도 영향을 미칩니다.
  • DelveInsight의 분석에 따르면, 주요 7개국(미국, EU 4개국, 영국, 일본)의 이식편대숙주질환(GVHD) 시장 규모는 2025년에 약 21억 달러로 평가되었습니다. 진단된 환자 수 증가와 새로운 치료법의 도입 확대에 힘입어, 예측 기간 동안 시장은 확대될 것으로 전망됩니다.
  • 미국은 여전히 가장 큰 시장 점유율을 차지하고 있으며, 2025년 매출액은 약 16억 달러였습니다. DelveInsight의 추산에 따르면, 이는 2036년까지 크게 증가할 것으로 보이며, 미국이 주요 7개국 전체 이식편대숙주질환(GVHD) 시장의 약 77%를 차지할 전망입니다.
  • DelveInsight의 조사에 따르면, 주요 7개국에서 2025년에 GVHD의 총 환자 수가 약 5만 2,000건으로 보고되었습니다. 조혈모세포 이식(HSCT) 시행 건수 증가와 생존율 향상을 배경으로, 환자 수는 2036년까지 증가할 것으로 예상되며, 이에 따라 해당 지역 전체의 의료 부담이 커질 것으로 전망됩니다.
  • 미국에서 DelveInsight의 조사에 따르면, 2025년 GVHD의 유형별 환자 수는 급성 GVHD가 약 4,300건, 만성 GVHD의 5년 유병 환자 수는 약 1만 8,500건으로 추정되며, 두 질환 형태 모두에서 상당한 부담이 발생하고 있는 것으로 나타났습니다.
  • 현재의 GVHD 예방 요법은 특히 고위험 이식 환경에서 그 유효성이 충분하지 않다는 한계가 있으며, 심각한 장기적 독성을 동반합니다. 표적을 명확히 한 지속적인 면역 관용 전략이 부재한 점이 계속해서 진전을 저해하고 있으며, 이에 따라 보다 안전하고 정밀한 예방 접근법의 필요성이 부각되고 있습니다.
  • GVHD의 치료 환경은 표적 치료 및 세포 치료를 통해 점차 형성되고 있으며, 룩솔리티닙(JAKAFI/JAKAVI), 이브루티닙(IMBRUVICA), 레메스템셀-L(RYONCIL/TEMCELL HS) 등의 주요 약제가 주요 7개국 전체 시장 역학을 주도하는 중심적인 역할을 하고 있습니다. 2025년에는 룩솔리티닙이 매출 1위를 차지하고, REZUROCK이 그 뒤를 이을 전망입니다.
  • GVHD(이식편대숙주질환)의 새로운 파이프라인은 새로운 면역 조절 요법 및 세포 요법에 초점을 맞추고 있으며, 주요 기업들이 각 임상시험 단계에서 후보 약물의 개발을 진행하고 있습니다. 특히, MaaT013(XERVYTEG), RGI-2001, CYP-001 등의 치료법이 향후 치료의 방향을 주도할 것으로 예상되며, 이 중 일부는 2028년까지 미국 시장에 진출할 것으로 전망됩니다.

이식편대숙주질환(GVHD) 시장 규모 및 전망

  • 주요 7개국(G7)의 이식편대숙주질환(GVHD) 시장 규모(2025년) : 약 21억 달러
  • 주요 7개국 이식편대숙주질환(GVHD) 시장 성장률(2026-2036년) : 연평균 성장률(CAGR) 6.4%

본 이식편대숙주질환(GVHD) 시장 보고서에서는 표준 치료, 임상 실무, 진화하는 치료 알고리즘 등 현재 시장 상황에 대한 종합적인 분석을 제공합니다. GVHD 환자의 부담 동향, 수익 및 시장 점유율 동향, 피크 시기의 환자 점유율 및 치료 도입 현황에 대한 분석을 평가함과 동시에, 주요 7개 국가 및 지역 전체에 걸친 상세한 시장 규모 평가 및 성장률 예측(과거 데이터 및 2026-2036년 예측)을 제시하고 있습니다. 본 보고서에서는 GVHD 분야의 주요 미충족 수요 사항을 부각시키고, 경쟁 구도 및 임상 현황을 분석하여 고부가가치 기회를 도출함으로써, 향후 시장 성장 가능성에 대한 명확한 전망을 제시하고 있습니다.

대상 지역 :

북미 : 미국

유럽 : 독일, 프랑스, 이탈리아, 스페인, 영국

아시아태평양 : 일본

이식편대숙주질환(GVHD)의 이해와 치료 알고리즘

GVHD의 개요와 진단

GVHD는 동종 조혈모세포 이식(HSCT) 후 발생하는 복잡한 면역매개성 질환으로, 기증자 유래 면역세포가 수혜자의 조직을 이물질로 인식하여 면역 반응을 일으키는 것입니다. 이 질환은 여러 장기에 영향을 미칠 수 있으며, 가장 흔한 부위는 피부, 간, 소화관입니다. 또한, 크게 급성형과 만성형으로 분류됩니다. GVHD의 임상 양상은 중증도나 진행 양상에 따라 크게 다르며, 경미한 국소 병변부터 환자의 예후와 삶의 질에 중대한 영향을 미치는 심각한 전신성 합병증에 이르기까지 그 증상은 매우 다양합니다.

GVHD를 진단하기 위해서는 임상 평가와 조직병리학적 소견 및 검사 소견을 종합한 종합적이고 체계적인 접근이 필요합니다. 우선, 병변이 발생한 장기에 나타나는 특징적인 징후나 증상을 파악하는 것부터 시작하며, 대부분의 경우 조직 생검을 통해 확인합니다. 검사 매개변수 및 영상 검사를 포함한 추가 평가는 장기 병변 및 질환의 중증도 평가를 보완하여, 정확한 분류와 시기적절한 임상적 판단을 가능하게 합니다.

현재 이식편대숙주질환(GVHD) 치료의 현황

GVHD의 치료 현황은 근본적인 면역 조절 이상에 대처하는 것을 목적으로 하는 표적 면역 조절 요법 및 세포 요법의 발전에 힘입어 꾸준히 진화하고 있습니다. 현재 표준 치료의 주요 구성 요소에는 GVHD의 병태생리에 관여하는 사이토카인 신호전달 경로를 조절하는 JAK1/JAK2 억제제인 JAKAFI/JAKAVI가 포함됩니다. 또한, B세포의 신호 전달 및 면역 활성화를 표적으로 하는 브루톤형 티로신 키나제 억제제인 'IMBRUVICA'도 특정 상황에서 사용되고 있습니다. 또한, 간엽계 지지 세포를 이용한 치료법인 ‘RYONCIL/TEMCELL HS’는 면역 조절 작용을 가진 첨단 치료법입니다. 이러한 치료법들은 전반적으로 GVHD 분야에서 지속되는 혁신과, 보다 표적화된 기전에 기반한 접근 방식으로의 전환을 반영하며, 끊임없이 진화하는 치료 패러다임에 기여하고 있습니다.

이식편대숙주질환(GVHD)의 역학

GVHD의 역학 분석 및 예측에 관한 주요 연구 결과

  • 2025년에는 주요 7개국 전체에서 등급 B(II) 급성 GVHD가 약 7,100건, 그 다음으로 등급 C(III)가 약 1,800건, 등급 D(IV)가 약 700건으로 추산되며, 이러한 수치는 2036년까지 증가할 것으로 예측됩니다.
  • 미국에서 2025년 장기별 급성 GVHD 사례 수는 피부가 약 3,700건으로 가장 많았고, 그 다음으로 소화관이 약 2,100건, 간이 약 1,300건으로 나타났으며, 이를 통해 이 질환이 여러 장기에 영향을 미치고 있음을 알 수 있습니다.
  • 일본에서는 2025년 만성 GVHD의 장기별 사례 수가 주로 구강 점막에서 약 3,100건, 폐에서 약 2,400건이 관찰되었으며, 그 다음으로 눈에서 약 1,900건, 피부에서 약 4,000건, 관절 및 근막에서 약 400건, 생식기에서 약 300건, 소화관에서 약 1,000건, 간에서 약 3,000건으로 나타나며, 다양한 다기관에 걸친 침범이 반영되었습니다.

이식편대숙주질환(GVHD) 시장 전망

GVHD는 동종 조혈모세포 이식(HSCT) 후 발생하는 중증이며 생명을 위협할 가능성이 있는 면역매개성 질환으로, 다발성 장기 손상을 유발하며, 가장 흔히 피부, 간, 그리고 소화관에 영향을 미칩니다. 이는 기증자의 면역 세포가 수혜자의 조직을 공격함으로써 발생하며, 심각한 발병률과 장기적인 합병증을 초래합니다. GVHD에는 급성형과 만성형이 있으며, 각각 중증도와 임상 증상이 다릅니다. 이러한 복잡한 면역 병태, 생존율 및 삶의 질에 미치는 영향, 그리고 주요 7개국 전체에서 이식 건수가 증가함에 따라 환자의 부담이 커지고 있다는 점으로 미루어 볼 때, 이 질환은 여전히 큰 과제로 남아 있습니다.

현재의 치료법을 형성하는 주요 시판 치료제

  • 벨모스딜(REZUROCK; 사노피(카도몬)/메이지제과) : 2021년 7월 미국 FDA로부터, 적어도 2가지 전신 요법에 반응하지 않은 만성 GVHD에 대해 승인된 경구 투여용 퍼스트-인-클래스 선택적 ROCK2 억제제로, 이후 다른 지역에서도 승인되었습니다. STAT3의 발현을 억제하고 STAT5의 발현을 촉진함으로써, 제17형 및 여포성 T 헬퍼 세포를 감소시키는 동시에 조절 T 세포의 기능을 강화합니다.
  • 악사틸리맙-CSFR(NIKTIMVO; 인사이트/신덱스) : CSF-1R을 표적으로 하는 퍼스트-인-클래스 단일클론 항체로, 2024년 8월, 최소 2가지 전신 치료에서 충분한 반응을 얻지 못한 만성 GVHD에 대해 미국 FDA의 승인을 획득했습니다. 단핵구 및 대식세포에서 CSF-1R 신호전달을 억제함으로써, 만성 GVHD의 주요 원인이 되는 병리적 염증 및 섬유화를 완화합니다.

전반적으로 볼 때, 이식편대숙주질환(GVHD) 시장은 중요한 전환점에 서 있습니다. 기존에는 CS(화학요법)를 중심으로 한 경험적 다층 면역억제 요법이 주를 이루었으나, 그 관리 방식은 오랫동안 지속성이 부족한 반응적이며 증상 중심의 전략에 의존해 왔습니다. 현재, 정밀 생물학적 제제, 세포 기반 면역 조절제, 미생물군집 회복제에 이르는 기전 특이적인 차세대 치료법이 2022년부터 2036년까지 주요 7개국 이식편대숙주질환(GVHD) 시장에서 예방, 면역 관용, 조직 복구를 통해 그 양상을 새롭게 바꾸고 있으며, 기존 시판 제품과 신흥 파이프라인 양쪽 모두에 큰 상업적 영향을 미치고 있습니다.

  • 2025년, 미국은 이식편대숙주질환(GVHD) 시장 전체에서 7대 주요 국가 중 독보적인 위치를 차지하며 전체 시장 점유율의 77%를 기록했습니다. 미국 시장 규모는 21억 달러로 평가되고 있으며, 이는 주로 신흥 치료법이 시장 성장에 미칠 것으로 예상되는 영향에 힘입은 결과입니다.
  • 저분자 화합물 : 저분자 화합물은 GVHD 치료 분야에서 핵심적인 역할을 수행하며, 주요 면역 신호 전달 경로를 표적으로 삼아 조절합니다. JAK 억제제, ROCK2 억제제, 알킬화제 등의 시판 약제가 현재 임상 현장을 계속해서 형성해 나가고 있는 반면, RGI-2001이나 이바르마시티닙(SHR0302) 등의 신흥 치료법은 면역 관용과 사이토카인 조절에 초점을 맞춘 차세대 전략을 추진하고 있습니다.
  • 단일클론 항체 : 단일클론 항체는 면역세포의 하위 집합이나 염증 경로를 정밀하게 표적으로 삼을 수 있기 때문에 GVHD 치료 분야의 혁신을 주도하고 있습니다. MEDI-5071(시프리주맙)이나 이트리주맙(EQ001)과 같은 신약들은 T세포의 활성을 선택적으로 조절하기 위해 개발이 진행되고 있는 반면, 악사틸리맙-CSFR(NIKTIMVO)과 같은 승인된 치료법은 이 약물군의 발전을 더욱 두드러지게 하고 있습니다. 이러한 생물학적 제제는 표적에 국한된 면역 억제, 오프타겟 효과의 감소, 그리고 지속적인 질환 관리 가능성을 통해 임상적 결과를 개선하는 것을 목표로 하고 있습니다.

자주 묻는 질문

  • 이식편대숙주질환(GVHD)의 시장 규모는 어떻게 되나요?
  • 미국의 이식편대숙주질환(GVHD) 시장 점유율은 어떻게 되나요?
  • 이식편대숙주질환(GVHD)의 환자 수는 어떻게 변화할 것으로 예상되나요?
  • 이식편대숙주질환(GVHD)의 치료 환경은 어떻게 변화하고 있나요?
  • 이식편대숙주질환(GVHD)의 주요 치료제는 무엇인가요?
  • 이식편대숙주질환(GVHD)의 급성형과 만성형 환자 수는 어떻게 되나요?

목차

제1장 주요 인사이트

제2장 서론

제3장 이식편대숙주질환(GVHD) : 시장 개요

제4장 주요 요약

제5장 주요 이벤트

제6장 질환 개요와 배경

제7장 역학 및 예측 조사 방법

제8장 역학 및 환자 인구

제9장 환자 경과

제10장 시판 치료제

제11장 신흥 치료제

제12장 이식편대숙주질환(GVHD) : 주요 7개국 분석

제13장 KOL(Key Opinion Leader)의 견해

제14장 미충족 수요

제15장 SWOT 분석

제16장 시장 참여 및 상환

제17장 부록

제18장 DelveInsight의 서비스 내용

제19장 면책사항

제20장 DelveInsight에 대해

KTH

Graft versus Host Disease (GvHD) Insights and Trends

  • Graft versus Host Disease (GvHD) is a serious immune complication following allogeneic HSCT, where donor immune cells attack recipient tissues, commonly affecting skin, liver, and gastrointestinal tract, leading to significant morbidity and impacting overall patient outcomes and quality of life.
  • According to DelveInsight's analysis, the GvHD market in the 7MM (the US, EU4, the UK, and Japan) was valued at approximately USD 2.1 billion in 2025. The market is expected to expand over the forecast period, driven by a growing diagnosed patient population and increasing adoption of emerging therapeutic approaches.
  • The US continues to hold the largest market share, with a revenue of ~USD 1.6 billion in 2025. DelveInsight estimates this will rise significantly by 2036, with the US expected to account for approximately 77% of the total GvHD market across the 7MM.
  • As per DelveInsight research, the 7MM reported approximately 52,000 total cases of GvHD in 2025. The patient pool is expected to increase through 2036, driven by the rising number of HSCT procedures and improved survival rates, contributing to a growing burden across these regions.
  • In US, as per DelveInsight research, type-specific cases of GvHD in 2025 included approximately 4,300 cases of acute GvHD, while the five-year prevalent cases of chronic GvHD were estimated at around 18,500, reflecting a substantial burden across both disease forms.
  • Current GvHD prophylaxis is limited by suboptimal efficacy, particularly in high-risk transplant settings, and is associated with significant long-term toxicities; the absence of targeted, durable immune tolerance strategies continues to constrain progress, underscoring the need for safer, more precise prophylactic approaches.
  • The GvHD therapeutic landscape is increasingly shaped by targeted and cell-based therapies, with key agents such as ruxolitinib (JAKAFI/JAKAVI), ibrutinib (IMBRUVICA), and remestemcel-L (RYONCIL/TEMCELL HS), among others playing a central role in driving market dynamics across the 7MM. In 2025, ruxolitinib leads in revenue generation, followed by REZUROCK
  • The emerging pipeline for GvHD is focused on novel immunomodulatory and cell-based approaches, with key players advancing assets across clinical trial stages. Notably, therapies such as MaaT013 (XERVYTEG), RGI-2001, CYP-001, and others are expected to shape the future landscape, with some anticipated to enter the US market by 2028.

Graft versus Host Disease (GvHD) Market size and forecast

  • 2025 GvHD Market Size in 7MM: ~USD 2.1 billion
  • GvHD Growth Rate (2026-2036) in the 7MM: 6.4% CAGR

DelveInsight's 'Graft versus Host Disease (GvHD) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of GvHD, historical and forecasted epidemiology, as well as the market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.

The GvHD market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates, GvHD patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment and growth rate projections (Historical & Forecast 2026 -2036) across the 7MM regions. The report highlights key unmet medical needs in GvHD and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

Geography Covered:

North America: The United States

Europe: Germany, France, Italy, and Spain and the UK

Asia-Pacific: Japan

Graft versus Host Disease (GvHD) Understanding and Treatment Algorithm

GvHD Overview and Diagnosis

GvHD refers to a complex immune-mediated condition that occurs following allogeneic HSCT, in which donor-derived immune cells recognize recipient tissues as foreign and initiate an immune response. This condition can affect multiple organs, most commonly the skin, liver, and gastrointestinal tract, and is broadly classified into acute and chronic forms. The clinical presentation of GvHD varies widely in severity and progression, with manifestations ranging from mild localized involvement to severe, systemic complications that significantly impact patient outcomes and quality of life.

The diagnosis of GvHD requires a comprehensive and systematic approach, integrating clinical evaluation with histopathological and laboratory findings. It begins with the identification of characteristic signs and symptoms across affected organs, followed by confirmation through tissue biopsy in many cases. Additional assessments, including laboratory parameters and imaging studies, support the evaluation of organ involvement and disease severity, ensuring accurate classification and timely clinical decision-making.

Current Graft versus Host Disease (GvHD) Treatment Landscape

The overall GvHD landscape is steadily evolving, driven by advances in targeted immunomodulatory therapies and cell-based approaches aimed at addressing the underlying immune dysregulation. Key components of current standard practice include JAKAFI/JAKAVI, a JAK1/JAK2 inhibitor that modulates cytokine signaling pathways implicated in GvHD pathophysiology. IMBRUVICA, a Bruton's tyrosine kinase inhibitor, is also utilized in specific settings, targeting B-cell signaling and immune activation. Additionally, RYONCIL/TEMCELL HS, a mesenchymal stromal cell-based therapy, represents an advanced therapeutic modality with immunomodulatory properties. These therapies collectively contribute to the evolving treatment paradigm, reflecting ongoing innovation and a shift toward more targeted and mechanism-based approaches in GvHD.

Graft versus Host Disease (GvHD) Unmet Needs

The section "unmet needs of GvHD" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.

1. Improved early diagnosis and risk stratification

2. Limited understanding of cGvHD pathophysiology

3. Lack of optimized combinatorial therapies

4. Need for novel therapies

Graft versus Host Disease (GvHD) Epidemiology

Key Findings from GvHD Epidemiological Analysis and Forecast

  • In 2025, there were an estimated ~7,100 cases of Grade B (II) acute GvHD, followed by ~1,800 cases of Grade C (III) and ~700 cases of Grade D (IV) across the 7MM, with these numbers expected to increase by 2036.
  • In US, acute GvHD cases by organ involvement in 2025 were primarily observed in the skin with ~3,700 cases, followed by the gastrointestinal tract with ~2,100 cases and the liver with ~1,300 cases, reflecting the multi-organ impact of the disease.
  • In Japan, chronic GvHD cases by organ involvement in 2025 were primarily observed in the oral mucosa with ~3,100 cases and lungs with ~2,400 cases, followed by the eye with ~1,900 cases, skin with ~4000 cases, joints and fascia with ~400 cases, genitals with ~300 cases, gastrointestinal tract with ~1,000 cases, and liver with ~3,000 cases, reflecting diverse multi-organ involvement.

Graft versus Host Disease (GvHD) Drug Analysis & Competitive Landscape

The GvHD drug chapter provides a detailed, market-focused review of approved therapies and the emerging pipeline across Phase I - Phase III clinical trials. It covers mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, strategic partnerships upcoming Key catalyst for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the GvHD treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the GvHD therapeutics market.

Approved Therapies for GvHD

Ruxolitinib (JAKAFI/JAKAVI): Incyte/Novartis

Ruxolitinib is an oral selective JAK1/2 inhibitor developed by Incyte and co-marketed by Novartis. It modulates the JAK-STAT pathway to reduce cytokine-driven inflammation and is indicated for steroid-refractory acute and chronic GvHD after failure of one or two prior systemic therapies in patients aged 12 years and older.

  • In August 2023, Novartis reported that ruxolitinib was approved as JAKAVI in Japan for use in GvHD after a HSCT.
  • In May 2022, Incyte reported that the EC approved ruxolitinib (JAKAVI) for patients aged 12 years and older with aGvHD or cGvHD who have an inadequate response to corticosteroids or other systemic therapies.
  • In September 2021, Incyte reported that the US FDA approved ruxolitinib (JAKAFI) for cGvHD after failure of one or two prior systemic therapies in patients aged 12 years and older.

Ibrutinib (IMBRUVICA): AbbVie (Pharmacyclics)/Janssen

Ibrutinib is an oral, once-daily BTK inhibitor that blocks B-cell signaling and reduces abnormal immune activity. It is indicated for cGvHD after failure of one or more prior systemic therapies.

  • In August 2022, the US FDA approved IMBRUVICA for pediatric patients >=1-year of age with cGvHD after the failure of one or more lines of systemic therapy.
  • In September 2021, the PMDA approved IMBRUVICA to treat cGvHD after HSCT.
  • In August 2017, the US FDA approved IMBRUVICA for treating adult patients with cGvHD after the failure of one or more lines of systemic therapy.

Graft versus Host Disease (GvHD) Pipeline Analysis

MaaT013 (XERVYTEG): MaaT Pharma

MaaT013 is an off-the-shelf, pooled-donor microbiome therapy designed to restore gut microbiota balance and modulate immune tolerance in GI-predominant acute GvHD. It has received ODD from the US FDA and EMA, with a Phase III trial ongoing showing favorable efficacy and safety signals.

  • In July 2025, MaaT Pharma and Clinigen signed an exclusive long-term licensing, distribution agreement, and commercial supply agreement for MaaT013 for the treatment of patients with aGvHD.
  • In July 2025, MaaT Pharma reported that it has secured a Euro 37.5 million, 4-tranche financing from the European Investment Bank (EIB). The financing will support the advancement of its late-stage hemato-oncology clinical programs including the lead-asset MaaT013.
  • In June 2025, MaaT Pharma reported the submission of an MAA to the EMA for MaaT013 (XERVYTEG), with potential approval anticipated by mid-2026, positioning it as a first-in-class microbiota-based therapy for third-line steroid-refractory acute GvHD.

RGI-2001: REGiMMUNE

RGI-2001 is a liposomal alpha-GalCer formulation that induces regulatory T cells to promote immune tolerance while preserving normal immune function. It has shown potential to reduce GvHD and improve survival without compromising GvL effects and has received ODD from the US FDA.

  • In April 2024, REGiMMUNE reported a poster presentation at the AACR Annual Meeting highlighting Phase IIb results of RGI-2001, including analyses of Treg and NKT-cell dynamics to evaluate its role in preventing acute GvHD.
  • In February 2024, REGiMMUNE reported positive Phase IIb results for RGI-2001 in preventing acute GvHD after alloHCT, with plans to initiate a Phase III randomized study to further evaluate its efficacy and safety.
  • In March 2023, REGiMMUNE and San Fu Biotech entered a licensing agreement to develop and commercialize RGI-2001 for aGvHD prophylaxis in major Asian countries, collaborating to accelerate development and conduct clinical studies evaluating its efficacy.

Graft versus Host Disease (GvHD) Key Players, Market Leaders and Emerging Companies

  • AbbVie
  • Johnson and Johnson
  • Sanofi
  • Incyte
  • Syndax Pharmaceuticals
  • Novartis
  • Neovii
  • Mundipharma
  • Platinum Life Excellence Biotech
  • Shionogi
  • JCR Pharmaceuticals
  • Bristol Myers Squibb
  • And more

Graft versus Host Disease (GvHD) Drug Updates

  • A pivotal Phase III trial of MaaT013 in corticosteroid- and ruxolitinib-refractory GI acute GvHD is ongoing, with DSMB review of the ARES study indicating a favorable benefit-risk profile with high efficacy and low toxicity.
  • RGI-2001 completed a Phase II clinical trial (NCT04014790) in 2022 for the prevention of aGvHD in subjects following alloHSCT, and earlier completed a Phase I/II trial (NCT01379209) in 2015 in patients undergoing alloHSCT.
  • CYP-001 was evaluated in a global Phase II clinical trial (NCT05643638) in combination with corticosteroids versus corticosteroids alone for high-risk acute GvHD under a cleared US FDA IND.

Graft versus Host Disease (GvHD) Market Outlook

GvHD is a serious and potentially life-threatening immune-mediated condition that occurs following allogeneic HSCT, leading to multi-organ involvement, most commonly affecting the skin, liver, and gastrointestinal tract. It arises when donor immune cells attack recipient tissues, resulting in significant morbidity and long-term complications. GvHD can present as acute or chronic forms, each with variable severity and clinical manifestations. The condition remains a major challenge due to its complex immunopathology, impact on survival and quality of life, and the increasing number of transplant procedures contributing to a growing patient burden across the 7MM.

Key marketed therapies shaping current management

  • Belumosudil (REZUROCK; Sanofi (Kadmon)/Meiji Seika Pharmaceuticals): An oral, first-in-class selective ROCK2 inhibitor approved by the US FDA in July 2021 for chronic GvHD after failure of at least two prior systemic therapies, with subsequent approvals across other regions. By downregulating STAT3 and upregulating STAT5, it reduces Type 17 and follicular T-helper cells while enhancing regulatory T-cell function.
  • Axatilimab-csfr (NIKTIMVO; Incyte/Syndax): A first-in-class monoclonal antibody targeting CSF-1R, granted US FDA approval in August 2024 for chronic GvHD following inadequate response to at least two previous systemic treatments. Inhibiting CSF-1R signaling on monocytes and macrophages helps reduce pathogenic inflammation and fibrosis, central mechanisms driving chronic GvHD.

Overall, the GvHD market stands at a pivotal transformation point. Historically defined by CS-centered, empirically layered immunosuppression, management has long relied on reactive and symptom-driven strategies with limited durability. Now, a new generation of mechanism-specific therapies spanning precision biologics, cell-based immunomodulators, and microbiome-restorative agents is reshaping the landscape toward prevention, immune tolerance, and tissue repair in 7MM GvHD market from 2022-2036, with strong commercial implications for both marketed products and emerging pipelines.

  • In 2025, the US dominated the GvHD market across the 7MM, accounting for 77% of the total market share. The market size in the US was valued at USD 2.1 billion, driven largely by the anticipated impact of emerging therapies on market growth.

Drug Class/Insights into Leading Emerging and Marketed Therapies in Graft versus Host Disease (GvHD) (2022-2036 Forecast)

The GvHD landscape includes both established therapies and a growing pipeline targeting acute GvHD, chronic GvHD, and preventive strategies in the context of HSCT. These approaches primarily focus on modulating immune dysregulation and reducing harmful donor-derived immune activity responsible for tissue damage. Small molecules and monoclonal antibodies form the backbone of current and emerging strategies, offering targeted pathway inhibition and immune cell modulation, while additional modalities such as MSC-based therapies and T-cell activation inhibitors are expanding the scope of innovation. Collectively, these advancements reflect a continuous evolution in addressing unmet needs and the complex clinical spectrum of GvHD across the 7MM.

  • Small molecules: Small molecules play a central role in the GvHD landscape, offering targeted modulation of key immune signaling pathways. Marketed agents such as JAK inhibitors, ROCK2 inhibitors, and alkylating agents continue to shape current clinical practice, while emerging therapies including RGI-2001 and ivarmacitinib (SHR0302) are advancing next-generation strategies focused on immune tolerance and cytokine regulation.
  • Monoclonal antibodies: Monoclonal antibodies are driving innovation in GvHD by enabling precise targeting of immune cell subsets and inflammatory pathways. Emerging agents such as MEDI-5071 (siplizumab) and itolizumab (EQ001) are being developed to selectively modulate T-cell activity, while approved therapies like axatilimab-csfr (NIKTIMVO) highlight progress in this class. These biologics aim to improve clinical outcomes with targeted immune suppression, reduced off-target effects, and potential for durable disease control.

Graft versus Host Disease (GvHD) Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the GvHD drug's uptake, performance at peak, factors affecting performance during prime years of growth, patient uptake by therapy, and anticipated sales generated by each drug.

Current therapies for GvHD primarily focus on immunosuppression and symptom control, highlighting a significant unmet need for approaches that restore immune tolerance. The future landscape appears promising, with ongoing development of innovative therapies such as MaaT013 (XERVYTEG), which is anticipated to enter the US market in 2027. The therapy is expected to generate notable revenue at launch, with growth projected through 2036, driven by a medium uptake.

Detailed insights of emerging therapies' drug uptake is included in the report...

Market Access and Reimbursement of Approved therapies in Graft versus Host Disease (GvHD)

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.

NOTE: Further Details are provided in the final report....

Graft versus Host Disease (GvHD) Price Scenario & Trends

Pricing and analogue assessment of GvHD therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, closest and most appropriate analogue selection for emerging therapies, and understanding of how pricing influences market access, adherence, and long-term uptake.

  • Pricing of GVHD Approved Drugs

Ruxolitinib (JAKAVI), a targeted JAK1/2 inhibitor used in GvHD, carries a relatively high treatment cost compared to conventional therapies, reflecting its targeted mechanism and clinical value. In Japan, where it is approved for GvHD, the price is approximately JPY 8,185, with no directly comparable standardized pricing established across the US and EU markets due to regional variations in reimbursement and access.

Industry Experts and Physician Views for Graft versus Host Disease (GvHD)

To keep up with GvHD market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the GvHD emerging therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in GvHD, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.

DelveInsight's analysts connected with 8+ KOLs to gather insights at country level. Centers such as the University of Michigan, University Hospital Regensburg, University Hospital Kyoto Prefectural University of Medicine, etc. were contacted.

Their opinion helps understand and validate current and emerging GvHD therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in GvHD.

Qualitative Analysis: SWOT and Attribute Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and attribute analysis.

In the SWOT analysis of GvH, 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. Attribute Analysis analyzes 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.

The team of analysts analyzes promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed. In addition, the scoring is also based on the route of administration, order of entry, 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.

Scope of the Report:

  • The report covers a segment of key events, an executive summary, a descriptive overview of GvHD, explaining their causes, signs and symptoms, pathogenesis, and currently available treatments.
  • Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
  • Additionally, an all-inclusive account of both the current and emerging treatments, along with the elaborative profiles of mid and late stage therapies, will have an impact on the current treatment landscape.
  • A detailed review of the GvHD 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 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 GvHD market.

Report Insights

  • GvHD Patient population forecast
  • GvHD therapeutics market size
  • GvHD pipeline analysis
  • GvHD market size and trends
  • GvHD market opportunity (Current and forecasted)

Report Key Strengths

  • Epidemiology-based (EPI - based) bottom-up forecasting
  • Artificial Intelligence (AI) - enabled market research report
  • 11-year forecast
  • GvHD market outlook (North America, Europe, Asia-Pacific)
  • GvHD Burden trends (by geography)
  • GvHD Treatment addressable Market (TAM)
  • GvHD Competitive Landscape
  • GvHD major companies Insights
  • GvHD Price trends and analogue assessment
  • GvHD Therapies Drug Adoption/Uptake
  • GvHD Therapies Peak Patient Share analysis

Report Assessment

  • GvHD Current treatment practices
  • GvHD Unmet needs
  • GvHD Clinical development Analysis
  • GvHD emerging drugs product profiles
  • GvHD Market attractiveness
  • GvHD Qualitative analysis (SWOT and conjoint analysis)

FAQs:

Market Insights

  • What was the GvHD market size, the market size by therapies, market share (%) distribution in 2025, and what would it look like by 2036? What are the contributing factors for this growth?
  • What are the anticipated pricing variations among different geographies for the emerging therapies in the future?
  • What can be the future treatment paradigm of GvHD?
  • What are the disease risks, burdens, and unmet needs of GvHD? What will be the growth opportunities across the 7MM concerning the patient population with GvHD?
  • Who is the major future competitor in the market, and how will the competitors affect their market share?
  • What are the current options for the treatment of GvHD? What are the current guidelines for treating GvHD in the US, Europe, and Japan?

Reasons to Buy:

  • The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the GvHD market.
  • Bottom up forecasting builds from the affected population to product forecasts, delivering a robust, data driven approach ideal for new therapies and novel classes.
  • 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.
  • Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
  • Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
  • Detailed analysis and ranking of class-wise potential current and emerging therapies under the attribute analysis section to provide visibility around leading classes.
  • To understand KOLs' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
  • Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
  • This Artificial Intelligence (AI) enabled report summarize and simplify complex datasets within the report into clear, actionable insights for stakeholders, investors, and healthcare providers, enabling faster, data driven decisions.

Table of Contents

1. Key Insights

2. Report Introduction

3. GvHD Market Overview at a Glance

  • 3.1. Market Share (%) Distribution of GvHD by Therapies in the 7MM in 2025
  • 3.2. Market Share (%) Distribution of GvHD by Therapies in the 7MM in 2036

4. Executive Summary

5. Key Events

  • 5.1. Upcoming Key Catalysts
  • 5.2. Key Conferences and Meetings
  • 5.3. Key Transactions and Collaborations
  • 5.4. News Flow

6. Disease Overview and Background

  • 6.1. Introduction
  • 6.2. Types of GvHD
    • 6.2.1. Acute GvHD (aGvHD)
    • 6.2.2. Chronic GvHD (cGvHD)
  • 6.3. Risk associated with GvHD
  • 6.4. Pathophysiology of GvHD
  • 6.5. Symptoms of GvHD
  • 6.6. Diagnosis
    • 6.6.1. Differential Diagnosis
    • 6.6.2. Diagnostic Algorithm
    • 6.6.3. Dianostic Guidelines
      • 6.6.3.1. The National Institutes of Health (NIH)/CIBMTR Guidelines (2018)
      • 6.6.3.2. NIH Consensus Report (2020)
      • 6.6.3.3. Chinese Society of Hematology/Chinese Medical Association Guidelines (2024)
  • 6.7. Treatment
    • 6.7.1. Prophylaxis of GvHD
    • 6.7.2. Current Treatment for GvHD
    • 6.7.3. Treatment Algorithm
    • 6.7.4. Treatment Guidelines
      • 6.7.4.1. National Comprehensive Cancer Network (NCCN), (2020)
      • 6.7.4.2. European Respiratory Society (ERS)/The European Society for Blood and Marrow Transplantation (EBMT) Clinical Practice Guidelines (2024)
      • 6.7.4.3. EBMT Consensus Recommendations (2022)

7. Epidemiology and Market Forecast Methodology

8. Epidemiology and Patient Population

  • 8.1. Key Findings
  • 8.2. Assumptions and Rationale
  • 8.3. Total Allogenic Transplant Cases in the 7MM
  • 8.4. Total Cases of GvHD in the 7MM
  • 8.5. The US
    • 8.5.1. Total Cases of GvHD in the US
    • 8.5.2. Type-specific Cases of GvHD in the US
    • 8.5.3. Acute GvHD Cases by Grading in the US
    • 8.5.4. Acute GvHD Cases by Organ Involvement in the US
    • 8.5.5. Chronic GvHD Cases by Grading in the US
    • 8.5.6. Chronic GvHD Cases by Organ Involvement in the US
    • 8.5.7. Total Treated Cases of GvHD in the US
    • 8.5.8. Mortality Adjusted Treated Cases of GvHD in the US
  • 8.6. EU4 and the UK
    • 8.6.1. Total Cases of GvHD in EU4 and the UK
    • 8.6.2. Type-specific Cases of GvHD in EU4 and the UK
    • 8.6.3. Acute GvHD Cases by Grading in EU4 and the UK
    • 8.6.4. Acute GvHD Cases by Organ Involvement in EU4 and the UK
    • 8.6.5. Chronic GvHD Cases by Grading in EU4 and the UK
    • 8.6.6. Chronic GvHD Cases by Organ Involvement in EU4 and the UK
    • 8.6.7. Total Treated Cases of GvHD in EU4 and the UK
    • 8.6.8. Mortality Adjusted Treated Cases of GvHD in EU4 and the UK
  • 8.7. Japan
    • 8.7.1. Total Cases of GvHD in Japan
    • 8.7.2. Type-specific Cases of GvHD in Japan
    • 8.7.3. Acute GvHD Cases by Grading in Japan
    • 8.7.4. Acute GvHD Cases by Organ Involvement in Japan
    • 8.7.5. Chronic GvHD Cases by Grading in Japan
    • 8.7.6. Chronic GvHD Cases by Organ Involvement in Japan
    • 8.7.7. Total Treated Cases of GvHD in Japan
    • 8.7.8. Mortality Adjusted Treated Cases of GvHD in Japan

9. Patient Journey

10. Marketed Drugs

  • 10.1. Key Cross Competition
  • 10.2. Ruxolitinib (JAKAFI/JAKAVI): Incyte/Novartis
    • 10.2.1. Drug Description
    • 10.2.2. Regulatory Milestones
    • 10.2.3. Other Developmental Activities
    • 10.2.4. Summary of Pivotal Trial
    • 10.2.5. Clinical Development
      • 10.2.5.1. Clinical Trials Information
    • 10.2.6. Analyst Views
  • 10.3. Ibrutinib (IMBRUVICA): AbbVie (Pharmacyclics)/Janssen
    • 10.3.1. Drug Description
    • 10.3.2. Regulatory Milestones
    • 10.3.3. Other Developmental Activities
    • 10.3.4. Summary of Pivotal Trial
    • 10.3.5. Clinical Development
      • 10.3.5.1. Clinical Trials Information
    • 10.3.6. Analyst Views
  • 10.4. Remestemcel-L (RYONCIL/TEMCELL HS): Mesoblast/JCR Pharmaceuticals
    • 10.4.1. Drug Description
    • 10.4.2. Regulatory Milestones
    • 10.4.3. Other Developmental Activities
    • 10.4.4. Summary of Pivotal Trial
    • 10.4.5. Analyst Views

11. Emerging Drugs

  • 11.1. Key Cross
  • 11.2. MaaT013 (XERVYTEG): MaaT Pharma
    • 11.2.1. Drug Description
    • 11.2.2. Other Development Activities
    • 11.2.3. Clinical Development
      • 11.2.3.1. Clinical Trial Information
    • 11.2.4. Safety and Efficacy
    • 11.2.5. Analyst Views
  • 11.3. RGI-2001: REGiMMUNE
    • 11.3.1. Drug Description
    • 11.3.2. Other Development Activities
    • 11.3.3. Clinical Development
      • 1.1.1.1. Clinical Trial Information
    • 11.3.4. Safety and Efficacy
    • 11.3.5. Analyst Views
  • 11.4. CYP-001: Cynata Therapeutics
    • 11.4.1. Drug Description
    • 11.4.2. Other Developmental Activities
    • 11.4.3. Clinical Development
      • 11.4.3.1. Clinical Trial Information
    • 11.4.4. Safety and Efficacy
    • 11.4.5. Analyst Views

12. GvHD - 7MM Market Analysis

  • 12.1. Key Findings
  • 12.2. Key Market Forecast Assumptions
    • 12.2.1. Cost Assumptions
  • 12.3. Market Outlook
  • 12.4. Attribute Analysis
  • 12.5. Total Market Size of GvHD in the 7MM
  • 12.6. Market Size of GvHD by Therapies in the 7MM
  • 12.7. Market Size of GvHD in the US
    • 12.7.1. Total Market Size of GvHD in the US
    • 12.7.2. Market Size of GvHD by Therapies in the US
  • 12.8. EU4 and the UK Market Size
    • 12.8.1. Total Market Size of GvHD in EU4 and the UK
    • 12.8.2. Market Size of GvHD by Therapies in EU4 and the UK
  • 12.9. Japan Market Size
    • 12.9.1. Total Market size of GvHD in Japan
    • 12.9.2. Market Size of GvHD by Therapies in Japan

13. Key Opinion Leaders' Views

14. Unmet Needs

15. SWOT Analysis

16. Market access and reimbursement

  • 16.1. The United States
    • 16.1.1. Centre for Medicare & Medicaid Services (CMS)
  • 16.2. EU4 and the UK
    • 16.2.1. Germany
    • 16.2.2. France
    • 16.2.3. Italy
    • 16.2.4. Spain
    • 16.2.5. The United Kingdom
  • 16.3. Japan
    • 16.3.1. MHLW
  • 16.4. Market Access and Reimbursement of GvHD Therapies

17. Appendix

  • 17.1. Acronyms and Abbreviations
  • 17.2. Bibliography
  • 17.3. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

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