Graft Versus Host Disease - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights:

According to DelveInsight's estimates, in 2023, there were approximately 52 thousand cases of GvHD in the 7MM. Of these, the United States accounted for 45% of the cases, while EU4 and the UK accounted for nearly 39% and Japan represented 16% of the cases, respectively.

The GvHD market is projected to see consistent growth, with a robust compound annual growth rate (CAGR) anticipated from 2025 to 2034. This expansion across the 7MM will be driven by the introduction of innovative therapies, including ZEMAIRA, EQ001, MaaT013, and RGI-2001, among others.

According to DelveInsight's analysis, the GvHD market in the 7MM was valued at approximately USD 1,301.8 million in 2023. Over the forecast period from 2025 to 2034, this market is projected to grow at a CAGR of 8.2%.

Currently, therapies comprising steroids, NIKTIMVO, JAKAVI/JAKAFI, IMBRUVICA, RYONCIL/TEMCELL HS, and ORENCIA, among others are available in the market for managing GvHD.

CSL Behring, Equillium, MaaT Pharma, and REGiMMUNE, among others are progressing their assets through various clinical trial phases, driving innovation in the GvHD market and creating significant growth opportunities.

In November 2024, Equillium accelerated the completion timeline for EQ001 of the Phase III EQUATOR study in aGvHD to Q1 2025.

DelveInsight's "GvHD - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of GvHD, historical and forecasted epidemiology, as well as the GvHD market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The GvHD market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM GvHD market size from 2020 to 2034. The report also covers GvHD treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Graft versus Host Disease Understanding and Treatment Algorithm

GvHD overview

GvHD is an immune condition that occurs after transplant procedures when immune cells (T cells) from the donor (known as the graft or graft cells) attack the recipient patient host's tissues (healthy cells); the disease is a side effect that is common after an allogeneic bone marrow transplant. In some cases, it can be even life-threatening. Before undergoing an allogeneic stem cell transplant, the patient will receive high doses of chemotherapy or radiation to destroy the diseased cells and prepare the body for the donor cells.

The two primary types of GvHD are acute and chronic. An allogeneic transplant recipient may experience either form, either forms, or none at all. On average, aGvHD occurs in about 30-50% of patients who undergo allogeneic hematopoietic cell transplantation GvHD diagnosis

Diagnosis of GvHD relies on clinical criteria, biopsies of affected organs (skin, liver, gastrointestinal tract), and exclusion of mimics like infection or drug reactions. While lab/imaging studies aid evaluation, a key challenge remains the lack of predictive biomarkers to preemptively identify at-risk patients or distinguish GvHD from overlapping conditions. For cGvHD, diagnosis requires =1 distinctive manifestation (e.g., lichenoid lesions, scleroderma) after ruling out alternatives. Emerging blood-based biomarker panels may refine risk stratification and guide personalized immunosuppressive therapy, addressing current diagnostic limitations.

GvHD treatment

GvHD is a major complication of allogeneic stem cell transplantation, with stem cells sourced from bone marrow, peripheral blood, or umbilical cord blood. Corticosteroids like prednisone and methylprednisolone are the primary first-line treatments, often combined with other immunosuppressants. Mild aGvHD is managed with topical steroids, while systemic cases require stronger immunosuppressive therapy. cGvHD is treated based on severity, with localized symptoms managed through topical treatments and more severe cases requiring systemic corticosteroids like prednisone.

Graft versus Host Disease Epidemiology

As the market is derived using a patient-based model, the GvHD epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total allogenic transplant cases, total cases of GvHD, type-specific cases of GvHD, aGvHD cases by grading, aGvHD cases by organ involvement, cGvHD cases by grading, cGvHD cases by organ involvement, total treated cases of GvHD, and mortality adjusted treated cases of GvHD in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

DelveInsight's epidemiology model estimates that in 2023, there were approximately 57 thousand allogenic transplant cases and nearly 52 thousand cases of GvHD across the 7MM which are expected to increase by 2034.

In 2023, the US reported the highest number of GvHD cases among the 7MM, with approximately 24 thousand, a figure expected to rise by 2034.

In 2023, Germany reported the highest number of GvHD cases among EU4 and the UK, with approximately 8 thousand cases, followed by Italy and France, with around 3 thousand cases each. Spain had the lowest number, with approximately 27 hundred cases.

In 2023, Japan reported approximately 8 thousand GvHD cases, a number projected to increase by 2034.

In 2023, the US reported around 5 thousand cases of aGvHD and approximately 18 thousand prevalent cases of cGvHD over five years.

In 2023, EU4 and the UK reported the following distribution of aGvHD cases by grading: around 2 thousand cases of Grade B[II] aGvHD, approximately 1 thousand cases of Grade C[III] aGvHD, and approximately 8 hundred cases of Grade D[IV] aGvHD.

In 2023, Germany reported approximately 9 hundred aGvHD cases involving the skin, approximately 4 hundred cases involving the liver, and approximately 8 hundred cases involving the gastrointestinal tract.

The UK reported approximately 6 hundred mild, approximately 1 thousand moderate, and approximately 8 hundred severe cases of cGvHD by grading in 2023.

In 2023, Japan reported the following cGvHD cases: around 1 thousand cases involving the skin, approximately 3 thousand cases affecting the oral mucosa, approximately 2 thousand cases impacting the eyes, around 3 hundred cases involving the liver,approximately 8 hundred cases in the gastrointestinal tract, around 2 thousand cases affecting the lungs, approximately 3 hundred cases involving the genitals, and approximately 4 hundred cases related to the joints and fascia.

Graft versus Host Disease Recent Developments

In December 2024, the FDA approved Mesoblast's cell therapy, Ryoncil, for treating graft-versus-host disease (GVHD) following stem cell or bone marrow transplants. Ryoncil is the first mesenchymal stromal cell therapy approved for pediatric patients aged two months and older whose GVHD symptoms have not responded to standard steroid therapy.

In August 14, 2024, AstraZeneca's IMFINZI (durvalumab) has received FDA approval for the treatment of adult patients with resectable early-stage (IIA-IIIB) non-small cell lung cancer (NSCLC) who do not have known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. The regimen includes IMFINZI in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery.

Graft versus Host Disease Drug Chapters

The drug chapter segment of the GvHD report encloses a detailed analysis of GvHD-marketed drugs and mid to late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the GvHD 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.

Marketed Drugs

JAKAFI/JAKAVI (ruxolitinib): Incyte

JAKAFI/JAKAVI (ruxolitinib) is a potent dual JAK1 and JAK2 inhibitor that exhibits low single-digit nanomolar biochemical IC50s for both kinases. The selectivity within the JAK family members is represented by a six-fold selectivity over TYK2 and approximately 130-fold selectivity over JAK3. Ruxolitinib demonstrates dose-dependent inhibition of JAK2/STAT signaling and inhibition of cell growth that is dependent on JAK2 activation.

IMBRUVICA (ibrutinib): Pharmacyclics (acquired by AbbVie)/ Janssen

IMBRUVICA (ibrutinib) is a first-in-class, oral, once-daily therapy that inhibits a protein called Bruton's tyrosine kinase (BTK). BTK is a key signaling molecule in the B-cell receptor signaling complex that plays an important role in the survival and spread of malignant B cells and other serious, debilitating conditions. IMBRUVICA blocks signals that tell malignant B cells to multiply and spread uncontrollably.

Emerging Drugs

ZEMAIRA (CSL 964, alpha-1 antitrypsin): CSL Behring

CSL 964 is an Alpha1-proteinase Inhibitor (A1-PI) being developed by CSL Behring to treat steroid-refractory aGvHD and prevent aGvHD in high-risk patients receiving an allogeneic HSCT. ZEMAIRA is approved to treat chronic augmentation and maintenance therapy in adults with alpha1-proteinase inhibitors. ZEMAIRA acts as augmentation therapy for increasing and maintaining serum levels and the levels of lung Epithelial Lining Fluid (ELF) of A1-PI.

The drug is currently in Phase III trials for treating steroid-refractory aGvHD and in Phase II/III trials for preventing aGvHD

EQ001 (itolizumab; Bmab600): Equillium/Biocon

EQ001 (Itolizumab; Bmab600) is a first-in-class immune-modulating antibody designed to inhibit CD6 to reduce the activation and trafficking of pathogenic T cells that release pro-inflammatory cytokines of autoimmune and inflammatory diseases like aGvHD, uncontrolled moderate-to-severe asthma, and lupus nephritis. Itolizumab targets the CD6-ALCAM signaling pathway to selectively downregulate pathogenic effector T cells (Teffs) while preserving Tregs critical for maintaining a balanced immune response.

Currently, EQ001 is being investigated in Phase III clinical study in combination with corticosteroids for the first-line treatment of aGvHD, which is in recruiting status. EQ001 topline data could see a potential acceleration to Q1 2025.

Drug Class Insights

GvHD treatment is evolving from broad immunosuppression to targeted and cell-based therapies. JAK inhibitors (e.g., ruxolitinib) are the backbone for steroid-refractory cases, while ROCK inhibitors (e.g., belumosudil) and anti-CSF-1R antibodies (e.g., axatilimab-csfr) address fibrosis in chronic GvHD. MSC therapies (e.g., RYONCIL) and microbiome-based approaches (e.g., MaaT013) offer novel strategies for severe cases. IL-2Ra antagonists (e.g., basiliximab) and CD6 inhibitors (e.g., itolizumab) provide additional immunomodulation, though market adoption varies. Future treatments will likely focus on combination strategies targeting both inflammation and tissue repair.

Market Outlook

GvHD is a major complication of allogeneic stem cell transplants. Corticosteroids (e.g., prednisone) are the first-line treatment for aGvHD, often combined with immunosuppressants like cyclosporine. Mild aGvHD may require topical steroids, while severe cases need systemic corticosteroids. For cGvHD, prednisone is preferred for widespread symptoms, with localized treatments (e.g., steroid creams, eye drops) for mild, organ-specific cases.

In August 2024, the US FDA approved NIKTIMVO (axatilimab-csfr) for treating cGVHD in adult and pediatric patients weighing at least 40 kg who have failed at least two prior lines of systemic therapy. Axatilimab-csfr is a monoclonal antibody targeting Colony-stimulating Factor-1 Receptors (CSF-1R) on monocytes and macrophages. By blocking CSF-1R, it reduces circulating proinflammatory and profibrotic monocytes and monocyte-derived macrophages, as evidenced by decreased nonclassical monocyte counts in nonclinical studies, while also inhibiting pathogenic macrophage activity in tissues. Syndax acquired exclusive global rights to develop and commercialize axatilimab from UCB in 2016. In September 2021, Syndax and Incyte entered into a global co-development and co-commercialization agreement for axatilimab in cGvHD and potential future indications.

In 2017, the US FDA gave a green signal to IMBRVICA [Ibrutinib, AbbVie (Pharmacyclics)] for the treatment of adult patients suffering from cGvHD after the failure of one or more lines of systemic therapy. It was the first ever approved treatment targeting this specific patient pool. In September 2021, the PMDA approved IMBRUVICA to treat cGvHD after hematopoietic stem cell transplantation.

Companies like CSL's ZEMAIRA (CSL964, Alpha-1 antitrypsin), Equillium Bio's EQ001 (Itolizumab), MaaT Pharma's MaaT013, Medac's MC0518, among others are involved in the development of drugs for the treatment of GvHD.

Apart from this, several drugs currently in the early stages of development include RLS-0071 by ReAlta Life Sciences, Vimseltinib by Deciphera Pharmaceuticals, ASC-930 by ASC Therapeutics, RGI-2001 by REGiMMUNE, CYP-001 by Cynata Therapeutics, arsenic trioxide (As2O3) by BioSenic (Medsenic), TRX103 (Tregs) by Tr1X, TCD601 (Siplizumab) by ITB-MED, F-652 by Evive Biotech, RHPRG4 by Lubris BioPharma, XBI302 by Xbiome, RG6287 by Genentech, ALTB-168 by AltruBio, and SER-155 by Seres Therapeutics.

The total market size of GvHD in the 7MM was approximately USD 1,302 million in 2023 and is projected to increase during the forecast period (2025-2034).

The market size for GvHD in the US was approximately USD 920 million in 2023 and is anticipated to increase due to the launch of emerging therapies.

The total market size of EU4 and the UK was calculated to be approximately USD 252 million in 2023, which was nearly 19% of the total market revenue for the 7MM.

In 2023, Germany dominated the market among EU4 and the UK, generating around USD 98 million. France followed closely with approximately USD 41 million, while the UK recorded around USD 40 million.

In 2023, the total market size of GvHD was approximately USD 130 million in Japan which is anticipated to increase during the forecast period (2025-2034).

Estimates suggest that REZUROCK is expected to generate approximately USD 173 million by 2034 in the 7MM.

Graft versus Host Disease Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034.

Graft versus Host Disease Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. 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 emerging therapies for GvHD.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on GvHD evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

DelveInsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers like the University of Michigan, US, University of Minnesota, US, New York University, US, University Hospital Regensburg, Germany, Universite de Bordeaux, France, Hospital Saint-Antoine - Ap-Hp Sorbonne University, France, Universita Cattolica del Sacro Cuore, Italy, Instituto de Investigaciones Biomedicas August Pi i Sunyer (IDIBAPS), Spain, University of Glasgow, the UK, University of Tokyo Hospital, Japan, and University Hospital Kyoto Prefectural University of Medicine, Japan, among others, were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or GvHD market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician's View

As per the KOLs from the US, genetic variations in cytokine-related genes, including TNF-a, IL-10, and IFN-?, contribute to the risk of GvHD by influencing the intensity of the immune response, particularly the cytokine storm. While the associations between these polymorphisms and GVHD remain variable and inconclusive, future advancements in donor selection are expected to incorporate both HLA and non-HLA genetic markers to refine transplant compatibility and improve patient outcomes.

As per the KOLs from France, acute GvHD is a significant immune complication following alloHCT, leading to substantial morbidity and mortality. It results from donor immune cells attacking recipient tissues, particularly the skin, gastrointestinal tract, and liver. Diagnosis relies on clinical evaluation, with preventive therapies given universally but not always effective. Steroids remain first-line treatment, followed by the JAK2 inhibitor ruxolitinib.

As per the KOLs from Japan, for patients with acute GvHD, early referral to a transplant center is crucial to access effective treatments and clinical trials, especially as the condition can become steroid-refractory. cGvHD typically begins with steroids, but referral to a specialized center ensures multidisciplinary care, addressing complications like steroid side effects, infections, and muscle weakness.

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 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.

To analyze the effectiveness of these therapies, have calculated their attributed analysis by giving them scores based on their ability to improve atrial and ventricular dimension/function and ability to regulate heart rate.

Further, the therapies' safety is evaluated wherein the adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials, which directly affects the safety of the molecule in the upcoming trials. 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

JAKAFI

IncyteCARES for JAKAFI Savings Program

The IncyteCARES for JAKAFI Savings Program offers eligible patients with commercial prescription insurance the opportunity to access JAKAFI for as little as USD 0 per month. To qualify, individuals must have commercial prescription drug coverage, as those insured under federal or state government programs such as Medicare Part D, Medicare Advantage, Medicaid, or TRICARE are not eligible. Patients without prescription drug coverage also do not qualify. Additionally, eligibility requires residency in the US or Puerto Rico and a valid prescription for JAKAFI for an FDA-approved indication.

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenarios, 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 Report:

The report covers a segment of key events, an executive summary, and a descriptive overview of GvHD, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.

Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines have been provided.

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 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 shape and drive the 7MM GvHD market.

Graft versus Host Disease report insights

Patient Population

Therapeutic Approaches

GvHD Pipeline Analysis

GvHD Market Size and Trends

Existing and Future Market Opportunity

Graft versus Host Disease report key strengths

10 years Forecast

The 7MM Coverage

GvHD Epidemiology Segmentation

Key Cross Competition

Attribute analysis

Drugs Uptake and Key Market Forecast Assumptions

Graft versus Host Disease report assessment

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Market Attractiveness

Qualitative Analysis (SWOT and Attribute Analysis)

Key Questions:

Market Insights

What was the total market size of GvHD, the market size of GvHD by therapies, and market share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?

How will CSL964 affect the treatment paradigm of GvHD?

How will JAKAFI compete with other upcoming products and marketed therapies?

Which drug is going to be the largest contributor by 2034?

What are the pricing variations among different geographies for approved and marketed therapies?

How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

What are the disease risks, burdens, and unmet needs of GvHD? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to GvHD?

What is the historical and forecasted GvHD patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?

Out of the countries mentioned above, which country would have the highest diagnosed prevalent GvHD population during the forecast period (2025-2034)?

What factors are contributing to the growth of GvHD cases?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

What are the current options for the treatment of GvHD? What are the current clinical and treatment guidelines for treating GvHD?

How many companies are developing therapies for the treatment of GvHD?

How many emerging therapies are in the mid-stage and late stage of development for treating GvHD?

What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?

What is the cost burden of current treatment on the patient?

Patient acceptability in terms of preferred treatment options as per real-world scenarios?

What are the accessibility issues of approved therapy in the US?

What is the 7MM historical and forecasted market of GvHD?

Reasons to Buy:

The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the GvHD market.

Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.

Understand the existing market opportunities in varying geographies and the growth potential over the coming years.

The distribution of historical and current patient share is based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Identifying upcoming solid 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 conjoint analysis section to provide visibility around leading classes.

Highlights of Access and Reimbursement policies for GvHD, barriers to accessibility of approved therapy, and patient assistance programs.

To understand Key Opinion Leaders' perspectives around 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.