Steroid Refractory Acute Graft-Versus-Host Disease (SR-aGvHD) - Market Insight, Epidemiology, and Market Forecast - 2036

Steroid-refractory acute Graft-versus-host Disease (SR-aGVHD) Market Summary

• The SR-aGVHD Market Size in the 7MM is expected to grow from significantly from 2026 to 2036
• The United States contribute to the largest market share amount 7MM (US, Japan, EU4 and UK). Also, United States accounted for the highest number of cases of GvHD in 2025. The rise in cases of GvHD in the US is driven by increased allogeneic stem cell transplants and improved post-transplant survival rates.
• The 7MM accounted for ~54,000 total allogenic transplant cases and ~52,000 total cases of GvHD in 2025, these cases are expected to increase at a significant CAGR throughout the study period (2022-2036).
• Steroid-refractory acute Graft-versus-host Disease (SR-aGVHD) remains a major barrier to post-transplant survival, representing one of the most severe complications following allogeneic Hematopoietic Cell Transplantation (HCT), with limited durable treatment options and poor prognosis in steroid-resistant cases.
• The global rise in allogeneic HCT procedures, particularly among aging populations and patients with high-risk hematologic malignancies, is expanding the SR-aGVHD patient pool and reinforcing the urgency for more effective, targeted interventions.
• In 2025, the US had the highest number of cases of GvHD with ~22,500 among the 7MM, followed by EU4 and the UK in the same year. These cases are expected to rise over the course of the study period from 2022 to 2036.
• Ruxolitinib, approved by the FDA in 2019, remains the cornerstone of SR-aGVHD treatment for both adult and pediatric populations, validating the JAK-STAT signaling pathway as a critical therapeutic target.
• The recent approval of RYONCIL (remestemcel-L) marks a significant advancement, particularly for pediatric SR-aGVHD, as the first approved mesenchymal stem cell therapy designed to modulate inflammation and support tissue regeneration.
• The pipeline for SR-aGVHD is expanding with novel immune-regulatory agents such as neihulizumab (ALTB-168), RLS-0071, and others, which aim to offer improved efficacy by targeting key inflammatory pathways while preserving graft-versus-leukemia effects.
• Overall, the SR-aGVHD treatment landscape is undergoing meaningful innovation, driven by advances in immunology, supportive regulatory frameworks, and the pursuit of precision and cell-based therapies to enhance long-term transplant success.

DelveInsight's comprehensive report titled "Steroid Refractory Acute Graft-Versus-Host Disease Market Insights, Epidemiology, and Market Forecast - 2036" offers a detailed analysis of SR-aGvHD. The report presents historical and projected epidemiological data covering Total Allogenic Transplant Cases, Total Acute GvHD Cases, Acute GvHD cases by grading, Acute GvHD cases by organ involvement, Total Cases of SR-aGvHD, and Total Treated Cases of SR-aGvHD. In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2022 to 2036.

The report analyzes the existing treatment practices and unmet medical requirements in SR-aGvHD. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Key Market Drivers

According to DelveInsight, the Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGvHD) market growth is driven by:

• Rising Number of HSCT Procedures: The increasing adoption of hematopoietic stem cell transplantation particularly among aging populations and patients with high-risk hematologic malignancies has led to a higher incidence of SR-aGvHD, expanding the treatable patient pool and reinforcing the urgency for more effective, targeted interventions.
• Improved Disease Recognition and Diagnosis: Enhanced clinical awareness, improved diagnostic criteria, and better monitoring practices are contributing to increased identification and treatment rates.
• Advancements in Immunology Research: Growing understanding of immune dysregulation and GVHD pathophysiology is supporting the development of more precise and effective therapies.
• Emergence of Targeted Therapies: Introduction and development of novel targeted agents, including JAK inhibitors and other immunomodulators, are improving treatment outcomes.
• Growing Development of Cell-Based Therapies: Increasing research into cellular therapies such as mesenchymal stem cells is providing promising new treatment options.
• Robust Emerging Pipeline: Strong pipeline activity from pharmaceutical and biotech companies is expected to introduce innovative therapies during the forecast period. Companies like CSL Behring, MaaT Pharma, ReAlta Life Sciences are expected to launch their drugs during the forecast period boosting the market of SR-aGvHD.
• Increasing Healthcare Investment: Rising healthcare expenditure and improved access to advanced treatments across major markets are supporting market expansion.
• Regulatory Support for Orphan Diseases: Favorable regulatory incentives, including orphan drug designations and expedited approvals, are encouraging innovation and commercialization.

Steroid Refractory Acute Graft-Versus-Host Disease Overview

Acute graft-versus-host disease (aGVHD) is a severe immune-mediated complication of allogeneic hematopoietic stem cell transplantation (alloHCT) in which donor immune cells continue to attack recipient tissues despite standard first-line corticosteroid therapy and is a major cause of morbidity and mortality. Systemic steroid therapy is the first-line treatment for aGVHD, although about half of patients will become refractory to treatment. As the number of patients undergoing alloHCT increases, developing safe and effective treatments for aGVHD will become increasingly important, especially for those whose disease becomes refractory to systemic steroid therapy

Steroid Refractory Acute Graft-Versus-Host Disease Diagnosis and Treatment Algorithm

aGVHD is diagnosed clinically after laboratory analysis, imaging, and/or endoscopic examination to exclude potential differential diagnoses. A biopsy may help confirm the diagnosis, but it lacks sensitivity and specificity. Following diagnosis, aGVHD severity is graded from mild (grade I) to very severe (grade IV). The recommended first-line treatment for aGVHD is systemic steroid therapy; however, many patients become refractory to steroid therapy. SR-aGVHD can be defined as a clear progression after 3-5 days of treatment or no response after 5-7 days of treatment. SR-aGVHD is associated with a high mortality risk.

Ruxolitinib, an oral Janus kinase (JAK) 1/2 inhibitor, has become a cornerstone treatment for SR-AGVHD. It modulates immune cell development, proliferation, and activation to inhibit cytokine signaling and promote T regulatory cells. The FDA approved ruxolitinib for SR-AGVHD in 2019 based on results of the REACH-1 trial, followed by the EMA in 2022, significantly altering the treatment paradigm and establishing its role as the standard second-line treatment. Mesenchymal stem cells (MSCs) are stem cells with a capacity for pluripotent differentiation. They can be obtained from bone marrow, adipose tissue, or umbilical cord and infused intravenously, usually without adverse events. Supportive care remains essential throughout treatment, including antimicrobial prophylaxis, nutritional support, skin care, and management of fluid and electrolyte imbalances. Regular reassessment at Days 7, 14, and 28 is recommended to monitor therapeutic response and guide escalation.

Steroid Refractory Acute Graft-Versus-Host Disease Epidemiology

The epidemiology section of the SR-aGVHD market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the epidem of SR-aGVHD. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

• Among the 7MM, the United States accounted for the highest number of cases of GvHD in 2025. The rise in cases of GvHD in the US is driven by increased allogeneic stem cell transplants and improved post-transplant survival rates.
• In 2025, the US had the highest number of cases of GvHD with ~22,500 among the 7MM, followed by EU4 and the UK in the same year. These cases are expected to rise over the course of the study period from 2022 to 2036.

According to DelveInsight's analysis, in 2025, Japan accounted for ~500 cases of acute GvHD.

• Among Acute GvHD cases by grading, Grade B[II] aGvHD accounts for the highest number of cases.

Steroid Refractory Acute Graft-Versus-Host Disease Market Outlook

The SR-aGVHD therapeutics market is further expected to increase by the major drivers, such as the rising population, technological advancements, and upcoming therapies in the forecast period (2026-2036).

• With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the SR-aGVHD in the 7MM is expected to change significantly during the forecast period 2026-2036.

Steroid Refractory Acute Graft-Versus-Host Disease Market Outlook

The SR-aGVHD therapeutics market is further expected to increase by the major drivers, such as the rising population, technological advancements, and upcoming therapies in the forecast period (2025-2034).

With ongoing research and continued dedication, the future holds hope for even more effective treatments and, ultimately, a cure for this challenging condition. According to DelveInsight, the SR-aGVHD in the 7MM is expected to change significantly during the forecast period 2025-2034.

Steroid Refractory Acute Graft-Versus-Host Disease Drug Chapters

Marketed Steroid Refractory Acute Graft-Versus-Host Disease Drugs

JAKAFI (ruxolitinib): Incyte

JAKAFI is a first-in-class JAK1/JAK2 inhibitor approved by the US FDA for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older. In May 2024, Incyte Corporation announced that the US Food and Drug Administration approved JAKAFI for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older. The approval was based on data from REACH1, an open-label, single-arm, multicenter study of JAKAFI in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. It was the first therapy to be approved for this indication.

RYONCIL (remestemcel-L-rknd): Mesoblast

RYONCIL is the first and only FDA-approved, allogeneic, off-the-shelf, bone marrow-derived mesenchymal stromal cell therapy indicated for the treatment of pediatric patients aged 2 months and older, including adolescents and teenagers, with SR-aGvHD. It got FDA approval for SR-aGvHD in December 2024.

Emerging Steroid Refractory Acute Graft-Versus-Host Disease Drugs

The SR-aGVHD market is expected to evolve gradually, driven by the limited number of emerging therapies currently in development. Key players such as RLS-0071, ALTB-168, CSL964 (Alpha 1 Antitrypsin), Pegtarazimod, MaaT013, and others, showing commitment to addressing this unmet need, with ongoing efforts to advance novel treatment options for this complex condition.

ZEMAIRA (CSL964 Alpha-1 Antitrypsin): CSL Behring

CSL964 Alpha-1 Antitrypsin is an Alpha1-Proteinase Inhibitor (A1 -PI) being developed by CSL Behring to treat steroid-refractory in aGvHD. As per the company's pipeline: The drug is currently in Phase III trials for treating steroid-refractory aGvHD.

MaaT013: MaaT Pharma

MaaT013, also known as XERVYTEG, is an off-the-shelf microbiome ecosystem therapy for SR-aGvHD, designed to restore gut microbiota balance and modulate immune responses, particularly in gastrointestinal-predominant disease. MaaT013 has received Orphan Drug Designation (ODD) from the US FDA and EMA. The company submitted its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) on June 2, 2025, with marketing authorization expected in the second half of 2026.

RLS-0071 (pegtarazimod): ReAlta Life Sciences

RLS 0071 is an investigational peptide therapy developed by ReAlta Life Sciences that leverages a novel dual mechanism of action to rebalance complement activation and neutrophil driven inflammation specifically through inhibiting myeloperoxidase, neutrophil extracellular traps (NETs), and complement components. The US FDA has granted it both Orphan Drug and Fast Track designations for SR aGVHD. The Company is currently conducting a Phase II, open label clinical trial of RLS-0071 in hospitalized patients with SR-aGvHD. In December 2025, ReAlta Therapeutics presented encouraging Phase II clinical data at the ASH 2025 Annual Meeting, highlighting rapid clinical responses and clear target engagement in patients with SR-aGvHD.

ALTB-168 (Neihulizumab): AltruBio

Neihulizumab is an immune checkpoint regulator that targets PSGL-1. It is designed to trigger the depletion of chronically activated T cells. The US FDA has granted it both Orphan Drug and Fast Track designations for SR aGVHD. Currently, it has completed Phase I trial, and ReAlta is enrolling a Phase II, open-label clinical trial of RLS-0071 in patients with SR-aGVHD

Steroid Refractory Acute Graft-Versus-Host Disease Market Segmentation

DelveInsight's 'Steroid Refractory Acute Graft-Versus-Host Disease - Market Insights, Epidemiology, and Market Forecast - 2036 report provides a detailed outlook of the current and future SR aGVHD market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Steroid Refractory Acute Graft-Versus-Host Disease Market Size by Countries

The SR aGVHD market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2024, the United States held a significant share of the overall 7MM (Seven Major Markets) SR aGVHD market. This dominance is projected to persist, especially with the potential early introduction of new products.

Steroid Refractory Acute Graft-Versus-Host Disease Market Size by Therapies

SR aGVHD Market Size by Therapies is categorized into current and emerging markets for the study period 2022-2036.

Steroid Refractory Acute Graft-Versus-Host Disease Drugs Uptake

This section focuses on the sales uptake of potential SR aGVHD drugs that have recently been launched or are anticipated to be launched in the SR aGVHD market between 2022 and 2036. It estimates the market penetration of SR aGVHD drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the SR aGVHD.

The emerging SR aGVHD therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the SR aGVHD market.

Steroid Refractory Acute Graft-Versus-Host Disease Market Access and Reimbursement

DelveInsight's 'Steroid Refractory Acute Graft-Versus-Host Disease - Market Insights, Epidemiology, and Market Forecast - 2036' report provides a descriptive overview of the market access and reimbursement scenario of SR aGVHD.

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

Latest Updates

• During ASH2025 ReAlta Life Sciences announced Phase 2 data showing that RLS-0071 (pegtarazimod) demonstrated rapid clinical responses, target engagement, and favorable safety in steroid-refractory acute GvHD. Notably, Pegtarazimod received EMA orphan drug designation for GVHD, supported by phase 2 AURORA trial data, highlighting its potential in addressing unmet needs in acute GVHD treatment.
• In Nov, 2025 Mesoblast, announced that in collaboration with the NIH-funded BMT CTN, it is initiating a pivotal Phase III trial of its MSC therapy Ryoncil (remestemcel-L) in adults with severe steroid-refractory acute GvHD to expand its use beyond pediatric patients and address a high-mortality population with limited treatment options.
• In June 2025: MaaT Pharma submitted the Marketing Authorization Application (MAA) to the European Medicines Agency for MaaT013 for steroid-refractory acute GvHD. In H2 EMA marketing authorization decision anticipated, which could enable the first commercialization of MaaT013 in Europe.

KOL Views

To keep up with current SR aGVHD market trends and fill gaps in secondary findings, we interview KOLs and SMEs' working in the SR aGVHD domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or SR aGVHD market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the SR-aGvHD unmet needs.

"SR-aGvHD in children presents additional complexity due to limited tolerance for prolonged immunosuppression. It emphasizes the importance of treatments that achieve rapid disease control without adding cumulative toxicity or impairing immune reconstitution." - University of Michigan, US

"Acute GvHD, once limited to occurring before Day 100, now includes late-onset acute GvHD and an overlap syndrome, especially after RIC. It mainly affects the skin, gastrointestinal tract, and liver, with emerging evidence suggesting lung involvement in experimental GvHD." - University of Tokyo Hospital, Japan

Steroid Refractory Acute Graft-Versus-Host Disease: KOL Insights

DelveInsight's analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as Mercer University, US, University Hospital Tubingen, Germany, University of Barcelona, Barcelona, Spain, ellvitge-Idibell University Hospital, Barcelona, Spain, University of Leeds, Leeds, UK, and University of Occupational and Environmental Health, Japan, among others.

"The norm is basically that you start them on steroids, and if they're progressing within 3 to 4 days, you'd call them refractory. If they don't get better and don't get worse, they're not responding well. Those who progress need to move quickly to something else because they're not going to do well."

"With ruxolitinib, the median time to response is pretty good, so you're hopefully going to see a response within 7 to 14 days. It's not always that early, but the median time is right around there. No matter what, I'm already trying to think about what I am going to be using next to help me try to get this patient's graft-versus-host disease under control."

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the SR aGVHD Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Steroid Refractory Acute Graft-Versus-Host Disease Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for SR aGVHD. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging SR aGVHD therapies.

Steroid Refractory Acute Graft-Versus-Host Disease Report Insights

• SR aGVHD Patient Population
• Therapeutic Approaches
• SR aGVHD Pipeline Analysis
• SR aGVHD Market Size and Trends
• SR aGVHD Market Opportunities
• Impact of Upcoming Therapies

Steroid Refractory Acute Graft-Versus-Host Disease Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• SR aGVHD Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed SR aGVHD Market
• SR aGVHD Drugs Uptake

Steroid Refractory Acute Graft-Versus-Host Disease Report Assessment

• SR aGVHD Current Treatment Practices
• Unmet Needs
• SR aGVHD Pipeline Product Profiles
• SR aGVHD Market Attractiveness

Key Questions:

• What are the key findings of steroid refractory acute graft-versus-host disease epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2022-2036)?
• What are the currently available treatments for steroid refractory acute graft-versus-host disease?

What are the disease risk, burden, and unmet needs of steroid refractory acute graft-versus-host disease?

• At what CAGR is the steroid refractory acute graft-versus-host disease market and its epidemiology is expected to grow in the 7MM during the forecast period (2026-2036)?
• How would the unmet needs impact the steroid refractory acute graft-versus-host disease market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of steroid refractory acute graft-versus-host disease in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2026-2036)?
• How many companies are currently developing therapies for the treatment of steroid refractory acute graft-versus-host disease?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the steroid refractory acute graft-versus-host disease Market.
• Insights on patient burden, 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 current treatment 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 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 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 the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.