Graft versus host disease (GVHD) is a potentially life-threatening complication of allogeneic hematopoietic stem-cell transplantation (HSCT). GVHD is a systemic inflammatory reaction triggered by donor T cells’ recognition of host cell antigens in nontarget tissue as foreign. For decades, steroids have been the first-line therapy for the prevention and treatment of GVHD, but interviewed experts call for better treatments for steroid-refractory patients, as well as better first-line and prophylactic treatments for GVHD. Although development in this space has historically been slow, the pipeline for GVHD therapies is now robust with a number of late-phase agents, including Mesoblast’s Ryoncil (remestemcel-L), Incyte’s itacitinib, Kadmon’s belumosudil, Elsalys / Mediolanum's Leukotac (inolimomab), and Xenikos's T-Guard. Here, we examine the clinical and commercial opportunity of these emerging therapies and how they may affect the commercial performance of current therapies, such as AbbVie / Janssen’s Imbruvica and Incyte / Novartis’s Jakafi / Jakavi.
QUESTIONS ANSWERED
How large is the GVHD population, and how will it change through 2029? What percentage of the GVHD population receives drug treatment?
How is GVHD currently managed in the United States and EU5? How well does the GVHD pipeline align with unmet needs in the space?
Which emerging therapies do experts consider most promising? If approved, how would emerging therapies affect the management of GVHD? Which emerging drugs hold the most commercial potential?
What are some of the key GVHD market drivers and constraints? How will continuing advances in patient and host human leukocyte antigen (HLA) matching affect the market?
CONTENT HIGHLIGHTS
Geographies: United States and EU5.
Primary research: Six country-specific interviews with thought-leading GVHD specialists, supported by survey data collected for this study.
Epidemiology: Diagnosed incident cases of acute and chronic GVHD, diagnosed incident cases of acute GVHD by grade, drug-treated cases of GVHD by country.
Forecast: Drug-level sales and patient share of key GVHD therapies in 2029.
Emerging therapies: Late phase / preregistered: 6 drugs; Phase II: >10 drugs; coverage of select preclinical and Phase I products.
Clinical Trial Outcomes for Antithymocyte Globulin
Key Results of Select Clinical Trials Investigating Antithymocyte Globulin for Graft Versus Host Disease Prophylaxis
Advantages and Disadvantages of Antithymocyte Globulin
Key Ongoing Clinical Trials of Antithymocyte Globulin for Graft Versus Host Disease Prophylaxis
Expert Insights: Antithymocyte Globulin
Clinical Trial Outcomes for Post-Transplant Cyclophosphamide
Advantages and Disadvantages of Post-Transplant Cyclophosphamide
Key Ongoing Clinical Trials of Post-Transplant Cyclophosphamide for Graft Versus Host Disease Prophylaxis
Expert Insights: Post-Transplant Cyclophosphamide
Clinical Trial Outcomes for Systemic Corticosteroids
Key Results of Select Clinical Trials Investigating Systemic Corticosteroids for the Treatment of Graft Versus Host Disease
Advantages and Disadvantages of Systemic Corticosteroids
Expert Insights: Systemic Corticosteroids
Clinical Trial Outcomes for Imbruvica
Key Results from Select Clinical Trials Investigating Imbruvica for Graft Versus Host Disease Treatment
Advantages and Disadvantages of Imbruvica
Key Ongoing Clinical Trials of Imbruvica for Graft Versus Host Disease
Expert Insights: Imbruvica
Clinical Trial Outcomes for Extracorporeal Photopheresis
Key Results from Select Clinical Trials Investigating Extracorporeal Photopheresis for Graft Versus Host Disease Prophylaxis
Advantages and Disadvantages of Extracorporeal Photopheresis
Medical Practice
Overview
Regional Graft Versus Host Disease Treatment Guidelines
Generalized Treatment Decision Tree for Acute Graft Versus Host Disease
Generalized Treatment Decision Tree for Chronic Graft Versus Host Disease
Unmet Need Overview
Current and Future Attainment of Unmet Needs in Graft Versus Host Disease
Top Unmet Needs in Graft Versus Host Diseaseu2014Current and Future Attainment
Expert Insight on Unmet Needs in Graft Versus Host Disease
Emerging Therapies
Key Findings
Pipeline Trends for Graft Versus Host Disease
Key Emerging Therapies
Key Therapies in Development for Graft Versus Host Disease
Estimated Launch Dates of Key Emerging Therapies for Graft Versus Host Disease
Jakafi / Jakavi Profile
Analysis of Clinical Development Program for Jakafi / Jakavi
Key Ongoing Clinical Trials of Jakafi / Jakavi in the Treatment of Graft Versus Host Disease
Expert Insight: Jakafi / Jakavi
Expectations for Launch and Sales Opportunity of Jakafi / Jakavi for Graft Versus Host Disease
Itacitinib Profile
Analysis of Clinical Development Program for Itacitinib
Key Ongoing Clinical Trials of Itacitinib in the Treatment of Graft Versus Host Disease
Expert Insights: Itacitinib
Expectations for Launch and Sales Opportunity of Itacitinib in Graft Versus Host Disease
Ryoncil Profile
Analysis of Clinical Development Program for Ryoncil
Expert Insights: Ryoncil
Expectations for Launch and Sales Opportunity of Ryoncil in Graft Versus Host Disease
UVADEX Profile
Analysis of Clinical Development Program for UVADEX
Expert Insight: ECP
Expectations for Launch and Sales Opportunity of UVADEX in Graft Versus Host Disease
Alpha-1 Antitrypsin Profile
Analysis of Clinical Development Program for Alpha-1 Antitrypsin
Key Ongoing Clinical Trials of Alpha-1 Antitrypsin in the Treatment of Graft Versus Host Disease
Expert Insights: Alpha-1 Antitrypsin
Expectations for Launch and Sales Opportunity of Alpha-1 Antitrypsin in Graft Versus Host Disease
Belumosudil Profile
Analysis of Clinical Development Program for Belumosudil
Expert Insights: Belumosudil
Expectations for Launch and Sales Opportunity of Belumosudil in Graft Versus Host Disease
Inolimomab
Inolimomab Profile
Analysis of Clinical Development Program for Inolimomab
Key Clinical Trials of Inolimomab in the Treatment of Graft Versus Host Disease
Expert Insights: Inolimomab
Expectations for Launch and Sales Opportunity of Inolimomab in Graft Versus Host Disease
T-Guard Profile
Analysis of Clinical Development Program for T-Guard
Expert Insights: T-Guard
Expectations for Launch and Sales Opportunity of T-Guard in Graft Versus Host Disease
Vedolizumab
CD24Fc
Expert Insights
Early-Phase Pipeline Analysis
Select Compounds in Phase II Development for Graft Versus Host Disease
Select Compounds in Early-Phase Development for Graft Versus Host Disease
Patient Registries
Patient Registries and Resources for Donor Bone Marrow
Prominent Graft Versus Host Disease Patient Organizations
Orphan Drug Designation
Access & Reimbursement Overview
Region-Specific Reimbursement Practices
General Reimbursement Environment: United States
General Reimbursement Environment: EU5
Appendix
Key Abbreviations Related to Graft Versus Host Disease
Brands, Marketers, and Generic Availability of Key Therapies for Graft Versus Host Disease, by Market
Graft Versus Host Disease Bibliography
Andrew Russell, Ph.D.
Andrew Russell, Ph.D., is a business insights analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. In this role, he authors reports that distill the findings of his research into pharmaceutical markets related to autoimmune disease and bacterial infection. He worked previously in neurodegenerative disease research at Brigham and Women’s Hospital in Boston. He holds a Ph.D. in cellular and molecular biology from Brandeis University.