Graft versus host disease (GVHD) is a potentially life-threatening complication of an allogeneic hematopoietic stem cell transplant (HCST). Improvements in allogeneic HSCT strategies have expanded indications for HSCT and improved outcomes for patients. However, the efficacy of HSCT is limited by a common and potentially lethal complication, GVHD, a systemic inflammatory reaction triggered by donor T cells’ recognition of host cell antigens in non-target tissue as foreign.

The GVHD market has historically been a slow-moving market in the pharmaceutical industry owing to the lack of development of new therapies. In May 2012, Osiris received the world’s first government approval to market a stem cell drug. Interviewed experts expect the mesenchymal stem cell emerging therapy Prochymal to reach the market and that it could see use as a solid second-line treatment option by 2024 for patients with aGVHD who fail to respond to steroid treatment. At the other end of the spectrum for preventive therapy is the emerging gene therapy TK. The infusion of TK engineered T cells is a therapeutic approach that should improve the chance of having a successful haploidentical allogeneic HSCT without the development of GVHD and improved survival. Given that fully compatible donors are available for approximately 50% of patients, many patients will receive a haploidentical allogeneic HSCT and are at a greater risk of developing GVHD. The stage is set for new therapeutic options unlike any of the current therapies for GVHD and competitors rush to enter what has been a slow-growing GVHD therapeutics market but with an enormous amount of opportunity.

This report provides an overview of the GVHD market in the United States and five major European markets (France, Germany, Italy, Spain, and the United Kingdom) including a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. The findings described in this report are derived from detailed interviews with expert U.S. and European specialists, secondary research, and best-in-class epidemiological analysis. This report provides deep insights into this complex and evolving clinical space, and includes a detailed analysis of specific opportunities for current and emerging therapies, including experts’ views on which unmet needs have not yet been addressed by developers of new therapies.

Questions Answered in This Report:

  • GVHD is a rare indication affecting only a small percentage of the population. What is the size of the GVHD patient population in the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) and how will the population change over the ten-year forecast period?

  • GVHD therapies in Phase III are on the cusp of potentially changing treatment options that have been in place for 20+ years. How will the emerging therapies reshape the treatment paradigm? What considerations will drive physician choice between the emerging therapies?

  • The pathogenesis of GVHD involves multiple immune signaling pathways. What new insights are informing experts’ understanding of GVHD? What is the current treatment landscape and medical practice in GVHD?

  • Unmet needs in GVHD are many and GVHD experts cite those that are of clear importance. What unmet needs are expected to remain unaddressed by mid– to late-stage emerging therapies for GVHD? What are the key avenues of preclinical and clinical research and development? What therapies are in early-stage development?


Market covered: United States, France, Germany, Italy, Spain, and the United Kingdom.

Primary research: Eight country-specific interviews with thought-leading GVHD specialists.

Epidemiology: Diagnosed incident cases of GVHD; diagnosed incident cases of allogeneic HSCT.

Emerging Therapies: Phase III: 3; coverage of select Phase I and II products.

Author(s): Kendra N.Taylor, Ph.D., M.M.Sc.
Kristin Dorfman, M.P.H., M.S.