Niche Markets and Rare Diseases

November 2012

Niche Markets and Rare Diseases: Graft Versus Host Disease

Report Authors
Benjamin Guikema, Ph.D.
Gilan Megeed, M.P.H.
  • Pages:83
  • Tables:34
  • Figures:6
  • Citations:79
  • Drugs:39
  • Interviews:8


Graft versus host disease (GVHD) is a life-threatening complication that hampers the efficacy of hematopoietic stem cell transplants (HCST) to treat hematological malignancies. Although the late-stage pipeline is small and underproductive, GVHD—which is difficult to prevent and poorly responsive to current treatments—presents opportunities for drug developers. Physicians are particularly dissatisfied with therapeutic options for treatment-refractory GVHD and are already willing to use almost any drug supported by a reasonable mechanistic theory, which bodes well for rapid uptake of novel GVHD drugs. Late-stage candidates are unlikely to displace patient-share-leading steroids or calcineurin inhibitors in preventing and treating GVHD, but the expected supplemental approval of bortezomib (Millennium [Takeda]/Johnson & Johnson’s Velcade) for the treatment of cGVHD will be a significant near-term growth driver in the GVHD market.

Questions Answered in This Report:

  *   Despite routine administration of prophylactic regimens, GVHD remains a common and potentially life-threatening complication of HSCT. How do steroids, calcineurin inhibitors, cytotoxic small molecules, and targeted biologics factor into first-line treatment? How do hematologist/oncologists navigate treatment choices for steroid-refractory GVHD, given a lack of highly effective or well-supported options?

  *   Current therapies primarily target T-cells, the major pathogenic mediators of GVHD. What emerging targets hold the most promise for new therapeutic approaches, according to thought leaders? Is the GVHD pipeline dominated by agents with novel targets or by “me too” products?

  *   We forecast that four agents currently in late stage development will launch for GVHD during the forecast period (2011-2021). Will steroids and calcineurin inhibitors lose majority patient share in GVHD prophylaxis and up-front treatment? Will novel therapies establish niches as treatment options for high-risk or treatment-refractory patients?

  *   As a rare disease, GVHD qualifies as an orphan indication in both the United States and Europe. Is support during clinical development, accelerated approval processes, and extended market exclusivity enticing drug developers into this market? Do thought-leading physicians believe orphan drug designations have encouraged activity in GVHD drug development?


Markets covered: United States, France, Germany, Italy, Spain, United Kingdom.

Primary research: 8 country-specific interviews with thought-leading hematologist/oncologists.

Epidemiology: Incident HSCT, acute GVHD, and chronic GVHD cases.

Population segments: Prophylaxis recipients, treatment-naive patients, and treatment-refractory patients.

Emerging therapies: Phase II: 1 drug; Phase III: 2 drugs; preregistration: 1 drug. Coverage of 6 select preclinical and Phase I products.

Fill out the form below to contact sales.

Search Reports

Mentioned in this report:

  • - Alder Pharmaceuticals
  • - Amgen
  • - Apogenix
  • - Astellas
  • - Athersys
  • - Biogen Idec
  • - Dava Pharmaceuticals
  • - Fresenius Biotech
  • - Galenica
  • - Genentech
  • - Immunomedics
  • - Genzyme
  • - GlaxoSmithKline
  • - Imtix Sangstat
  • - Janssen Biotech
  • - Jazz Pharmaceuticals
  • - Johnson & Johnson
  • - Merck
  • - Merck KGaA
  • - Millennium
  • - Mitsubishi Tanabe
  • - MolMed
  • - Monarch
  • - Novartis
  • - Osiris Therapeutics
  • - Pfizer
  • - Roche
  • - Sanofi
  • - Takara Bio
  • - Takeda
Decision Resources Group brands include: