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