Biomarkers Pinpoint Immune Dysfunction in Patients with GVHD, FDA Guidance Says
Developers of graft-versus-host disease (GVHD) therapies can design trials based on a new FDA draft guidance that focuses on using biomarkers to identify individual patients’ specific immune dysfunction rather than suppressing the entire immune system.
Traditional treatment of GVHD has depended largely on drugs that impair T cells, the FDA says, often resulting in “profound immunosuppression.” But recent research has helped identify other components of the adaptive immune system that can be targeted.
The 39-page draft guidance, “Graft-versus-Host Diseases: Developing Drugs, Biological Products, and Certain Devices for Prevention or Treatment,” applies to development of drugs, biologics and certain devices to prevent/treat acute and chronic GVHD occurring after allogeneic hematopoietic stem cell transplantation.
The guidance covers overall clinical development and design elements for early- and late-phase trials and delves into drug combinations, efficacy endpoints for prevention/treatment of acute GVHD and treatment of chronic GVHD, and recommendations for raw data submitted in marketing applications. It is not intended to provide advice on the technical aspects of therapeutic or cell-processing devices.
Read the GvHD guidance here.