FDA Issues Grants for Clinical Trial Research for Orphan Diseases

In an effort to enhance development of medical products for orphan diseases (or rare diseases), the U.S. Food and Drug Administration announced on October 8, 2020 that it awarded six new grants for clinical trial research, including a grant for ustekinumab (STELARA®) for the prevention of graft versus host disease.  STELARA®, marketed by Janssen Biotech, is currently approved for the treatment of Crohn’s disease, ulcerative colitis, psoriasis, and psoriatic arthritis.

Forty seven grant applications were reviewed and evaluated by more than 90 rare disease and clinical trial experts.  The six awarded grants were for clinical research in Stargardt disease, graft versus host disease, CF patients with nontuberculosis mycobacterium, CD4 positive T cell neoplasms, pancreatic cancer, and peripheral T-cell lymphoma.  “Now, more than ever, we see the important role of these FDA grants to support clinical trials of potentially life-changing treatments for patients with rare diseases,” said FDA Commissioner Stephen M. Hahn, M.D. “As interest in the program has grown, so has the houlcerative colitispe for promising approved therapies for patients with rare diseases that currently have no treatment options. These important clinical trials are at a critical time where additional resources are needed to support rare disease research during the COVID-19 pandemic.”