FDA Awards Six Grants to Fund New Clinical Trials of Medical Products for Treatment of Rare Diseases
The U.S. Food and Drug Administration (FDA) announced it has awarded six new clinical trial research grants to principal investigators from academia and industry, totaling more than $16 million over the next 4 years. These grants, awarded through the Congressionally funded Orphan Products Grants Program, are intended to enhance the development of medical products for patients with rare diseases.
The FDA received 47 clinical trial grant applications, which were reviewed and evaluated for scientific and technical merit by more than 90 rare disease and clinical trial experts, including members of academia. The grants were awarded to:
- Ryo Kubota, MD, PhD, Acucela, Inc: Phase III study of emixustat hydrochloride to treat Stargardt disease ($1.6 million over 3 years)
- Stephanie Lee, MD, MPH, Fred Hutchinson Cancer Research Center: Phase II study of ustekinumab for the prevention of graft-vs-host disease ($3.5 million over 4 years)
- Christopher Goss, MD, Seattle Children’s Hospital: Phase Ib study of intravenous gallium nitrate for the treatment of cystic fibrosis colonized with nontuberculosis mycobacterium ($3 million over 4 years)
- Huda Salman, MD, State University of New York Stony Brook: Phase I study of CD4-redirected chimeric antigen receptor T-cell therapy for CD4-positive T-cell neoplasms ($3.1 million over 4 years)
- Devendra Sohal, MD, MPH, University of Cincinnati: Phase I/II study of ABTL0812 (a small molecule with anticancer activity) in the treatment of pancreatic cancer ($1.9 million over 4 years)
- Owen O’Connor, MD, MPH, University of Virginia: Phase II study of oral azacitidine plus romidepsin in the treatment of peripheral T-cell lymphoma ($3.2 million over 4 years).