FDA Awards Grants for Rare Diseases Clinical Trials

The U.S. Food and Drug Administration (FDA) has awarded six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years. These trial research grants, awarded through the Congressionally-funded Orphan Products Grants Program, enhance the development of medical products for patients with rare diseases.

"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 hope 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."

The FDA received 47 clinical trial grant applications that 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 awarded support clinical studies of products that address unmet needs in rare diseases or conditions, or provide highly significant improvements in treatment or diagnosis. Below is a complete list in alphabetical order:

Acucela (Seattle, Washington), Ryo Kubota, phase 3 study of emixustat hydrochloride for the treatment of Stargardt disease – $1.6 million over three years.

Fred Hutchinson Cancer Research Center (Seattle, Washington), Stephanie Lee, phase 2 study of ustekinumab for the prevention of graft versus host disease – $3.5 million over four years.

Seattle Children's Hospital (Seattle, Washington), Christopher Goss, phase 1b study of IV gallium nitrate for the treatment of cystic fibrosis patients colonized with nontuberculosis mycobacterium – $3 million over four years.

State University of New York Stony Brook (Stony Brook, New York), Huda Salman, phase 1 study of CD4 redirected chimeric antigen receptor T cell therapy for the treatment of CD4 positive T cell neoplasms – $3.1 million over four years.

University of Cincinnati (Cincinnati, Ohio), Devendra Sohal, phase 1/2 study of ABTL0812 (a small molecule with anti-cancer activity) for the treatment of pancreatic cancer – $1.9 million over four years.

University of Virginia (Charlottesville, Virginia), Owen O'Connor, phase 2 study of oral azacytidine plus romidepsin for the treatment of peripheral T-cell lymphoma – $3.2 million over four years.

"Since its inception over 35 years ago, the Office of Orphan Products Development has successfully continued to support necessary research for rare diseases to inform product development, support scientific advancements, contribute to the development of treatment guidelines, and result in marketing approvals especially for rare diseases without prior-approved therapies. This grants program continues to fill important gaps in rare disease product development, such as supporting recent FDA approvals that help address unmet needs in the treatment of rare diseases," said Janet Maynard, M.D., M.H.S., director of OOPD.

Examples of recent approvals supported by the grants program include teprotumumab, for the treatment of a rare thyroid eye disease, and triheptanoin, a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.

This year, as there are new challenges and increased costs for clinical trials due to the COVID-19 pandemic, the FDA remains committed to supporting rare disease research by providing existing grantees with additional funding. These new resources allow ongoing studies to implement necessary steps to allow their research to continue and assure the safety of study participants, to maintain compliance with good clinical practice, and to minimize risks to trial integrity.