FDA funds studies of 6 rare-disease drugs

The FDA announced that it awarded six new clinical trial research grants to principal investigators totaling more than $16 million over four years.

Awarded through the Congressionally-funded Orphan Products Grants Program, the research grants seek to enhance the development of medical products for patients with rare diseases, according to a news release.

More than 90 rare disease and clinical trial experts reviewed and evaluated the merit of 47 grant applications before deciding on the six winners for which grants would be awarded to support the studies of products addressing unmet needs in those rare diseases or conditions, or ones which provide highly significant improvements in treatment or diagnosis.

The six selected companies/institutions are:

• Acucela, Inc. (Seattle), 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), 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), 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, N.Y.), 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), 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, Va.), 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.

“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,” FDA Commissioner Dr. Stephen Hahn said in the news release. “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.”