US FDA awards six clinical trials research grants to develop medical products for rare diseases

The US Food and Drug Administration announced that it has awarded six new clinical trial research grants to principal investigators from academia and industry totalling 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.

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 colonised 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.

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 paediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.