Six Grants From the FDA Will Help Fund Critical Rare Disease Clinical Trials

According to a story from BioSpace, the US Food and Drug Administration (FDA) recently announced that it has awarded grant funding that will go towards six clinical trials that will be testing potential therapies for diseases that are considered rare. These grants combined provide $16 million in funding and were given through the Orphan Products Grants program, which is funded by Congress.

The agency received a total of 47 applications for these grants. These grants must go towards the investigation of experimental therapies that have the potential to fulfill an unmet need in rare disease or have the potential to offer substantive advantages over current treatment options.

The projects being funded are as follows:

  1. Ryo Kubota of Acucela, Inc., will be conducting a phase 3 trial of emixustat hydrochloride as a treatment for Stargardt disease. Funding will top out at $1.6 million over a three year period.
  2. Stephanie Lee of the Fred Hutchinson Cancer Research Center will be leading a phase 2 clinical trial that will be testing ustekinumab as a preventative treatment for graft-versus-host disease (GvHD). Funding will total $3.5 million over a four year period.
  3. Christopher Goss of the Seattle Children’s Hospital will be conducting a phase 1b trial of gallium nitrate as a treatment for cystic fibrosis patients that are infected with nontuberculous mycobacterium. This funding will total $3 million over a four year period.
  4. Huda Salman of the State University of New York Stony Brook will lead a phase 1 study of CD4 redirected chimeric antigen receptor (CAR) T-cell therapy for T-cell neoplasms that are CD4 positive. This funding will top out at $3.1 million over a four year period.
  5. Devendra Sohal of the University of Cincinnati is conducting a phase 1/2 clinical trial testing ABTL0812 as a potential treatment for pancreatic cancer. This will total $1.9 million in funding over a four year period.
  6. Owen O’Connor of the University of Virginia is leading a phase 2 trial that will test a combination of azacytidine and romidepsin as a treatment for peripheral T-cell lymphoma. Funding totals $3.2 million over a four year period.

The Office of Orphan Products Development was first established more than 35 years ago and plays a valuable role in supporting rare disease product development and research.

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