The U.S. Food and Drug Administration (FDA) awarded $15 million in grants to fund 12 new clinical trials over the next four years to foster the development of rare disease treatments.

The grants were awarded through the FDA’s Orphan Products Clinical Trials Grants Program that was provided by Congress to specifically encourage the development of treatments for rare diseases. The grants are intended to substantially contribute to the marketing approval of products to treat rare diseases or provide essential data needed for the development of such products. The FDA selected the 12 recipients out of 89 different applications that were evaluated by more than 100 rare disease experts, the agency said.

FDA Principal Deputy Commissioner Amy Abernethy said the regulatory agency has provided “much-needed financial support” to clinical trials for rare disease treatments for more than three decades. Over that time, the more than $400 million provided by these grants has led to the approval of more than 60 different drugs for rare diseases, she said.

“We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases,” Abernathy said in a statement.

The 12 grant awards are primarily focused on discovering treatments for rare diseases affecting children, with about two-thirds looking at rare cancers, including a type of brain cancer. More than three-fourths of the trials will enroll children, including infants, the FDA said. Some of these diseases include Duchenne Muscular Dystrophy, sickle cell disease and Fanconi Anemia, a rare inherited condition that can result in bone marrow failure and has a high risk for squamous cell cancers. Another of the funded studies is evaluating a novel drug delivery system that delivers chemotherapy on a sustained basis directly to the eye to treat retinoblastoma, a rare cancer in the eye most commonly affecting young children.

Janet Maynard, director of the FDA’s Office of Orphan Products Development, said the majority of rare diseases do not have approved therapies and this grant program is aimed at the hope of developing treatments for these diseases.

“By helping to spark research, we hope to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most,” Maynard said.

The awarded grants are:

  • Chemocentryx, Inc. — $1 million over two years to support a Phase II study of avacopan for the treatment of complement 3 glomerulopathy
  • Cincinnati Children’s Hospital Medical Center — $750,000 over three years to support a Phase I study of PTC596 for the treatment of diffuse intrinsic pontine glioma & high-grade gliomas
  • Cincinnati Children’s Hospital Medical Center — $1.7 million over four years to fund a Phase II study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia
  • Columbia University Health Sciences — $2 million over four years for a Phase II study of daily vitamin D for the treatment of sickle-cell respiratory complications
  • Cumberland Pharmaceuticals, Inc. — $1 million over three years to support a Phase II study for oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy
  • Massachusetts General Hospital — $1 million over three years to fund a Phase II study of anti-PD1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis
  • New York Medical College — $1.7 million over four years to support a Phase II study of viral-specific cytotoxic T-lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency
  • Privo Technologies, LLC. — $2 million over four years to fund a Phase I/II study of cisplatin patch (PRV111) for the treatment of oral cancer
  • Targeted Therapy Technologies, LLC — A Phase 1 study of episcleral topotecan for the treatment of retinoblastoma will receive $660,000 in support over three years
  • University of Alabama at Birmingham — $750,000 over three years to support a Phase I study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors
  • University of California San Diego – A Phase II study of temozolomide for the treatment of gastrointestinal stromal tumor will received $1.5 million over three years
  • University of Texas MD Anderson Cancer Center – A Phase I/II study of imipridone for treatment of acute myeloid leukemia will receive $1 million over four years.



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