FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases

FDAThe U.S. Food and Drug Administration today announced that it has awarded 12 new clinical trial research grants totaling more than $15 million over the next four years to enhance the development of medical products for patients with rare diseases. The grants were awarded to principal investigators from academia and industry across the country.

The FDA awarded the grants through the Orphan Products Clinical Trials Grants Program, funded by Congress to encourage clinical development of drugs, biologics, medical devices and medical foods for the treatment of rare diseases. The grants are intended to substantially contribute to marketing approval of products to treat rare diseases or provide essential data needed for development of such products.

"For more than 35 years, the FDA has been providing much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. To date, the Orphan Products Clinical Trials Grants Program's grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases," said FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D. "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."

The FDA received 89 clinical trial grant applications that were reviewed and evaluated for scientific and technical merit by more than 100 rare disease experts, including members of academia.

The grants awarded are focused on supporting product development to meet the needs of patients impacted by a variety of rare diseases, mainly those affecting children and cancers. The recipients, principal investigators and approximate funding amounts, listed alphabetically, are:

  • Chemocentryx, Inc. (Mountain View, California), Peter Staehr, phase 2 study of avacopan for the treatment of complement 3 glomerulopathy - $1 million over two years
  • Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), Maryam Fouladi, phase 1 study of PTC596 for the treatment of diffuse intrinsic pontine glioma & high-grade gliomas - $750,000 over three years
  • Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio), Parinda Mehta, phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia - $1.7 million over four years
  • Columbia University Health Sciences (New York, New York), Gary Brittenham, phase 2 study of daily vitamin D for the treatment of sickle-cell respiratory complications - $2 million over four years
  • Cumberland Pharmaceuticals, Inc. (Nashville, Tennessee), Ines Macias-Perez, phase 2 study for oral ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy - $1 million over three years
  • Massachusetts General Hospital (Boston, Massachusetts), Sara Pai, phase 2 study of anti-PD1 therapy for the treatment of HPV-associated recurrent respiratory papillomatosis - $1 million over three years
  • New York Medical College (Valhalla, New York), Mitchell Cairo, phase 2 study of viral specific cytotoxic T-lymphocytes for the treatment of refractory viral infections and T-cell immunodeficiency - $1.7 million over four years
  • Privo Technologies, LLC. (Peabody, Massachusetts), Manijeh Goldberg, phase 1/2 study of cisplatin patch (PRV111) for the treatment of oral cancer - $2 million over four years
  • Targeted Therapy Technologies, LLC (Somerset, New Jersey), Ricardo Carvalho, phase 1 study of episcleral topotecan for the treatment of retinoblastoma - $660,000 over three years
  • University of Alabama at Birmingham (Birmingham, Alabama), Gregory Friedman, phase 1 study of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors - $750,000 over three years
  • University of California San Diego (La Jolla, California), Jason Sicklick, phase 2 study of temozolomide for the treatment of gastrointestinal stromal tumor - $1.5 million over three years
  • University of Texas MD Anderson Cancer Center (Houston, Texas), Michael Andreeff, phase 1/2 study of the imipridone (ONC201) for treatment of acute myeloid leukemia - $1 million over four years

"The majority of rare diseases still do not have approved therapies and the FDA is committed to fostering product development in areas of unmet need. The Orphan Products Grants Program is one of several ways that the FDA supports the development of products for rare diseases. Since its creation in 1983, the program has provided more than $400 million to fund more than 600 new clinical studies," said Janet Maynard, M.D., director of the FDA's Office of Orphan Products Development. "We are pleased to continue to support research for a variety of rare diseases that have little, or no, treatment options for patients. 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."

Three-quarters (75%) of the new awards fund studies enrolling children, including children as young as one month. These studies target a variety of rare diseases affecting children and have the potential to advance treatments for these diseases. Some of these diseases include Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle loss and weakness; sickle cell disease, a group of inherited red blood disorders which can cause anemia, infections and stroke; and Fanconi Anemia, a rare inherited condition that can result in bone marrow failure and has a high risk for squamous cell cancers.

Two-thirds (67%) of the new awards fund clinical studies of products for use in various rare cancers. For example, one of the new awards aims to advance research in brain cancers affecting children. This study enrolls affected children as young as 36 months and tests an innovative treatment approach to treat brain cancers using an engineered virus therapy designed to target and kill tumor cells while sparing normal brain cells. Another newly funded study evaluates 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. This delivery system may help address current barriers to drug delivery that patients face.

Past studies funded by this program that have resulted in or contributed to recent product approvals, include: fish oil triglycerides, a first FDA-approved lipid emulsion made from fish oil, approved as a source of calories and fatty acids in children with parenteral nutrition-associated cholestasis; and the first treatment (tafamidis meglumine and tafamidis) for heart disease caused by transthyretin mediated amyloidosis.

In addition to the FDA Orphan Products Clinical Trials Grants Program, the agency also supports natural history studies for rare diseases to further advance the FDA's mission of bringing new therapies to approval and patients.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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