The FDA Awards 12 Grants that Fund New Clinical Trials to Advance Development of Medical Products for Treatment of Rare Disease

Oct 9, 2019 | FDA, Financing, Grants, Rare Diseases

The U.S. Food and Drug Administration (FDA) has awarded 12 new clinical trial research grants amounting to over $15 million over the next four years. They seek to enhance the development of medicinal products for patients with rare diseases. The grants were awarded to both academia and industry principal investigators.

The Orphan Products Clinical Trials Grants Program

The funds originate from 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 more rare diseases or provide essential data needed for the development of such products.

The Grants

89 clinical trial grant applications were received, reviewed and evaluated by the FDA’s panel of expert reviewers. The winners focused on supporting product development to meet the needs of patients impacted by a variety of rare diseases, with an emphasis on children and cancers.  The recipients are principal investigators and approximate funding amounts listed below:

  • 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


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.

Lead Research/Contact

Amy Abernethy, M.D., Ph.D., FDA Principal Deputy Commissioner

Janet Maynard, M.D., director of the FDA’s Office of Orphan Products Development


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