To advance the development of medical products for the treatment of rare diseases mainly those affecting children and cancers
The US Food and Drug Administration announced that it has awarded 12 new clinical trial research grants totalling 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 grants awarded are focussed on supporting product development to meet the needs of patients impacted by a variety of rare diseases, mainly those affecting children and cancers.
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 Amy Abernethy, Principal Deputy Commissioner, FDA. “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 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 programme has provided more than $400 million to fund more than 600 new clinical studies,” said Janet Maynard, Director of the FDA’s Office of Orphan Products Development.
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