The U.S. Food and Drug Administration today announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics.
The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.
“The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program,” said Gayatri R. Rao, M.D., director of the FDA’s Office of Orphan Product Development. “The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”
The program is administered through the FDA’s Orphan Products Grants Program. This program was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.
A panel of independent experts with experience in the disease-related fields reviewed the grant applications and made recommendations to the FDA.
The 2014 grant recipients are:
- Denise Adams, Cincinnati Children's Hospital Medical Center (Cincinnati, Ohio), Phase 2 Study of Vincristine vs. Sirolimus for the Treatment of High Risk Kaposiform Hemangioendothelioma—$1.6 million over four years
- Mitesh Borad, Mayo Clinic Arizona (Scottsdale, Ariz.), Phase 1 Study of VSV-hIFN-B for the Treatment of Hepatocellular Carcinoma— approximately $600,000 over three years
- Andrew Brenner, University of Texas Health Center San Antonio (San Antonio, Texas), Phase 2 Study of TH-302 for the Treatment of Glioblastoma— approximately $1.6 million over four years
- Kelly Dooley, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of PA-824 for the Treatment of Pulmonary Tuberculosis— $1.6 million over four years
- Donald Durden, University of California San Diego (San Diego, Calif.), Phase 2 Study of Poly-ICLC for the Treatment of Pediatric Low Grade Gliomas— $1.6 million over four years
- Alfred Lane, Stanford University (Stanford, Calif.), Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations— approximately $1.6 million over four years
- Dung Le, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Folfirinox followed by Ipilimumab/GVAX for the Treatment of Pancreatic Cancer— $1.6 million over four years
- Phillip Low, Mayo Clinic Rochester (Rochester, Minn.), Phase 1 Study of Intrathecal Autologous Mesenchymal Stem Cell Therapy for the Treatment of Multiple System Atrophy—$600,000 over three years
- Guido Magni, River Vision Development Corporation (New York, N.Y.), Phase 2 Study of RV001 for the Treatment of Thyroid Eye Disease—$1.2 million over three years
- Michael Portman, Seattle Children's Hospital (Seattle, Wash.), Phase 3 Study of Triiodothyronine Supplementation for the Treatment of Young Infants After Cardiopulmonary Bypass— approximately $1.6 million over four years
- Jana Portnow, City of Hope Beckman Research Institute (Duarte, Calif.), Phase 1 Study of Neural Stem Cells & 5-FC/Leucovorin for the Treatment of Recurrent High Grade Gliomas—$600,000 over three years
- Scott Rollins, Selexys Pharmaceuticals Corporation (Oklahoma City, Okla.), Phase 2 Study of SelG1 for the Treatment of Sickle Cell Disease— $1.6 million over four years
- Beena Sood, Wayne State University (Detroit, Mich.), Phase 1/2 Study of Aerosolized Survanta for the Treatment of Neonatal Respiratory Distress Syndrome— approximately $1.4 million over four years
- Warren Stern, Aesrx, LLC (Newton, Mass.), Phase 2 Study of Aes103 (5-HMF) for the Treatment of Stable Sickle Cell Disease—approximately $1.6 million over four years
- Pamela Zeitlin, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Digitoxin for the Treatment of Cystic Fibrosis— approximately $290,000 for one year
For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the United States. There are about 7,000 rare diseases and conditions, according to the National Institutes of Health. In total, nearly 30 million Americans suffer from at least one rare disease.
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.